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Certified Rare Disease Pharmacist (CRDP)
Official Examination Content Outline
This document provides the official content outline for the Certified Rare Disease Pharmacist (CRDP) examination. This gold-standard blueprint certifies that a pharmacist possesses the specialized, performance-based competencies required to provide comprehensive care for patients with rare diseases. The outline covers the full spectrum of practice, from advanced clinical and genomic management to global access, health economics, and emerging therapies.
Examination Specifications
| Name of Credential | Certified Rare Disease Pharmacist (CRDP) |
|---|---|
| Certification-Issuing Body | The Council on Pharmacy Standards (CPS) |
| Designation Awarded | CRDP |
| Target Population | Clinical pharmacists in specialty pharmacy, health systems, and managed care. |
| Examination Length | 120 multiple-choice items |
| Administration Time | 3.0 hours |
Examination Content Outline
The CRDP examination is weighted according to the five domains listed below. This structure reflects the modern, interdisciplinary competencies required for rare disease pharmacy practice, ensuring certificants are proficient in all aspects of patient care from diagnosis to long-term management.
| Domain 1: Clinical Management and Pharmacogenomics | 30% |
| Domain 2: Specialty Logistics and Advanced Therapies | 20% |
| Domain 3: Global Patient Access and Health Economics | 20% |
| Domain 4: Evidence Evaluation and Care Coordination | 15% |
| Domain 5: Genomics, Regulatory Pathways, and Emerging Therapies | 15% |
Domain 1: Clinical Management and Pharmacogenomics (30%)
Task 1: Design evidence-based therapeutic plans based on patient-specific clinical and genomic data.
- Integrate pathophysiology, natural history, and patient goals to formulate a comprehensive care plan.
- Apply pharmacogenomic principles to select appropriate therapies and predict patient response.
- Evaluate the clinical significance of variants identified in molecular diagnostic reports (e.g., NGS, WES).
- Design a monitoring plan to track efficacy and toxicity using validated clinical endpoints and biomarkers.
- Manage complex polypharmacy, assessing for drug-disease and drug-drug interactions.
Task 2: Interpret results from newborn screening, genetic panels, and biomarker assays.
- Differentiate between screening and diagnostic genetic tests and assess their clinical validity.
- Evaluate the implications of a positive newborn screening result and manage follow-up testing.
- Correlate specific genotypes with disease phenotypes, prognosis, and therapeutic eligibility.
- Assess the utility of various biomarkers for diagnosis, prognosis, and monitoring of treatment response.
- Communicate complex genetic test results and their clinical implications to patients and interprofessional teams.
Task 3: Manage the unique pharmacological challenges of orphan drugs and novel therapeutics.
- Analyze the mechanisms of action, pharmacokinetics, and pharmacodynamics of rare disease therapies.
- Develop strategies to manage and mitigate the unique adverse effect profiles of orphan drugs.
- Apply dosing adjustments for pediatric patients and those with organ dysfunction.
- Assess the risk of immunogenicity and manage the clinical implications of neutralizing antibodies.
- Evaluate the pharmacology of emerging modalities like antisense oligonucleotides, siRNAs, and enzyme replacement therapies.
Task 4: Develop protocols for the management of treatment-related toxicities and supportive care needs.
- Design management plans for acute events such as infusion-related reactions, anaphylaxis, and cytokine release syndrome.
- Implement proactive monitoring to detect and manage long-term or cumulative drug toxicities.
- Integrate non-pharmacologic and supportive care measures to manage disease symptoms and treatment side effects.
- Manage the safe use of REMS drugs with Elements to Assure Safe Use (ETASU).
- Differentiate between an adverse drug event and a manifestation of the underlying disease.
Task 5: Apply principles of genetic counseling in patient communication.
- Explain patterns of inheritance and recurrence risk to patients and families.
- Assess patient understanding of genetic information using health literacy and teach-back principles.
- Manage the ethical, legal, and social implications (ELSI) of genetic testing, including privacy and potential discrimination.
- Facilitate informed decision-making regarding genetic testing for at-risk family members.
- Collaborate with certified genetic counselors to provide comprehensive patient support.
Task 6: Manage the transition of care for patients with rare diseases across different life stages and care settings.
