CPPA Certification Review

Certified Pharmacy Policy Analyst (CPPA) Review

A Review Guide for the Certified Pharmacy Policy Analyst (CPPA) Exam

Block 1: Foundations of Health Policy

A-C

  • ACA: Affordable Care Act.
  • AHRQ: Agency for Healthcare Research and Quality.
  • AMA: American Medical Association.
  • AMP: Average Manufacturer Price.
  • APhA: American Pharmacists Association.
  • ASHP: American Society of Health-System Pharmacists.
  • CBO: Congressional Budget Office.
  • CDC: Centers for Disease Control and Prevention.
  • CFR: Code of Federal Regulations.
  • CMS: Centers for Medicare & Medicaid Services.

D-H

  • DEA: Drug Enforcement Administration.
  • DSH: Disproportionate Share Hospital.
  • EBM: Evidence-Based Medicine.
  • ERISA: Employee Retirement Income Security Act.
  • FDA: Food and Drug Administration.
  • FFS: Fee-For-Service.
  • GAO: Government Accountability Office.
  • HHS: Department of Health and Human Services.
  • HIPAA: Health Insurance Portability and Accountability Act.
  • HRSA: Health Resources and Services Administration.

I-N

  • ICER: Institute for Clinical and Economic Review.
  • IOM: Institute of Medicine (now National Academy of Medicine).
  • IRA: Inflation Reduction Act.
  • IRB: Institutional Review Board.
  • MAC: Maximum Allowable Cost.
  • MCO: Managed Care Organization.
  • MedPAC: Medicare Payment Advisory Commission.
  • NAM: National Academy of Medicine.
  • NIH: National Institutes of Health.
  • NQF: National Quality Forum.

O-R

  • OIG: Office of Inspector General.
  • OMB: Office of Management and Budget.
  • P&T: Pharmacy and Therapeutics (Committee).
  • PBM: Pharmacy Benefit Manager.
  • PCORI: Patient-Centered Outcomes Research Institute.
  • PDUFA: Prescription Drug User Fee Act.
  • PHS: Public Health Service.
  • QALY: Quality-Adjusted Life Year.
  • RFI: Request for Information.
  • RFP: Request for Proposal.

S-Z

  • SAMHSA: Substance Abuse and Mental Health Services Administration.
  • SGR: Sustainable Growth Rate.
  • SPAP: State Pharmaceutical Assistance Program.
  • TRICARE: Military Health System.
  • UM: Utilization Management.
  • URAC: Utilization Review Accreditation Commission.
  • USC: United States Code.
  • USP: United States Pharmacopeia.
  • VA: Department of Veterans Affairs.
  • VBP: Value-Based Purchasing.

Definition and Scope of Health Policy

  • Health policy refers to the decisions, plans, and actions that are undertaken to achieve specific healthcare goals within a society.
  • It is a field of study and practice concerned with the allocation of resources to health.
  • Pharmacy policy is a subfield that focuses specifically on medications and pharmacy services.
  • The scope is broad, covering drug approval, pricing, reimbursement, access, and safety.
  • It encompasses laws passed by legislatures (statutes), rules created by agencies (regulations), and decisions made by courts (judicial law).
  • It also includes the internal policies of healthcare organizations and insurance companies.
  • A CPPA analyzes these policies to understand their impact on patients, providers, and the healthcare system.
  • The ultimate goal of health policy is to improve the health of the population.

The U.S. Healthcare System Structure

  • The U.S. has a complex, pluralistic healthcare system with a mix of public and private financing.
  • There is no single, unified system; it is a patchwork of different programs and payers.
  • The private sector is dominated by employer-sponsored insurance.
  • The public sector includes major programs like Medicare (for the elderly and disabled) and Medicaid (for the low-income population).
  • Other public programs include the VA and TRICARE for military personnel.
  • This fragmentation creates significant challenges for policy development and implementation.
  • A CPPA must understand the structure of this system and the roles of its different components.
  • The interaction between the public and private sectors is a key area of policy analysis.

The Policy Cycle

  • The policy cycle is a conceptual model that describes the process by which policies are made.
  • Problem Identification & Agenda Setting: A problem is recognized as a public issue requiring government action.
  • Policy Formulation: Various policy options are developed to address the problem.
  • Policy Adoption: A specific policy is formally adopted through legislation or rulemaking.
  • Policy Implementation: The adopted policy is put into action by government agencies.
  • Policy Evaluation: The results of the policy are assessed to determine its effectiveness.
  • This cycle is iterative, as the evaluation often leads to the identification of new problems.
  • A CPPA can be involved at every stage of this cycle, from analyzing problems to evaluating outcomes.

Key Federal Agencies in Pharmacy Policy

  • Department of Health and Human Services (HHS): The main federal agency for protecting the health of Americans.
  • Food and Drug Administration (FDA): A branch of HHS responsible for ensuring the safety and efficacy of drugs and medical devices.
  • Centers for Medicare & Medicaid Services (CMS): A branch of HHS that administers the Medicare and Medicaid programs. CMS policies have an enormous impact on pharmacy reimbursement.
  • Health Resources and Services Administration (HRSA): A branch of HHS that oversees programs for underserved populations, including the 340B Drug Pricing Program.
  • Drug Enforcement Administration (DEA): Part of the Department of Justice, responsible for enforcing the Controlled Substances Act.
  • A CPPA must be an expert on the roles and jurisdictions of these key agencies.

State-Level Policy Making

  • In the U.S. federalist system, states have significant authority over healthcare policy.
  • States are the primary regulators of the practice of pharmacy through their State Boards of Pharmacy.
  • They are also the primary regulators of private health insurance.
  • Each state administers its own Medicaid program, leading to 50+ different sets of rules.
  • States often act as "laboratories of democracy," testing new policy ideas that are later adopted at the federal level.
  • A CPPA must be knowledgeable about the policy-making process at the state level, including the role of the state legislature and state agencies.
  • This local expertise is critical, as state policy often has a more direct and immediate impact on pharmacy practice than federal policy.

Block 2: Policy Analysis & Evaluation

The Purpose of Policy Analysis

  • Policy analysis is the systematic evaluation of a policy problem or proposed solution.
  • Its purpose is to provide objective, evidence-based information to policymakers to help them make better decisions.
  • It is not the same as policy advocacy, which involves arguing for a specific position.
  • The analyst's role is to be a neutral and objective source of information.
  • The analysis should clarify the problem, identify the potential options, and assess the likely consequences of each option.
  • A CPPA uses these skills to analyze proposed laws, regulations, or organizational policies.

Problem Structuring

  • The first and most important step in policy analysis is to clearly define the problem.
  • A poorly defined problem will lead to a poor analysis.
  • This involves gathering data to understand the magnitude and scope of the problem.
  • It also involves identifying the root causes of the problem, not just its symptoms.
  • The problem should be stated in a clear, concise, and quantifiable way whenever possible.
  • For example, instead of "drug prices are too high," a better problem definition is "patient out-of-pocket spending on specialty drugs has increased by 15% annually for the past five years."
  • A CPPA must be skilled at this initial problem-framing stage.

Identifying and Evaluating Policy Options

  • Once the problem is defined, the next step is to identify a range of potential policy options to address it.
  • This should always include the "status quo" (doing nothing) as a baseline for comparison.
  • Each option must then be evaluated against a set of criteria.
  • Common evaluation criteria include:
  • Effectiveness: How well will the option solve the problem?
  • Efficiency/Cost: What are the costs of the option, and do the benefits justify them?
  • Equity: How will the option affect different population groups? Is it fair?
  • Political Feasibility: Is the option likely to be supported by key stakeholders and policymakers?
  • Administrative Feasibility: Can the option be realistically implemented?

Bardach's Eightfold Path

  • This is a well-known, practical framework for conducting policy analysis.
  • Step 1: Define the Problem.
  • Step 2: Assemble Some Evidence.
  • Step 3: Construct the Alternatives (policy options).
  • Step 4: Select the Criteria for evaluation.
  • Step 5: Project the Outcomes (forecast the consequences of each option).
  • Step 6: Confront the Trade-offs.
  • Step 7: Decide! (make a recommendation).
  • Step 8: Tell Your Story (communicate the analysis).
  • This framework provides a structured and logical path for a CPPA to follow.

Presenting Policy Analysis

  • The final step of the analysis is to communicate the findings.
  • The format is typically a policy brief or a memo.
  • The writing must be clear, concise, and aimed at a busy, non-expert audience.
  • It should start with a short executive summary that states the problem and the key recommendation.
  • It should present the evidence and the analysis of the options in a logical and easy-to-follow manner.
  • Visual aids like charts and tables can be very effective.
  • The goal is to provide a useful and actionable document that informs the decision-making process.
  • A CPPA must be an excellent writer and communicator.

Quantitative vs. Qualitative Research

  • Quantitative Research: Deals with numbers and statistical analysis. It is used to measure, test hypotheses, and identify correlations.
  • Examples include analyzing claims data to measure the impact of a policy on drug spending.
  • Qualitative Research: Deals with non-numerical data, such as text from interviews or focus groups. It is used to understand concepts, opinions, and experiences.
  • An example would be interviewing pharmacists to understand their perceptions of a new workflow.
  • A good policy analysis often uses a mix of both quantitative and qualitative methods.

Key Data Sources in Pharmacy Policy

  • Administrative/Claims Data: Data from insurance claims (e.g., from Medicare or large commercial databases) is a primary source for quantitative analysis.
  • Survey Data: National surveys like the National Health Interview Survey (NHIS) or the Medical Expenditure Panel Survey (MEPS) provide rich data on health status and utilization.
  • Clinical Data: Data from Electronic Health Records (EHRs) can be used to analyze prescribing patterns and clinical outcomes.
  • Government Data: Data from agencies like the FDA (e.g., adverse event reports) or the CDC (e.g., public health statistics).
  • A CPPA must be familiar with these key data sources and their strengths and limitations.

Literature Review

  • A literature review is a systematic search and synthesis of the existing research on a topic.
  • It is a foundational step in any policy analysis.
  • It helps to define the problem, understand what is already known, and identify gaps in the evidence.
  • Key databases for searching the medical and health policy literature include PubMed, Embase, and Google Scholar.
  • A CPPA must be skilled at conducting a literature review and critically appraising the quality of the studies they find.

Program Evaluation

  • Program evaluation is the systematic assessment of a program's design, implementation, and outcomes.
  • It is a key part of the "Evaluation" stage of the policy cycle.
  • It seeks to answer questions like: Did the program achieve its goals? What were the unintended consequences? Was it cost-effective?
  • There are various research designs for program evaluation, ranging from simple pre-post studies to more rigorous randomized controlled trials.
  • A CPPA may be involved in designing or interpreting program evaluations.

Health Economics and Outcomes Research (HEOR)

  • HEOR is a discipline that evaluates the clinical and economic value of medical interventions.
  • Key types of HEOR studies include:
  • Cost-Effectiveness Analysis (CEA): Compares the costs and health outcomes (e.g., QALYs) of different interventions.
  • Budget Impact Analysis (BIA): Forecasts the financial impact of adopting a new drug on a health plan's budget.
  • Payers like PBMs and organizations like ICER heavily use HEOR to make formulary decisions.
  • A CPPA must have a strong understanding of the basic principles of HEOR.

How a Bill Becomes a Law (Federal)

  • A bill is introduced by a member of Congress (House or Senate).
  • It is referred to a committee with jurisdiction over the issue.
  • The committee holds hearings, "marks up" (amends), and votes on the bill.
  • If it passes the committee, it goes to the full chamber (House or Senate) for debate and a vote.
  • If it passes one chamber, it is sent to the other chamber to go through the same process.
  • If both chambers pass the same version of the bill, it is sent to the President.
  • The President can sign the bill into law or veto it.
  • A CPPA who engages in advocacy must understand this complex process.

The Role of Congressional Committees

  • Most of the detailed work of legislating happens in committees.
  • Key committees for pharmacy policy include:
  • Senate Finance Committee: Has jurisdiction over Medicare and Medicaid.
  • Senate HELP Committee: (Health, Education, Labor, and Pensions) Has jurisdiction over the FDA and public health.
  • House Ways and Means Committee: The House equivalent of Senate Finance.
  • House Energy and Commerce Committee: The House equivalent of Senate HELP.
  • A CPPA should know which committees are responsible for which issues.

The Regulatory Process (Rulemaking)

  • When Congress passes a law, it often gives a federal agency (like HHS) the authority to "fill in the details."
  • This is done through a process called rulemaking.
  • The agency first publishes a Notice of Proposed Rulemaking (NPRM) in the Federal Register.
  • This proposed rule explains how the agency intends to implement the law.
  • There is then a public comment period where anyone can submit feedback on the proposed rule.
  • The agency must review and consider all comments.
  • Finally, the agency publishes a Final Rule, which has the force of law.
  • A CPPA often analyzes and writes comments on proposed rules on behalf of their organization.

Advocacy and Lobbying

  • Advocacy is the process of influencing public policy.
  • This can be done by providing information, building relationships, and making a case for a particular position.
  • Lobbying is a specific type of advocacy that involves direct communication with policymakers about legislation.
  • Professional associations like APhA and ASHP are major advocates for the pharmacy profession.
  • A CPPA may be involved in their organization's advocacy efforts.
  • This can include meeting with legislators, testifying at hearings, or participating in grassroots campaigns.

The Role of the Judicial Branch

  • The courts play a key role in policy by interpreting statutes and regulations.
  • Stakeholders who believe a law or regulation is unconstitutional or that an agency has overstepped its authority can file a lawsuit.
  • The resulting court decisions can have a major impact on policy.
  • For example, the Supreme Court has made landmark rulings on the Affordable Care Act.
  • A CPPA must stay aware of major judicial decisions that affect pharmacy policy, as they can change the entire landscape.

Block 3: Payer & Reimbursement Policy

Medicare Part A Policy (Inpatient)

  • Medicare Part A covers drugs provided during an inpatient hospital stay.
  • Payment for these drugs is typically bundled into the Diagnosis-Related Group (DRG) payment.
  • The hospital does not receive a separate payment for each drug administered.
  • This creates an incentive for hospitals to manage their inpatient drug costs carefully.
  • The hospital P&T committee and formulary are key tools for managing these costs.
  • A CPPA in a hospital setting must understand this bundled payment system.

Medicare Part B Policy (Provider-Administered Drugs)

  • Medicare Part B covers drugs that are administered by a physician in an outpatient setting.
  • Reimbursement for these drugs is based on the Average Sales Price (ASP).
  • The payment formula is typically ASP + 6%.
  • CMS has experimented with changing this formula through demonstration projects.
  • Local Coverage Determinations (LCDs) are policies created by regional Medicare contractors that define the specific clinical criteria for covering a Part B drug.
  • A CPPA must be an expert on these complex Part B drug policies.

Medicare Part D Policy (Outpatient Rx)

  • The Part D program is the main source of prescription drug coverage for seniors.
  • It is delivered through private plans that are regulated by CMS.
  • CMS sets the rules for the standard benefit design, formulary requirements (including the six protected classes), and utilization management (PAs).
  • CMS also regulates the MTM programs that plans must offer.
  • The Inflation Reduction Act (IRA) made major changes to Part D, including drug price negotiation and an out-of-pocket cap.
  • A CPPA must be up-to-date on all of these complex Part D policies.

Medicare Advantage (Part C) Policy

  • Part C plans are private plans that cover all Part A and B benefits, and usually Part D.
  • CMS pays these plans a fixed monthly capitation payment for each member.
  • The plans are then at risk for managing the total cost of care.
  • This gives them a strong incentive to use utilization management tools like prior authorization.
  • While they must cover all medically necessary services, they can have different rules and networks than Original Medicare.
  • A CPPA must understand how the policies of these private MA plans can differ from traditional FFS Medicare.

The Role of MedPAC

  • The Medicare Payment Advisory Commission (MedPAC) is an independent congressional agency.
  • Its role is to advise the U.S. Congress on issues affecting the Medicare program.
  • MedPAC conducts research and policy analysis and makes recommendations to Congress.
  • They issue two major reports each year that are highly influential in the policy debate.
  • Their recommendations often form the basis for future Medicare legislation.
  • A CPPA should follow MedPAC's work to understand the future direction of Medicare policy.

Federal-State Partnership

  • Medicaid is jointly financed by the federal government and the states.
  • The federal government establishes broad national guidelines.
  • Each state then designs and administers its own program within these guidelines.
  • This results in 50+ different Medicaid programs with significant variation.
  • A CPPA must focus on the policies of their specific state(s).
  • States need a "State Plan" approved by CMS to receive federal matching funds.

Medicaid Drug Rebate Program (MDRP)

  • The MDRP is a cornerstone of Medicaid pharmacy policy.
  • It requires manufacturers to provide a rebate to states in exchange for coverage of their drugs.
  • This ensures that state Medicaid programs get the "best price" available in the commercial market.
  • Because of this rebate, states must cover nearly all FDA-approved drugs, making their formularies relatively open.
  • The policy tools they use to manage their drug benefit, like Preferred Drug Lists (PDLs) and PAs, must operate within this "open formulary" context.
  • A CPPA must understand how the MDRP shapes state pharmacy policy.

Managed Care vs. Fee-for-Service

  • States are increasingly delivering Medicaid benefits through private Managed Care Organizations (MCOs).
  • This shifts the financial risk from the state to the MCO.
  • It also creates a different set of policies for providers to follow.
  • A CPPA must analyze the policies of both the state's FFS program and the various MCOs operating in the state.
  • The MCOs often have their own formularies and PA criteria that may differ from the state's FFS PDL.
  • This adds a significant layer of complexity to policy analysis and compliance.

340B and Medicaid

  • There is a complex interaction between the 340B program and Medicaid.
  • The "duplicate discount" prohibition prevents a manufacturer from having to pay both a 340B discount and a Medicaid rebate on the same drug.
  • State Medicaid policies are the primary mechanism for preventing this.
  • A CPPA must be an expert on their state's policies for identifying 340B claims.
  • This is a high-risk area of compliance for 340B providers.
  • The policies in this area are constantly evolving.