- Design a structured transition plan for adolescent patients moving from pediatric to adult care providers.
- Execute a comprehensive medication reconciliation process during any care transition (e.g., hospital admission/discharge).
- Ensure continuity of access to critical therapies during changes in insurance or location of care.
- Educate receiving care teams who may be unfamiliar with the patient's rare disease.
- Coordinate with the multidisciplinary team to address the medical, psychosocial, and logistical aspects of care transitions.
Domain 2: Specialty Logistics and Advanced Therapies (20%)
Task 1: Manage the supply chain for high-cost, limited distribution, and temperature-sensitive medications.
- Navigate the operational requirements for procuring drugs from limited distribution drug (LDD) networks.
- Design and implement protocols to ensure cold chain integrity from procurement to administration.
- Manage inventory of high-cost medications to minimize waste and prevent therapy interruptions.
- Troubleshoot supply chain disruptions and coordinate with manufacturers and distributors to resolve issues.
- Ensure compliance with all data reporting and contractual requirements of LDD agreements.
Task 2: Oversee the handling, preparation, and administration of Advanced Therapy Medicinal Products (ATMPs).
- Apply standards for the safe handling and preparation of gene therapies (e.g., AAV-based) and cell therapies (e.g., CAR-T).
- Manage the chain of identity and chain of custody for autologous and allogeneic cell therapies.
- Develop institutional protocols for the unique storage requirements of ATMPs, including cryopreservation.
- Coordinate just-in-time delivery and administration of patient-specific therapies with infusion centers.
- Educate clinical staff on the preparation, administration, and immediate monitoring requirements for ATMPs.
Task 3: Manage long-term safety monitoring and patient registries for novel therapies.
- Design patient-specific, long-term follow-up plans to monitor for delayed adverse events from gene and cell therapies.
- Manage the operational aspects of patient enrollment and data submission to post-marketing safety registries.
- Assess registry data for emerging safety signals or trends in long-term outcomes.
- Ensure compliance with regulatory requirements for long-term follow-up (e.g., 15 years for gene therapies).
- Communicate the importance of long-term follow-up and registry participation to patients and caregivers.
Task 4: Implement wastage mitigation and financial stewardship programs for ultra-high-cost therapies.
- Design protocols for dose rounding and vial sharing to minimize the waste of expensive medications.
- Evaluate the financial impact of different dosing strategies and vial sizes.
- Implement inventory management systems to track and reduce drug waste.
- Collaborate with payers to establish reimbursement policies for unavoidable drug waste.
- Analyze and report on the financial savings achieved through stewardship initiatives.
Task 5: Manage investigational drug services for rare disease clinical trials.
- Apply Good Clinical Practice (GCP) principles to the management of investigational drugs.
- Oversee the procurement, storage, accountability, and dispensing of study medications.
- Design protocols for the sterile or non-sterile preparation of investigational products, including blinding procedures.
- Ensure compliance with all sponsor and regulatory requirements for record-keeping and documentation.
- Collaborate with the principal investigator and research team to maintain the integrity of the study protocol.
Task 6: Coordinate complex administration across various sites of care.
- Evaluate and select appropriate sites of care (e.g., hospital outpatient, infusion suite, home infusion) based on drug and patient needs.
- Develop and disseminate detailed administration guidelines for infusion nurses and other healthcare providers.
- Manage the logistics of scheduling and coordinating infusions, especially for therapies requiring specialized monitoring.
- Troubleshoot administration-related challenges, such as difficult IV access or device malfunctions.
- Ensure all sites of care meet the safety and handling requirements for the specific therapy.
Domain 3: Global Patient Access and Health Economics (20%)
Task 1: Design and execute comprehensive strategies to secure medication access and financial assistance.
- Manage multi-level prior authorization appeals, including peer-to-peer reviews and external appeals.
- Assemble and submit robust clinical arguments to payers, integrating evidence and letters of medical necessity.
- Navigate manufacturer patient assistance programs (PAPs), co-pay programs, and independent charitable foundations.
- Develop strategies to mitigate the impact of accumulator and maximizer programs on patient out-of-pocket costs.
- Challenge restrictive payer policies that conflict with established standards of care or FDA labels.
Task 2: Apply principles of health economics and outcomes research (HEOR) to demonstrate product value.