State-Specific Initiatives

  • States often experiment with new pharmacy policy initiatives.
  • This can include things like subscription models for high-cost drugs (e.g., for Hepatitis C).
  • It can also include value-based purchasing arrangements.
  • Some states are seeking federal waivers to have more flexibility in managing their drug benefits.
  • A CPPA must stay current on these state-level innovations and waiver requests.
  • They are often at the cutting edge of pharmacy policy development.

The Role of PBMs

  • In the commercial market, pharmacy policy is largely driven by Pharmacy Benefit Managers (PBMs).
  • PBMs are hired by employers and health plans to manage the prescription drug benefit.
  • They create the formularies, design the utilization management programs (PAs, step therapy), and process claims.
  • A CPPA working for a health system or pharmacy must be an expert at analyzing PBM policies.
  • A CPPA working for a PBM would be involved in creating these policies.

Formulary and Utilization Management

  • PBM formularies are often much more restrictive than in Medicare or Medicaid.
  • They may exclude high-cost drugs entirely if a cheaper alternative is available.
  • They make extensive use of tiered copayments to steer patients to preferred drugs.
  • Their PA and step therapy criteria are a key focus of policy analysis.
  • A CPPA would analyze whether these criteria are evidence-based and how they impact patient access.

Rebate-Driven Policies

  • A key driver of PBM policy is the negotiation of rebates with drug manufacturers.
  • A drug's formulary status is often determined by the size of the rebate its manufacturer is willing to pay.
  • This can lead to situations where a more expensive drug with a large rebate is preferred over a cheaper drug with a small rebate.
  • This rebate system is a central and controversial aspect of U.S. drug pricing policy.
  • A CPPA must understand how this dynamic influences all other PBM policies.

Value-Based Contracting

  • This is an emerging area of commercial payer policy.
  • In a value-based contract, the price of a drug is tied to its real-world effectiveness.
  • For example, a manufacturer might agree to provide a larger rebate if a new diabetes drug does not achieve a certain level of A1c reduction in the plan's population.
  • These contracts are complex to design and administer.
  • A CPPA might be involved in analyzing or designing these innovative payment models.

State Regulation of PBMs

  • In recent years, states have become very active in regulating PBMs.
  • Many states have passed laws to increase the transparency of PBM practices.
  • These laws may regulate how PBMs create their MAC lists for generic drugs.
  • They may also prohibit certain practices, like "spread pricing" in the Medicaid MCO market.
  • A CPPA must track this rapidly evolving area of state law.
  • It represents a major shift in the balance of power between states and PBMs.

The Pharmaceutical Supply Chain and Pricing

  • Drug prices are determined by a complex and often opaque set of interactions between manufacturers, wholesalers, PBMs, and pharmacies.
  • A key area of policy debate is the role of rebates and the growing gap between a drug's list price and the net price after rebates.
  • A CPPA analyzes policies that aim to increase the transparency of this system.
  • This includes policies that would require the disclosure of net prices or limit the role of rebates.
  • Understanding this complex financial flow is essential for any analysis of drug pricing policy.

Key Pricing Benchmarks (AWP, WAC, ASP)

  • Pharmacy reimbursement policy is based on a set of standard pricing benchmarks.
  • A CPPA must be an expert on these benchmarks and their flaws.
  • AWP (Average Wholesale Price): A historical "sticker price" that is often inflated.
  • WAC (Wholesale Acquisition Cost): The manufacturer's list price to wholesalers.
  • ASP (Average Sales Price): The average price manufacturers receive after commercial rebates. Used for Medicare Part B.
  • Policy proposals often involve shifting reimbursement away from AWP towards more cost-based benchmarks like ASP.

Generic Drug Pricing Policy

  • Generic drugs are a key source of cost savings for the healthcare system.
  • Policy in this area is focused on promoting competition and preventing price gouging.
  • The Hatch-Waxman Act created the modern generic drug approval pathway.
  • Recent policy debates have focused on issues that can delay generic competition, such as "pay-for-delay" agreements.
  • Sudden, dramatic price increases for old generic drugs have also been a major policy issue.
  • A CPPA may analyze policies aimed at addressing these market failures.

Biosimilar Policy

  • Biosimilars are the "generic" equivalent of complex biologic drugs.
  • The Biologics Price Competition and Innovation Act (BPCIA) created an abbreviated approval pathway for biosimilars.
  • Policy in this area is focused on encouraging the development and uptake of these lower-cost alternatives.
  • This includes policies around naming, interchangeability, and reimbursement.
  • The uptake of biosimilars in the U.S. has been slower than in other countries.
  • A CPPA would analyze the policy barriers to a more robust biosimilar market.

The Inflation Reduction Act (IRA) and Price Negotiation

  • The IRA of 2022 represents the most significant change in drug pricing policy in decades.
  • For the first time, it gives Medicare the authority to directly negotiate the price of certain high-cost drugs.
  • It also requires manufacturers to pay a rebate to Medicare if their prices rise faster than inflation.
  • It also redesigned the Part D benefit to cap out-of-pocket costs for seniors.
  • A CPPA must have a deep understanding of all the provisions of this landmark law.
  • Its implementation and effects will be a major focus of policy analysis for years to come.

The FDA's Role and Structure

  • The Food and Drug Administration (FDA) is responsible for ensuring the safety and efficacy of all drugs marketed in the U.S.
  • The Center for Drug Evaluation and Research (CDER) is the specific branch of the FDA that oversees prescription and over-the-counter drugs.
  • The FDA's authority comes from the Food, Drug, and Cosmetic Act and its many amendments.
  • A CPPA must understand the FDA's central role as the gatekeeper for the pharmaceutical market.
  • FDA policy determines which drugs are available to patients.

The New Drug Approval (NDA) Process

  • Before a new drug can be marketed, it must go through a rigorous approval process.
  • This involves pre-clinical (animal) testing, followed by three phases of human clinical trials.
  • The manufacturer then submits a New Drug Application (NDA) to the FDA with all of the data.
  • The FDA's team of scientists reviews the data to determine if the drug's benefits outweigh its risks.
  • This process can take many years and cost billions of dollars.
  • Policy debates in this area often focus on the balance between speed and certainty.

Prescription Drug User Fee Act (PDUFA)

  • PDUFA is a law, first passed in 1992 and reauthorized every five years, that allows the FDA to collect fees from drug manufacturers to fund the new drug approval process.
  • In exchange for these fees, the FDA agrees to meet specific timelines for its review of NDAs.
  • PDUFA has been credited with significantly speeding up the drug approval process in the U.S.
  • The reauthorization of PDUFA is a major legislative event that often includes other FDA policy changes.
  • A CPPA should be aware of PDUFA and its role in FDA funding and performance.

Expedited Approval Pathways

  • To speed access to drugs for serious conditions, the FDA has several expedited approval pathways.
  • Fast Track: For drugs that treat a serious condition and fill an unmet medical need.
  • Breakthrough Therapy: For drugs that show a substantial improvement over existing therapies early in development.
  • Accelerated Approval: Allows for approval based on a surrogate endpoint (e.g., tumor shrinkage) rather than a clinical outcome (e.g., survival). The manufacturer must conduct post-market studies to confirm the benefit.
  • Priority Review: The FDA sets a goal to make a decision within 6 months, instead of the standard 10 months.
  • These pathways are a key area of policy debate, balancing the need for faster access with the need for complete data.

Post-Market Surveillance and Safety (FAERS)

  • The FDA's job does not end when a drug is approved.
  • They are also responsible for post-market surveillance to monitor the drug's safety in the real world.
  • The FDA Adverse Event Reporting System (FAERS) is a database that contains reports of adverse events submitted by patients, providers, and manufacturers.
  • The FDA uses this data to identify potential new safety problems.
  • If a new risk is identified, the FDA can require a label change, a formal Risk Evaluation and Mitigation Strategy (REMS), or, in rare cases, market withdrawal.
  • A CPPA should understand this post-market safety system.

Block 4: Federal Pharmacy Policy

The New Drug Approval (NDA) Process

  • The FDA's authority is rooted in the Food, Drug, and Cosmetic Act of 1938.
  • The NDA process requires manufacturers to prove both safety and efficacy through extensive clinical trials.
  • Phase 1 trials focus on safety in small groups of healthy volunteers.
  • Phase 2 trials assess efficacy and side effects in a small group of patients.
  • Phase 3 trials are large-scale, randomized controlled trials to confirm efficacy and monitor adverse reactions.
  • A CPPA analyzes policies that affect the standards for evidence and the rigor of this process.
  • The balance between speed of access and certainty of benefit is a constant policy tension.

Prescription Drug User Fee Act (PDUFA)

  • PDUFA is a law, reauthorized every five years, that allows the FDA to collect user fees from manufacturers.
  • These fees provide a significant portion of the FDA's budget for drug review.
  • In exchange, the FDA agrees to performance goals, including timelines for reviewing drug applications.
  • PDUFA has been credited with significantly reducing drug review times.
  • Each reauthorization is a major legislative event where other FDA policies are often debated and updated.
  • A CPPA must track PDUFA reauthorizations as they are a key driver of FDA policy.

Expedited Approval Pathways

  • The FDA has several pathways to speed the approval of drugs for serious or life-threatening conditions.
  • Fast Track: Facilitates development and expedites review of drugs that fill an unmet medical need.
  • Breakthrough Therapy: Designed to expedite drugs that show substantial improvement over existing therapies.
  • Accelerated Approval: Allows for approval based on a surrogate endpoint, with a requirement for post-market confirmatory trials.
  • Priority Review: The FDA sets a goal of reviewing the application within 6 months instead of the standard 10.
  • The use and standards for these pathways are a major area of policy analysis and debate.

Generic Drug & Biosimilar Policy

  • The Hatch-Waxman Act of 1984 created the abbreviated new drug application (ANDA) process for generic drugs.
  • The Biologics Price Competition and Innovation Act (BPCIA) of 2010 created a similar pathway for biosimilars.
  • Policy in this area focuses on promoting competition and timely market entry for lower-cost alternatives.
  • Issues analyzed include patent challenges, "pay-for-delay" agreements, and the standards for interchangeability of biosimilars.
  • A CPPA would analyze the impact of these policies on drug prices and patient access.

Post-Market Surveillance and REMS

  • FDA oversight continues after a drug is approved.
  • The FDA Adverse Event Reporting System (FAERS) collects post-market safety data.
  • For drugs with serious safety concerns, the FDA can require a Risk Evaluation and Mitigation Strategy (REMS).
  • A REMS can include elements like special training for prescribers or required patient monitoring.
  • The policy debate around REMS often centers on balancing safety with patient and provider burden.
  • A CPPA analyzes the design and impact of these post-market safety policies.

The Controlled Substances Act (CSA)

  • The CSA is the federal law that regulates the manufacture, distribution, and dispensing of controlled substances.
  • It establishes a "closed system" of distribution intended to prevent diversion.
  • The law created the five schedules (I-V) for classifying drugs based on their abuse potential and medical use.
  • The DEA is the agency responsible for enforcing the CSA.
  • A CPPA must understand the legal framework of the CSA as it is the foundation for all controlled substance policy.

Prescription Drug Monitoring Programs (PDMPs)

  • PDMPs are state-level electronic databases that track the dispensing of controlled substances.
  • They are a key policy tool for combating the opioid crisis.
  • Prescribers and pharmacists are required or encouraged to check the PDMP before prescribing or dispensing.
  • This helps to identify patients who may be "doctor shopping" or receiving dangerous combinations of drugs.
  • Policy issues in this area include interstate data sharing and the integration of PDMPs into EHR workflows.
  • A CPPA would analyze the effectiveness of PDMPs and policies related to their use.

Opioid Prescribing Policies

  • In response to the opioid crisis, many federal and state policies have been implemented to change prescribing habits.
  • This includes laws that limit the duration or dose of an initial opioid prescription for acute pain.
  • Many states have adopted guidelines from the CDC on opioid prescribing.
  • Payers have also implemented their own policies, such as requiring PAs for high-dose or long-acting opioids.
  • A CPPA analyzes the intended and unintended consequences of these policies on pain management and patient access.

Policies to Expand Access to Treatment

  • Alongside prescribing restrictions, there is a major policy focus on expanding access to treatment for substance use disorder.
  • This includes policies to increase the availability of naloxone to reverse overdoses.
  • The Drug Addiction Treatment Act of 2000 (DATA 2000) allowed physicians to prescribe buprenorphine for opioid use disorder in an office setting.
  • Subsequent laws have expanded prescribing authority to other providers and removed certain training requirements.
  • Policies also focus on increasing insurance coverage for addiction treatment services.
  • A CPPA analyzes these policies aimed at the public health side of the crisis.

Telemedicine for Controlled Substances

  • The Ryan Haight Act generally requires an in-person medical evaluation before a controlled substance can be prescribed via telemedicine.
  • During the COVID-19 Public Health Emergency, these restrictions were waived.
  • This led to a dramatic increase in the use of telemedicine for addiction treatment and other conditions.
  • The future of these telemedicine flexibilities is a major, ongoing policy debate.
  • The debate centers on balancing the clear benefits of increased access with the potential risk of diversion.
  • A CPPA would be involved in analyzing the data and policy options related to this issue.

Program Intent and Eligibility

  • The 340B program requires manufacturers to provide deep discounts to safety-net providers.
  • The stated intent is to allow these Covered Entities (CEs) to "stretch scarce federal resources."
  • A key policy debate is whether the program is functioning as intended.
  • Policy analysts evaluate the criteria for hospital and grantee eligibility.
  • Proposals to change these eligibility thresholds are a frequent topic of debate.
  • Another key area is the policy around the registration of off-site "child" clinics.

The Patient Definition

  • The 340B statute does not define the term "patient."
  • HRSA has created a three-part definition through guidance, which has been the subject of legal challenges.
  • This ambiguity is a central policy issue in the 340B program.
  • Many legislative proposals have sought to create a clear, statutory definition of a patient.
  • A CPPA would analyze the impact of different proposed definitions on CEs and patient access.
  • This is a core issue related to the prohibition on diversion.

Contract Pharmacy Policy

  • The use of contract pharmacies to dispense 340B drugs has allowed for massive program growth.
  • However, this practice is based on HRSA guidance, not specific statutory language.
  • In recent years, many manufacturers have restricted the use of 340B pricing for drugs dispensed at contract pharmacies.
  • This has led to intense litigation and policy debate.
  • A CPPA would analyze the legal arguments on both sides and the impact of these restrictions on patient access.
  • This is one of the most contentious and dynamic areas of 340B policy.

Duplicate Discount Policy

  • The statute prohibits a duplicate discount (a 340B discount and a Medicaid rebate on the same drug).
  • The mechanism for preventing this is the Medicaid Exclusion File (MEF).
  • However, the rise of Medicaid Managed Care has complicated this issue.
  • Policy analysis in this area focuses on the effectiveness of the MEF and the challenges of preventing duplicate discounts in the MCO context.
  • A CPPA must understand the complex interaction between 340B and the Medicaid Drug Rebate Program.

Use of Savings and Transparency

  • A major policy debate centers on how CEs use the savings generated by the 340B program.
  • Critics argue for greater transparency and requirements for how savings are used.
  • CEs argue that the savings are used to support their safety-net mission and that such requirements would be overly burdensome.
  • Many legislative proposals have included new transparency and reporting requirements for 340B hospitals.
  • A CPPA would analyze the potential impact of these proposals on CEs.
  • This is a core political and policy issue for the future of the program.

The Drug Quality and Security Act (DQSA)

  • The DQSA was passed in 2013 in response to a deadly fungal meningitis outbreak linked to a compounding pharmacy.
  • It created a new category of compounder known as a 503B Outsourcing Facility.
  • These facilities can compound large batches of sterile products without a prescription.
  • In exchange, they must register with the FDA and comply with current Good Manufacturing Practices (cGMPs).
  • The law also clarified the standards for traditional 503A compounding pharmacies.
  • A CPPA analyzes the implementation of this key law.

503A vs. 503B Policy

  • The distinction between 503A and 503B is a key area of FDA policy.
  • 503A pharmacies are for patient-specific compounding and are primarily regulated by state boards.
  • 503B facilities are for non-patient-specific ("office use") compounding and are regulated by the FDA.
  • Policy issues include the interpretation of what constitutes a "patient-specific" prescription.
  • Another key issue is the FDA's development of a list of drugs that are "difficult to compound."
  • A CPPA must understand the different legal and regulatory frameworks for these two types of compounding.

Biologics and the BPCIA

  • Biologics are complex drugs derived from living organisms (e.g., monoclonal antibodies).
  • They are regulated by the FDA's Center for Biologics Evaluation and Research (CBER).
  • The Biologics Price Competition and Innovation Act (BPCIA) of 2010 created an abbreviated approval pathway for biosimilars.
  • A biosimilar is a product that is highly similar to an already-approved biologic.
  • This policy is intended to create competition and lower prices for these high-cost drugs.

Biosimilar Interchangeability

  • A key policy issue for biosimilars is "interchangeability."
  • An interchangeable biosimilar is one that can be substituted for the reference product by a pharmacist without the intervention of the prescriber.
  • To be deemed interchangeable, a manufacturer must conduct additional studies to show that there is no difference when a patient is switched back and forth.
  • State pharmacy practice laws also govern the rules for this substitution.
  • A CPPA analyzes the federal and state policies that are shaping the market for interchangeable biosimilars.

Advanced and Regenerative Therapies

  • This is a new and rapidly evolving area of policy.
  • It includes cell therapies, gene therapies, and tissue engineering products.
  • The 21st Century Cures Act created a new Regenerative Medicine Advanced Therapy (RMAT) designation to speed the development of these products.
  • These therapies pose unique challenges for the FDA's traditional approval framework.
  • They also pose major challenges for pricing and reimbursement due to their extremely high costs and potential for one-time cures.
  • A CPPA would be involved in analyzing the emerging clinical, regulatory, and payment policies for these innovative therapies.

The Role of the FTC

  • The Federal Trade Commission (FTC) is a federal agency whose mission is to protect consumers and promote competition.
  • In the pharmaceutical space, the FTC works to prevent anticompetitive practices.
  • This includes reviewing mergers between drug companies and challenging practices that may harm competition.
  • The FTC's work is grounded in federal antitrust laws like the Sherman Act and the Clayton Act.
  • A CPPA should understand the FTC's role in the pharmaceutical market.