- Evaluate cost-effectiveness and budget impact models for ultra-high-cost therapies.
- Interpret value frameworks from organizations like the Institute for Clinical and Economic Review (ICER).
- Analyze the concepts of quality-adjusted life-years (QALYs) and their application in rare disease value assessment.
- Synthesize real-world evidence to demonstrate the long-term clinical and economic value of a therapy.
- Communicate the value proposition of a rare disease therapy to payers and health system decision-makers.
Task 3: Manage market access strategies for novel and high-cost therapies.
- Assess the implications of different payment models, such as outcomes-based agreements, for rare disease therapies.
- Develop formulary submission dossiers that articulate the clinical and economic value of a new product.
- Differentiate between the access and reimbursement pathways for pharmacy versus medical benefits.
- Analyze how payer utilization management tools (e.g., step therapy, quantity limits) impact patient access.
- Contribute to the development of institutional policies for managing the financial impact of high-cost drugs.
Task 4: Navigate global health policy and cross-border access for rare diseases.
- Compare international rare disease frameworks and orphan policies (e.g., FDA, EMA, PMDA, WHO).
- Assess the role of international patient registries and advocacy networks in shaping global policy.
- Manage the logistical and regulatory challenges of cross-border therapy importation and early access programs.
- Evaluate the impact of World Health Organization (WHO) policies and initiatives on rare disease care.
- Advocate for policy changes at the local, national, and international levels to improve access to care.
Task 5: Lead patient advocacy and engagement initiatives.
- Collaborate with patient advocacy organizations to develop educational resources and support programs.
- Empower patients and caregivers with the skills and knowledge for effective self-advocacy.
- Integrate the patient perspective and patient-reported outcomes into clinical care and research.
- Connect patients and families with peer support networks and community resources.
- Ensure that care delivery is culturally competent and patient-centered.
Task 6: Differentiate global regulatory frameworks for orphan products.
- Compare the criteria for orphan drug designation between the FDA (Orphan Drug Act) and the EMA (Regulation EC No 141/2000).
- Evaluate the different market exclusivities and financial incentives offered in the US, EU, and Japan.
- Assess how different regulatory bodies approach the use of surrogate endpoints and accelerated approvals for rare diseases.
- Manage the submission of orphan drug designation applications to global health authorities.
- Analyze how global regulatory convergence initiatives impact orphan product development.
Domain 4: Evidence Evaluation and Care Coordination (15%)
Task 1: Critically appraise clinical evidence in the context of rare diseases.
- Evaluate the design and limitations of small-population clinical trials.
- Assess the validity and applicability of real-world evidence, including patient registries and natural history studies.
- Differentiate between statistical significance and clinical meaningfulness of trial endpoints.
- Synthesize evidence from disparate sources (e.g., clinical trials, case reports, mechanistic data) to guide clinical decisions.
- Analyze clinical practice guidelines and consensus statements for their applicability to individual patients.
Task 2: Lead interprofessional care teams and coordinate complex patient care.
- Lead multidisciplinary rare disease care teams, integrating genetic counselors, specialists, and advocacy stakeholders.
- Facilitate collaboration between local providers and specialists at national Centers of Excellence.
- Design and implement patient-centered, shared care plans that span multiple specialties.
- Apply telemedicine, digital health platforms, and remote monitoring tools to coordinate rare disease care.
- Ensure seamless communication and care continuity across the entire healthcare team.
Task 3: Design and implement comprehensive patient and caregiver education programs.
- Develop educational materials that adhere to health literacy principles for complex topics like genetics and advanced therapies.
- Assess patient and caregiver understanding using methods like teach-back.
- Provide training on medication administration, including self-injection techniques and infusion management.
- Create customized adherence strategies based on an assessment of individual patient barriers.
- Empower patients with knowledge to become active participants in their care.
Task 4: Contribute to outcomes research and quality improvement initiatives.
- Design and evaluate long-term outcomes in rare disease registries, including patient-reported outcomes and quality-of-life metrics.
- Manage the collection and submission of data to patient registries and other real-world evidence platforms.
- Analyze quality improvement data to identify and address gaps in care.
- Disseminate research findings through publications and presentations to advance the field.
- Use outcomes data to demonstrate the value of specialized pharmacy services for patients with rare diseases.