Mergers and Acquisitions

  • The pharmaceutical industry has seen significant consolidation through mergers and acquisitions.
  • The FTC reviews these mergers to ensure they do not substantially lessen competition.
  • If a merger would give a single company a monopoly on a specific type of drug, the FTC may require the companies to sell off that part of their business as a condition of approval.
  • The FTC also reviews vertical mergers, such as a health insurer acquiring a PBM.
  • A CPPA would analyze the potential impact of these mergers on drug prices and market competition.

"Pay-for-Delay" Agreements

  • This is a practice where a brand-name drug company pays a generic company to delay the entry of its generic version into the market.
  • These agreements are often made to settle patent litigation.
  • The FTC has argued that these "reverse payment" settlements are anticompetitive and harm consumers by keeping generic drugs off the market.
  • The Supreme Court has ruled that these agreements can be challenged under antitrust law.
  • This is a major area of focus for FTC policy and enforcement.
  • A CPPA would analyze the impact of these agreements on drug spending.

Product Hopping

  • Product hopping is a strategy used by some brand-name manufacturers just before their patent expires.
  • They make a minor change to their product (e.g., switching from a capsule to a tablet) and market it as a new, improved version.
  • They then pull the original product from the market.
  • This can force patients to switch to the new, still-patented version, even if the old version is about to become a much cheaper generic.
  • The FTC has challenged this practice as an illegal attempt to maintain a monopoly.
  • A CPPA would analyze the clinical and economic impact of this strategy.

PBMs and Vertical Integration

  • In recent years, there has been significant vertical integration in the healthcare industry.
  • Large health insurers have merged with the largest PBMs.
  • These integrated companies now have enormous market power.
  • The FTC has been closely scrutinizing the business practices of PBMs.
  • This includes their role in negotiating rebates and their impact on independent pharmacies.
  • The role of PBMs and the lack of transparency in their operations is a major, ongoing policy debate.
  • A CPPA would be heavily involved in analyzing the policy proposals aimed at regulating PBMs.

Block 5: Payer Reimbursement & Access Policy

The Inflation Reduction Act (IRA)

  • The IRA of 2022 is the most significant pharmacy policy reform since the creation of Part D.
  • Its three key provisions are Medicare drug price negotiation, inflation rebates, and a redesign of the Part D benefit.
  • A CPPA must have an expert-level understanding of this law.
  • The implementation of the IRA will be the dominant theme in Medicare policy for the next several years.
  • It represents a fundamental shift in how Medicare pays for drugs.

Medicare Drug Price Negotiation

  • For the first time, the IRA gives Medicare the authority to directly negotiate prices for a select number of high-spend drugs.
  • The first 10 drugs were selected in 2023, with the negotiated prices taking effect in 2026.
  • The law outlines a complex process for selecting drugs and conducting the negotiation.
  • This policy is being challenged in court by pharmaceutical manufacturers.
  • A CPPA would analyze the impact of this policy on drug prices, manufacturer revenues, and future innovation.

Inflation Rebates

  • The IRA requires manufacturers to pay a rebate to Medicare if they raise the price of their drugs faster than the rate of inflation.
  • This policy applies to drugs covered under both Part B and Part D.
  • It is designed to curb the rapid growth in list prices for existing drugs.
  • Beneficiaries may also see lower coinsurance for drugs whose prices have risen faster than inflation.
  • A CPPA would analyze the effectiveness of this policy in controlling drug price growth.

Part D Benefit Redesign

  • The IRA completely redesigns the Part D benefit, effective in 2025.
  • It eliminates the coverage gap ("donut hole").
  • It establishes a new, much lower out-of-pocket cap for beneficiaries (initially $2,000).
  • It also shifts a greater portion of the liability in the catastrophic phase from Medicare to the health plans.
  • This is intended to give plans a stronger incentive to negotiate lower prices.
  • A CPPA analyzes how this redesign will impact patient affordability and plan premiums.

Part B Reimbursement Policy (ASP+6%)

  • Reimbursement for provider-administered drugs under Part B is a perennial policy issue.
  • The current methodology is Average Sales Price (ASP) plus a 6% add-on payment.
  • Critics argue this model incentivizes the use of more expensive drugs.
  • CMS has experimented with alternative payment models through its Innovation Center.
  • MedPAC has repeatedly recommended changes to the add-on payment.
  • A CPPA would analyze the various proposals to reform Part B drug reimbursement.

Medicaid Waivers (Section 1115)

  • Section 1115 of the Social Security Act gives the Secretary of HHS the authority to approve experimental, pilot, or demonstration projects in the Medicaid program.
  • These "1115 waivers" allow states to test new approaches to delivering and paying for care that differ from the standard federal rules.
  • In pharmacy, states have used waivers to implement more restrictive formularies or to negotiate supplemental rebates directly with manufacturers.
  • A CPPA must track the 1115 waiver proposals and approvals in their state and nationally.
  • They represent the cutting edge of Medicaid policy innovation.

Managed Care and Carve-Outs

  • The shift to Medicaid Managed Care is a dominant trend.
  • A key policy decision for states is whether to "carve out" the pharmacy benefit from the MCO contract.
  • If carved out, the state's FFS program manages the pharmacy benefit for all Medicaid patients, even those in MCOs.
  • This gives the state more control and allows them to collect all of the rebates.
  • If "carved in," each MCO manages its own pharmacy benefit.
  • There is a major policy debate about which model is more cost-effective and provides better care.
  • A CPPA would analyze the pros and cons of these different models.

Spread Pricing in Medicaid MCOs

  • Spread pricing is a controversial practice used by PBMs that contract with Medicaid MCOs.
  • The PBM pays the pharmacy one price for a drug but bills the MCO a higher price.
  • The difference, or "spread," is kept by the PBM as profit.
  • Many states have passed laws to prohibit spread pricing in their Medicaid programs.
  • These policies are designed to increase transparency and ensure that taxpayer dollars are spent on patient care, not PBM profits.
  • A CPPA would analyze the impact of these policies on state spending and pharmacy reimbursement.

Policies on High-Cost Drugs

  • The advent of very high-cost drugs, like those for Hepatitis C or spinal muscular atrophy, has created major budget challenges for state Medicaid programs.
  • States have used various policy tools to manage these costs.
  • This includes very strict PA criteria and negotiating supplemental rebates.
  • Some states have experimented with innovative payment models, like a "subscription" model where the state pays a flat fee to a manufacturer for unlimited access to a drug.
  • A CPPA analyzes the effectiveness and legality of these different state strategies.

340B and Medicaid Policy

  • As mentioned, the interaction between 340B and Medicaid is a key policy area.
  • States are increasingly focused on identifying 340B claims to avoid seeking duplicate rebates.
  • Some states have tried to lower their reimbursement to 340B pharmacies, effectively sharing in the 340B discount.
  • The legality of this practice has been challenged.
  • A CPPA must be an expert on their state's specific policies for reimbursing 340B providers.
  • This is a complex and often contentious area of policy.

Formulary Exclusion Lists

  • A major trend in commercial policy is the use of formulary exclusion lists.
  • PBMs now exclude hundreds of brand-name drugs from their formularies entirely.
  • This is done when there are other, lower-cost drugs in the same therapeutic class.
  • This is a powerful tool to force manufacturers to offer larger rebates to avoid exclusion.
  • A CPPA analyzes the clinical and economic impact of these exclusion lists.
  • They assess whether the exclusions are leading to access issues for patients.

Rebate Walls

  • A "rebate wall" is a term for a situation where a high-rebate brand drug creates a barrier to entry for a new, potentially more innovative or lower-cost competitor.
  • The PBM may be unwilling to add the new drug to its formulary because it would jeopardize the large rebates it receives for the existing drug.
  • This practice has been criticized for stifling competition and keeping prices high.
  • A CPPA would analyze the competitive effects of this rebate-driven formulary management.
  • This is an area of interest for antitrust regulators like the FTC.

Copay Accumulator Programs

  • These are policies implemented by PBMs and health plans that have a major impact on patients using manufacturer copay cards.
  • Normally, a patient's copay counts towards their annual deductible and out-of-pocket maximum.
  • With an accumulator program, the value of the manufacturer's copay card does *not* count.
  • This means the patient is still responsible for their full deductible, which can be thousands of dollars.
  • This policy has been very controversial.
  • A CPPA analyzes the impact of these programs on patient adherence and affordability.
  • Several states have passed laws to ban this practice.

Specialty Pharmacy Networks

  • PBMs often have exclusive or limited networks for specialty drugs.
  • They may require that a patient receive their specialty medication from the PBM's own specialty pharmacy.
  • This practice is known as "patient steering."
  • It has been criticized for limiting patient choice and disrupting continuity of care, especially for patients receiving care at a hospital-based clinic.
  • A CPPA would analyze the policy and legal issues surrounding these restrictive networks.
  • This is another area where states have begun to legislate.

State Regulation of PBMs

  • In response to many of the issues above, states have dramatically increased their regulation of PBMs.
  • Laws have been passed to require PBMs to be licensed, to increase the transparency of their practices, and to ban practices like spread pricing and retroactive DIR fees.
  • A recent Supreme Court decision (Rutledge v. PCMA) affirmed the rights of states to regulate PBMs.
  • This has opened the door for even more state-level action.
  • A CPPA must be an expert on the rapidly evolving landscape of state PBM regulation.

Health Technology Assessment (HTA)

  • HTA is the systematic evaluation of the properties and effects of a health technology.
  • It is a multidisciplinary field that combines clinical, economic, and ethical analysis.
  • The goal is to inform policy and decision-making about which technologies provide the best value.
  • In many other developed countries, a formal HTA process is a required step before a new drug is covered by the national health system.
  • While the U.S. does not have a formal government HTA body, the principles are widely used by payers.
  • A CPPA should understand the role of HTA in shaping coverage and reimbursement policy.

The Institute for Clinical and Economic Review (ICER)

  • ICER is a prominent, independent, non-profit organization in the U.S. that conducts value assessments of new drugs.
  • They are the closest thing the U.S. has to a national HTA body.
  • ICER produces detailed reports that analyze a drug's clinical effectiveness and its cost-effectiveness.
  • They calculate a value-based price benchmark for new drugs.
  • Their reports are highly influential and are used by PBMs and payers to inform their formulary and coverage decisions.
  • A CPPA must be familiar with ICER, its methodology, and its reports.

Cost-Effectiveness Analysis (CEA)

  • CEA is a core method used in HTA and by organizations like ICER.
  • It compares the costs and the health outcomes of two or more different interventions.
  • The outcome is often measured in Quality-Adjusted Life Years (QALYs).
  • A QALY is a measure that combines both the quantity and the quality of life.
  • The result of a CEA is an ICER (Incremental Cost-Effectiveness Ratio), which is the cost per QALY gained.
  • There is a policy debate about the appropriate willingness-to-pay threshold for a QALY.
  • A CPPA should understand the basic principles of how these analyses are conducted.

Budget Impact Analysis (BIA)

  • A BIA is another key tool used in value assessment.
  • Unlike a CEA, which looks at cost-effectiveness from a societal perspective, a BIA looks at affordability from a specific budget holder's perspective (e.g., a health plan).
  • It forecasts the net financial impact of adopting a new drug on the health plan's budget over the next 1-5 years.
  • It considers the drug's price, the size of the eligible population, and any potential cost offsets (e.g., reduced hospitalizations).
  • A drug can be cost-effective but still have a very high budget impact that makes it unaffordable.
  • Payers rely heavily on BIAs.

The Role of Value Frameworks in Policy

  • Value assessment frameworks, like the one used by ICER, are increasingly shaping U.S. pharmacy policy.
  • PBMs are using these analyses to justify formulary exclusions and to negotiate rebates.
  • The Medicare drug price negotiation process will also involve an assessment of therapeutic alternatives and value.
  • There is a major policy debate about the role these frameworks should play and the methodologies they use.
  • A CPPA must be fluent in the language of value assessment, as it is becoming central to all pricing and reimbursement policy discussions.

The Uninsured and Underinsured

  • Despite the ACA, millions of Americans remain uninsured.
  • Many more are "underinsured," meaning their insurance has such high cost-sharing that it is inadequate.
  • This lack of adequate coverage is a major barrier to accessing needed medications.
  • Policies aimed at addressing this issue include expanding Medicaid, increasing subsidies on the ACA marketplaces, and supporting safety-net providers.
  • A CPPA analyzes the impact of these broad coverage policies on medication access.

High-Deductible Health Plans (HDHPs)

  • HDHPs have become the dominant form of employer-sponsored insurance.
  • These plans have very high deductibles that patients must pay before their coverage kicks in.
  • This means that even insured patients can face the full list price of their medications at the beginning of the year.
  • This can lead to "pharmacy sticker shock" and medication non-adherence.
  • Policy debates in this area focus on how to protect patients with chronic conditions from this high cost exposure.
  • This includes policies to allow for pre-deductible coverage of certain maintenance medications.

Manufacturer and Foundation Assistance

  • Patient Assistance Programs (PAPs) and copay cards are a key part of the affordability landscape.
  • These programs provide a crucial safety net for many patients.
  • However, they are also a subject of policy debate.
  • Critics argue that copay cards can steer patients to more expensive brand drugs, increasing overall costs for the health system.
  • This is why accumulator programs were created by PBMs.
  • A CPPA analyzes the role and impact of these assistance programs on the healthcare system.

Out-of-Pocket Caps and Smoothing

  • A major policy trend is to cap the amount that patients have to pay out-of-pocket for their drugs.
  • The IRA's $2,000 cap for Medicare Part D is a landmark example.
  • Some states and commercial plans have also implemented caps, especially for insulin.
  • Another emerging policy is "cost-smoothing."
  • This would allow patients to pay their annual out-of-pocket costs in fixed monthly installments, avoiding a large bill in January.
  • A CPPA would be involved in analyzing the design and impact of these affordability policies.

The Role of Social Determinants of Health (SDoH)

  • There is a growing recognition that clinical care is only one factor that determines a person's health.
  • Social Determinants of Health (SDoH) are the conditions in the places where people live, learn, work, and play that affect a wide range of health risks and outcomes.
  • These include things like income, housing stability, and access to transportation.
  • SDoH are a major driver of health inequities and can be a huge barrier to medication access.
  • A patient cannot be adherent to their medication if they cannot afford it or do not have transportation to the pharmacy.
  • Forward-thinking health policy is increasingly focused on addressing SDoH.
  • A CPPA should understand this broader context of health policy.

Block 6: Policy Implementation & Evaluation

The Importance of a Communication Plan

  • A policy is only effective if people know about it and understand it.
  • A formal communication plan is a critical part of policy implementation.
  • The plan should identify all key audiences (e.g., pharmacists, physicians, patients, payers).
  • It should define the key message to be delivered to each audience.
  • It should also specify the channels to be used for communication (e.g., email, meetings, newsletters).
  • The CPPA is often responsible for developing and executing this plan.

Crafting Policy Briefs and Memos

  • Policy briefs are concise summaries of a policy issue, designed for busy decision-makers.
  • They typically start with an executive summary that gets straight to the point.
  • They present the problem, the policy options, and the recommendation in a clear and logical way.
  • Data should be presented visually using charts and graphs.
  • The language should be non-technical and accessible.
  • A CPPA must be an expert at writing these types of documents.

Presenting to Diverse Audiences

  • A policy analyst must be able to present their findings verbally to a wide range of audiences.
  • Presenting to a group of expert pharmacists requires a different approach than presenting to a community advisory board.
  • The level of technical detail and the key messages must be tailored to the audience.
  • A CPPA must be a skilled public speaker and facilitator.
  • They must be able to answer tough questions and defend their analysis.

Working with the Media

  • The media can be a powerful tool for disseminating information about a policy issue.
  • A CPPA may be involved in preparing press releases or fact sheets for the media.
  • They may also serve as a subject matter expert for interviews with journalists.
  • When working with the media, it is crucial to have a clear and consistent message.
  • The goal is to accurately inform the public debate.
  • All media interactions should be coordinated with the organization's communications department.

Dissemination in the Digital Age

  • Modern policy dissemination uses a variety of digital channels.
  • This includes publishing reports and data on the organization's website.
  • Social media can be used to share key findings and engage with stakeholders.
  • Webinars are an effective way to present a detailed analysis to a wide audience.
  • Infographics and short videos can make complex policy issues more accessible.
  • A CPPA should be familiar with these digital tools and how to use them effectively.

Identifying Stakeholders

  • A stakeholder is any person, group, or organization that has an interest in or is affected by a policy.
  • The first step in engagement is to conduct a thorough stakeholder analysis to identify all relevant parties.
  • This includes supporters, opponents, and those who are neutral.
  • For a pharmacy policy, stakeholders could include patient advocacy groups, professional associations, pharmaceutical companies, and payers.
  • A CPPA must be skilled at mapping out the stakeholder landscape for any given issue.

Stakeholder Analysis Matrix

  • This is a tool used to categorize stakeholders to prioritize engagement efforts.
  • Stakeholders are typically plotted on a matrix with two axes:
  • Interest: How much does the policy affect them? (Low to High)
  • Power/Influence: How much influence do they have over the policy outcome? (Low to High)
  • Stakeholders in the "High Interest / High Power" quadrant are the key players who must be actively managed.
  • This analysis helps to focus limited time and resources where they will have the greatest impact.

Building Coalitions

  • A coalition is a temporary alliance of different groups who come together to work towards a common policy goal.
  • Coalitions are a powerful tool in advocacy, as they demonstrate a broad base of support for an issue.
  • Effective coalitions often include "strange bedfellows" - groups that may not normally work together but share a common interest on a specific issue.
  • A CPPA may be involved in identifying potential coalition partners and building these relationships.
  • This requires strong negotiation and interpersonal skills.

Engaging with Policymakers

  • Policymakers and their staff are the primary audience for policy analysis and advocacy.
  • Building long-term, trusted relationships with these offices is key.
  • This involves providing them with timely, accurate, and objective information.
  • The goal is to become a go-to resource for them on pharmacy policy issues.
  • A CPPA must understand the legislative process and how to effectively communicate in that environment.
  • This includes providing concise testimony at hearings and writing effective leave-behind documents.