Task 5: Manage the psychosocial impact of rare diseases on patients and families.
- Assess patients and caregivers for psychosocial distress, including anxiety, depression, and caregiver burnout.
- Provide empathetic support and connect individuals with mental health professionals and support groups.
- Integrate psychosocial assessment into the routine clinical care workflow.
- Develop resources to help families navigate the challenges of the diagnostic odyssey and chronic illness.
- Collaborate with social workers and case managers to address non-medical barriers to care.
Task 6: Evaluate the ethical frameworks for resource allocation and genetic medicine.
- Apply principles of bioethics to clinical dilemmas in rare disease care.
- Analyze the ethical considerations surrounding access to ultra-high-cost therapies and resource allocation.
- Assess the ethical implications of genetic testing, including pre-symptomatic testing and incidental findings.
- Navigate conflicts of interest in a healthcare landscape with significant industry involvement.
- Advocate for equitable and just access to care for all patients with rare diseases.
Domain 5: Genomics, Regulatory Pathways, and Emerging Therapies (15%)
Task 1: Apply principles of human genetics and molecular diagnostics in rare disease management.
- Differentiate between various types of genetic mutations (e.g., missense, nonsense, frameshift) and their functional impact.
- Evaluate the diagnostic yield and limitations of different testing platforms (e.g., single gene, panel, WES/WGS).
- Assess the clinical actionability of genetic variants based on established classification guidelines (e.g., ACMG).
- Apply genetic findings to confirm diagnoses, predict disease course, and guide therapy selection.
- Manage the interpretation of variants of uncertain significance (VUS) in clinical practice.
Task 2: Evaluate global regulatory pathways for orphan drugs and accelerated approvals.
- Manage the process for obtaining Orphan Drug Designation from the FDA and the EMA.
- Assess a drug's eligibility for expedited pathways such as FDA's Accelerated Approval and EMA's Conditional Marketing Authorisation.
- Design post-marketing confirmatory study plans required for converting an accelerated to a full approval.
- Analyze the use of novel clinical trial designs (e.g., basket trials, n-of-1 trials) in rare disease drug development.
- Differentiate the evidentiary standards for approval among major global health authorities.
Task 3: Manage patient enrollment in clinical trials, registries, and expanded access programs (EAPs).
- Assess patient eligibility for participation in interventional clinical trials.
- Facilitate patient access to investigational therapies through EAPs ("compassionate use").
- Manage the operational requirements for enrolling patients in long-term observational registries.
- Differentiate the regulatory frameworks for clinical trials versus EAPs.
- Apply ethical principles to ensure informed consent and protect patient welfare in the research setting.
Task 4: Assess the unique regulatory and clinical requirements for gene and cell therapies.
- Evaluate the specific CMC (Chemistry, Manufacturing, and Controls) challenges for ATMPs.
- Manage the clinical requirements for long-term follow-up to monitor the durability and safety of gene therapies.
- Assess the regulatory pathways for specific ATMPs, such as CAR-T cells and AAV-based gene replacement therapies.
- Differentiate the regulatory frameworks for ATMPs between the US (RMAT designation) and EU (ATMP Regulation).
- Apply risk management principles to the unique safety profiles of gene and cell therapies.
Task 5: Apply ethical principles to genetic testing and precision medicine in rare pediatric populations.
- Manage the ethical complexities of pre-symptomatic and carrier testing in minors.
- Assess the challenges of obtaining informed consent and assent from pediatric patients and their families for genomic medicine.
- Evaluate the ethical considerations of using novel or off-label therapies in children with rare diseases.
- Navigate dilemmas related to the return of secondary or incidental genetic findings.
- Advocate for the equitable inclusion of pediatric patients in clinical research.
Task 6: Evaluate emerging therapeutic technologies and their clinical application.
- Assess the mechanism of action and therapeutic potential of novel modalities like CRISPR/gene editing and mRNA therapies.
- Analyze the clinical development and potential applications of radiopharmaceuticals in rare diseases.
- Evaluate the role of digital therapeutics and artificial intelligence in rare disease management.
- Monitor the evolving regulatory landscape for these emerging technologies.
- Translate the science of novel platforms into understandable concepts for patients and providers.