Grassroots vs. Grasstops Advocacy

  • Grassroots Advocacy: Mobilizing a large number of individuals from the general public to contact their elected officials about an issue.
  • This shows policymakers that an issue is important to their constituents.
  • Grasstops Advocacy: Engaging a small number of influential community leaders to advocate on an issue.
  • These are individuals who have a personal relationship with the policymaker.
  • An effective advocacy campaign often uses both strategies.
  • A CPPA may be involved in providing the policy information that fuels these campaigns.

The Purpose of Program Evaluation

  • Program evaluation is the systematic collection of information about a program to assess its effectiveness and make decisions.
  • It is the final stage of the policy cycle.
  • It answers the question: "Did the policy work?"
  • The findings from an evaluation are used to improve, continue, or terminate a program.
  • A CPPA must be able to design and interpret program evaluations.

Types of Evaluation

  • Process Evaluation: Assesses how a program is being implemented. Is it reaching the target population? Are the activities being delivered as planned?
  • Outcome/Impact Evaluation: Assesses the extent to which a program has achieved its intended outcomes. Did the policy actually solve the problem it was designed to address?
  • Economic Evaluation: Assesses the cost-effectiveness of the program. Were the benefits worth the costs?
  • A comprehensive evaluation often includes all three types.

Evaluation Design: The Gold Standard

  • The "gold standard" for an impact evaluation is the Randomized Controlled Trial (RCT).
  • In an RCT, individuals are randomly assigned to either receive the policy intervention (the treatment group) or not (the control group).
  • By comparing the outcomes of the two groups, the analyst can determine the true causal effect of the policy.
  • However, RCTs are often expensive and may not be feasible or ethical in a real-world policy setting.

Quasi-Experimental Designs

  • When an RCT is not possible, quasi-experimental designs are used.
  • These designs attempt to mimic an experiment without random assignment.
  • Difference-in-Differences (DID): Compares the change in outcomes over time between a group that received the policy and a similar group that did not.
  • Regression Discontinuity Design (RDD): Used when a policy has a sharp cutoff (e.g., an age or income threshold). It compares outcomes for people just above and just below the cutoff.
  • Interrupted Time Series (ITS): Compares the trend in an outcome before and after a policy was implemented.
  • A CPPA should be familiar with these common evaluation designs.

Qualitative Evaluation Methods

  • Qualitative methods are important for understanding the "how" and "why" behind the numbers.
  • This includes conducting interviews with program staff and participants.
  • Focus groups can be used to gather in-depth feedback from a group of stakeholders.
  • Case studies can provide a detailed look at how the policy was implemented in a specific setting.
  • These methods provide rich, contextual information that complements the quantitative data.
  • A good evaluation uses a mixed-methods approach.

Stakeholder Analysis Matrix

  • An assessment tool used to categorize stakeholders to prioritize engagement efforts.
  • It plots stakeholders on a two-by-two grid based on their level of **Interest** in the policy and their level of **Power** to influence it.
  • High Power, High Interest (Manage Closely): These are the key players.
  • High Power, Low Interest (Keep Satisfied): Can be powerful allies or opponents if their interest is sparked.
  • Low Power, High Interest (Keep Informed): Good source of on-the-ground intelligence and potential coalition partners.
  • Low Power, Low Interest (Monitor): Require minimal effort.
  • This tool helps a CPPA to develop a strategic engagement plan.

Policy Implementation Fidelity Scale

  • A rating scale used in a process evaluation to measure how faithfully a policy or program is being implemented.
  • It breaks down the program into its core components.
  • Each component is then rated on a scale (e.g., 1-5) based on how well it is being delivered in practice compared to the original design.
  • This helps to distinguish between a policy that is ineffective because it was a bad idea (theory failure) versus one that was ineffective because it was poorly implemented (implementation failure).
  • This is a critical tool for understanding evaluation results.

The AGREE II Instrument

  • The AGREE (Appraisal of Guidelines for Research & Evaluation) II is an internationally recognized tool for assessing the quality of clinical practice guidelines.
  • It consists of 23 items organized into six domains (e.g., Scope and Purpose, Stakeholder Involvement, Rigor of Development).
  • A CPPA would use this tool to critically appraise a guideline before using it as evidence in a policy analysis.
  • It helps to determine whether a guideline is well-developed and trustworthy.
  • This is a key skill for an evidence-based analyst.

The Gunning Fog Index (or other readability scales)

  • A readability scale is a tool used to measure how easy a piece of writing is to read.
  • The Gunning Fog Index estimates the years of formal education a person needs to understand the text on the first reading.
  • A CPPA uses this tool to assess the readability of policy documents, patient education materials, and other communications.
  • The goal is to ensure that the writing is clear and accessible to the intended audience.
  • It is a practical tool for improving the effectiveness of policy communication.

Cost-Benefit Analysis (CBA) Framework

  • A framework for structuring an economic evaluation.
  • It involves identifying all the potential costs and benefits of a policy.
  • A monetary value must then be assigned to each cost and benefit.
  • This can be challenging, especially for intangible benefits like improved health or saved lives.
  • The total costs are then compared to the total benefits.
  • If the benefit-to-cost ratio is greater than 1, the policy is considered to have a net positive value.
  • This tool is used to assess the overall societal value of a policy.

Cost-Benefit Ratio

  • This formula is the final output of a cost-benefit analysis. It provides a single number that summarizes the relationship between the total benefits and total costs of a policy.

\( \text{Benefit-Cost Ratio} = \frac{\sum \text{Present Value of Benefits}}{\sum \text{Present Value of Costs}} \)

Incremental Cost-Effectiveness Ratio (ICER)

  • The primary output of a cost-effectiveness analysis. It represents the additional cost required to gain one additional unit of health outcome (like a Quality-Adjusted Life Year, or QALY). This is used to compare the value of a new drug to the current standard of care.

\( \text{ICER} = \frac{(\text{Cost}_{\text{New}} - \text{Cost}_{\text{Old}})}{(\text{Effectiveness}_{\text{New}} - \text{Effectiveness}_{\text{Old}})} = \frac{\Delta C}{\Delta E} \)

Budget Impact Model (BIM) Calculation

  • A BIM estimates the net change in spending for a health plan if a new drug is added to the formulary. It considers the size of the eligible population, the market share the new drug will capture, and any cost offsets from reduced utilization of other services.

\( \text{Budget Impact} = \sum (\text{Cost}_{\text{New Scenario}}) - \sum (\text{Cost}_{\text{Current Scenario}}) \)

Number Needed to Treat (NNT)

  • A common measure of clinical effectiveness used in policy analysis. It represents the average number of patients who need to be treated with a new therapy to prevent one additional bad outcome. A lower NNT indicates a more effective therapy.

\( \text{NNT} = \frac{1}{\text{Absolute Risk Reduction (ARR)}} \)

Relative Risk (RR)

  • A fundamental statistic from clinical trials used in policy analysis. It compares the probability of an outcome in the treatment group to the probability of the outcome in the control group. An RR < 1 indicates the treatment reduces the risk of the outcome.

\( \text{RR} = \frac{\text{Probability of Event in Treatment Group}}{\text{Probability of Event in Control Group}} \)

Block 7: Advanced Topics & Final Review

Defining CER

  • CER is the direct comparison of existing healthcare interventions to determine which work best for which patients and in which settings.
  • It compares drugs, devices, and procedures to each other to see which is most effective in real-world practice.
  • This is different from the placebo-controlled trials often used for FDA approval.
  • The goal is to provide actionable evidence to help patients, clinicians, and policymakers make better-informed decisions.
  • A CPPA must understand the role of CER in the evidence ecosystem.

The Patient-Centered Outcomes Research Institute (PCORI)

  • PCORI was created by the Affordable Care Act to fund CER.
  • It is an independent, non-profit organization.
  • Its mission is to fund research that is guided by patients, caregivers, and the broader healthcare community.
  • PCORI has funded hundreds of studies comparing different treatment options.
  • The results of these studies are a key source of evidence for policy analysis.
  • A CPPA should be familiar with PCORI and how to access its research findings.

Real-World Evidence (RWE)

  • CER often relies on Real-World Data (RWD), such as data from EHRs and insurance claims.
  • The evidence generated from the analysis of RWD is called Real-World Evidence (RWE).
  • RWE provides insights into how treatments work in a broader, more diverse patient population than can be studied in a traditional clinical trial.
  • The FDA is increasingly using RWE in its regulatory decision-making.
  • A CPPA must understand the strengths and weaknesses of RWE compared to evidence from RCTs.

CER and Payer Policy

  • Payers use the results of CER to inform their coverage policies.
  • If a CER study shows that two drugs have equal effectiveness, a payer will likely prefer the cheaper one on its formulary.
  • The results of CER can be used to develop evidence-based PA criteria.
  • By law, Medicare is prohibited from using CER to make coverage decisions that are solely based on cost-effectiveness. However, the evidence can still be used to inform coverage.
  • A CPPA would analyze how payers are using CER to shape their policies.

Patient-Centered Outcomes

  • A key focus of PCORI and CER is on patient-centered outcomes.
  • This means looking at outcomes that matter most to patients, not just clinical endpoints.
  • This can include things like quality of life, ability to function, and symptom burden.
  • This patient-centered approach is a key part of modern health policy.
  • It ensures that the definition of "effectiveness" is not just determined by clinicians, but also by patients themselves.

Defining Health Equity and Disparities

  • Health Disparities: Differences in health outcomes that are closely linked with social, economic, and/or environmental disadvantage.
  • Health Equity: The principle that everyone should have a fair and just opportunity to be as healthy as possible.
  • Achieving health equity requires removing obstacles to health such as poverty and discrimination.
  • Pharmacy policy can either worsen or help to reduce health disparities.
  • A CPPA must analyze all policies through a health equity lens.

Social Determinants of Health (SDoH)

  • SDoH are the conditions in the environments where people are born, live, learn, work, and play that affect health.
  • These include factors like socioeconomic status, education, neighborhood and physical environment, and social support networks.
  • SDoH are the root causes of many health disparities.
  • For example, a patient living in a "pharmacy desert" has a transportation barrier to medication access.
  • A CPPA must consider how policies will impact patients with adverse SDoH.

Equity in Formulary Design

  • Payer policies can have a disproportionate impact on vulnerable populations.
  • For example, a high copay for an essential medication can be a major barrier for a low-income patient.
  • A formulary that excludes a drug for a condition that is more prevalent in a minority population could worsen disparities.
  • A CPPA would analyze formulary policies to assess their impact on health equity.
  • This includes looking at the placement of drugs for conditions like sickle cell disease or HIV.

Equitable Access to Clinical Trials

  • Historically, clinical trials have often failed to enroll a diverse population.
  • This means that the evidence base for new drugs may not be generalizable to all racial and ethnic groups.
  • There is a major policy effort to increase the diversity of clinical trial participants.
  • This includes policies to reduce the logistical and financial barriers to participation.
  • A CPPA would analyze these policies aimed at improving equity in clinical research.

Policy Levers to Advance Health Equity

  • Policy is a powerful tool for advancing health equity.
  • This can include expanding insurance coverage through Medicaid.
  • It can involve creating value-based payment models that reward providers for reducing disparities.
  • It can also include policies that address the upstream SDoH, such as investments in housing or nutrition.
  • A CPPA should be familiar with these policy levers.
  • Analyzing the equity impact should be a standard part of every policy analysis.

Comparative Health Systems

  • Understanding how other countries structure their healthcare systems provides valuable context for U.S. policy debates.
  • There are four basic models:
  • Beveridge Model: Healthcare is provided and financed by the government (e.g., United Kingdom).
  • Bismarck Model: Uses a system of private insurance plans, but they are tightly regulated and non-profit (e.g., Germany).
  • National Health Insurance Model: Has private-sector providers, but payment comes from a government-run insurance program (e.g., Canada).
  • Out-of-Pocket Model: The rich get care, the poor stay sick or die (the model in most poor countries).
  • The U.S. system has elements of all four models.

International Drug Pricing

  • Brand-name drug prices are significantly lower in almost every other developed country than in the U.S.
  • This is because most other countries have a system of government price negotiation or regulation.
  • They use tools like Health Technology Assessment (HTA) and reference pricing to determine what they will pay for a new drug.
  • The U.S. has historically not had such a system, though the IRA is a first step.
  • A key policy debate is whether the U.S. should adopt international reference pricing.
  • A CPPA would analyze the pros and cons of such a policy.

Health Technology Assessment (HTA) Abroad

  • HTA is a formal part of the coverage and reimbursement process in countries like the UK (NICE), Canada (CADTH), and Germany (IQWiG).
  • These bodies conduct rigorous cost-effectiveness analyses of new drugs.
  • Their recommendations determine whether a drug will be covered by the national health system.
  • A CPPA should be familiar with these international HTA bodies.
  • Their analyses are often used as a benchmark by U.S. organizations like ICER.

Parallel Trade

  • Parallel trade, or drug importation, is the practice of buying a drug in a country where it is cheap and reselling it in a country where it is expensive.
  • There have been many policy proposals to legalize the large-scale importation of prescription drugs into the U.S. from Canada and other countries.
  • Proponents argue this would lower prices for U.S. consumers.
  • Opponents, including the pharmaceutical industry and the FDA, raise concerns about safety and the integrity of the supply chain.
  • A CPPA would analyze the economic and safety implications of these proposals.

Lessons from Abroad

  • While no country's system is perfect, there are many lessons the U.S. can learn from international experience.
  • This includes lessons on universal coverage, primary care investment, and cost containment.
  • A CPPA with a global perspective is a more effective analyst.
  • They are able to bring a wider range of policy options and evidence to the table.
  • Understanding international systems provides a valuable benchmark for evaluating the performance of the U.S. system.

The Role of Ideology

  • Health policy debates in the U.S. are often shaped by fundamental ideological disagreements.
  • This includes debates about the proper role of government versus the private market in healthcare.
  • There are different views on whether healthcare is a right or a commodity.
  • These ideological differences often explain why policymakers can look at the same evidence and come to very different conclusions.
  • A CPPA must be aware of this ideological context.

Interest Group Politics

  • The policy process is heavily influenced by a wide range of powerful interest groups.
  • In pharmacy policy, these include the pharmaceutical industry (PhRMA), insurance companies (AHIP), PBMs (PCMA), and professional associations (AMA, APhA).
  • These groups spend billions of dollars on lobbying and campaign contributions to advance their interests.
  • A CPPA must understand the positions and influence of these key interest groups.
  • The final shape of any legislation is often the result of a compromise between these competing interests.

The Role of Public Opinion

  • Public opinion can be a powerful force in the policy process.
  • Elected officials are highly responsive to the views of their constituents, especially on salient issues like healthcare costs.
  • The high cost of prescription drugs is consistently a top concern for voters.
  • This public pressure is what creates the political will to act on an issue.
  • A CPPA should monitor public opinion polls to understand the political environment.

The Federal Budget Process

  • Many major health policy changes are made through the federal budget process.
  • This is because major health programs like Medicare and Medicaid are a huge part of the federal budget.
  • The budget reconciliation process has special parliamentary rules that allow a bill to pass the Senate with a simple majority.
  • This is how major laws like the ACA and the IRA were passed.
  • A CPPA must have a basic understanding of the federal budget process, as it is often the vehicle for significant policy change.

The Analyst in the Political Arena

  • A CPPA must be able to navigate this political environment while maintaining their objectivity.
  • This requires political savvy.
  • It means understanding the motivations and constraints of policymakers.
  • It means framing evidence-based arguments in a way that is persuasive to a political audience.
  • It requires building a reputation as a trusted, non-partisan source of information.
  • This is a challenging but essential part of being an effective policy analyst.

Policy is About Trade-Offs

  • There is rarely a single "perfect" policy solution.
  • Every policy choice involves trade-offs between competing values.
  • The classic trade-off is the "Iron Triangle" of Cost, Quality, and Access.
  • A policy that expands access might increase costs. A policy that cuts costs might reduce quality.
  • Another key trade-off is between innovation and affordability. Strong patent protection encourages innovation but keeps prices high.
  • The role of the policy analyst is not to find a solution with no downsides, but to clearly identify and analyze these trade-offs for policymakers.
  • Understanding that there are no easy answers is a sign of a mature policy analyst.

Evidence is Necessary, but Not Sufficient

  • Policy analysis is grounded in the use of rigorous evidence.
  • A strong recommendation must be supported by data from clinical trials, economic models, and program evaluations.
  • However, evidence alone does not make policy.
  • The policy-making process is inherently political.
  • Values, ideology, stakeholder interests, and public opinion all play a powerful role.
  • A policy that is technically sound according to the evidence may be politically impossible to pass.
  • A successful CPPA understands this and can operate effectively at the intersection of evidence and politics.
  • They know how to frame their evidence-based arguments in a way that is persuasive to policymakers.

Unintended Consequences are Inevitable

  • Every policy intervention in a complex system like healthcare will have unintended consequences.
  • A key part of a policy analyst's job is to anticipate these consequences as much as possible.
  • For example, a policy to limit opioid prescriptions for acute pain might have the unintended consequence of making it harder for chronic pain patients to get needed medication.
  • A policy that shifts costs from the payer to the patient (high deductibles) may have the unintended consequence of reducing adherence to essential medications.
  • A good analysis will always include a section on potential unintended consequences and risks.
  • After a policy is implemented, program evaluation is needed to measure these effects.

Objectivity is the Goal

  • A policy analyst serves the decision-maker, not a particular political position.
  • The goal is to be a neutral, objective, and credible source of information.
  • This is different from an advocate, whose job is to argue for a specific cause.
  • The analyst must be willing to follow the evidence wherever it leads, even if it contradicts their own personal beliefs.
  • This means fairly presenting the pros and cons of all policy options, not just the preferred one.
  • This commitment to objectivity is the foundation of the analyst's credibility.
  • Losing that credibility is a fatal blow to a policy analyst's career.

Communication is a Core Competency

  • A brilliant analysis is useless if it cannot be communicated effectively.
  • A CPPA must be able to translate complex clinical, economic, and legal information into clear, concise language.
  • They must be able to tailor their communication to different audiences, from expert committees to the general public.
  • Strong writing skills are essential for producing effective policy briefs and memos.
  • Strong verbal communication skills are needed for presenting findings and briefing policymakers.
  • The ability to tell a compelling story with data is a key skill.
  • Ultimately, policy analysis is a form of communication.
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