CASP Module 25, Section 3: Decentralized Trials and Digital Therapeutics
MODULE 25: YOUR GUIDE TO THE GLOBAL & FUTURE LANDSCAPE

Section 25.3: Decentralized Trials and Digital Therapeutics

Exploring the rise of remote clinical trial models and FDA-approved digital therapeutics (DTx) in specialty care, focusing on the HSSP’s role in patient recruitment, remote monitoring, data integration, and managing unique access/reimbursement pathways.

SECTION 25.3

Decentralized Trials and Digital Therapeutics

Beyond the Pill Bottle: The HSSP as Research Partner and Tech Integrator.

25.3.1 The “Why”: Healthcare is Moving Beyond the Clinic Walls

For centuries, healthcare has been tethered to physical locations: the doctor’s office, the hospital, the local pharmacy. Clinical research, the engine of medical innovation, was even more centralized, requiring patients to travel, often long distances, to participate in trials at major academic centers. Your HSSP practice itself is often physically located within the hospital or a clinic. However, the confluence of digital technology, the COVID-19 pandemic, and a push for greater patient centricity is rapidly dismantling these geographic constraints.

Two major forces are driving this shift, and both fundamentally reshape the role and opportunities for the HSSP:

  1. Decentralized Clinical Trials (DCTs): The traditional clinical trial model is notoriously slow, expensive, and struggles with patient recruitment and retention, particularly among diverse populations. DCTs leverage technology (telehealth, remote monitoring devices, mobile apps, direct-to-patient drug shipment) to conduct trial activities in or near the patient’s home, rather than requiring frequent visits to a central site.
  2. Digital Therapeutics (DTx): Beyond simply monitoring or delivering care remotely, DTx represent a new therapeutic modality altogether. These are FDA-approved, evidence-based software applications designed to prevent, manage, or treat a medical condition. They are prescribed by clinicians, often alongside or instead of traditional medications.

Why must the HSSP master these domains?

  • You Are the Bridge Between Research and Care: Your health system’s research department may run DCTs, but who understands the complexities of managing investigational drug logistics for specialty biologics shipped directly to patients? Who can integrate trial requirements into the EMR and existing clinical workflows? You can.
  • You Are the Medication Expert, Period: DTx are prescribed. They have indications, contraindications, and require patient adherence. They need to be evaluated for safety and efficacy by P&T committees. They need a pathway for access and reimbursement. Who is better positioned than the pharmacist to manage this new class of therapeutics? No one.
  • You Control Key Infrastructure: Your specialty pharmacy already has the licenses, processes, temperature-controlled shipping capabilities, and patient support infrastructure needed to support complex DCT logistics. Your EMR access and data analytics capabilities are essential for identifying eligible patients for both DCTs and DTx.
  • Patient Trust and Technology Adoption: Patients trust their pharmacists. As these new technologies emerge, patients will look to you to explain how they work, how to use them, and whether they are safe and effective. Your role as educator and advocate is paramount.

This section is your guide to navigating this new frontier. We will move beyond the buzzwords and provide practical, actionable frameworks for integrating DCTs and DTx into your HSSP practice. This is not just about adapting to change; it’s about leading it. Your ability to leverage these innovations will significantly enhance your value proposition to your patients, your providers, and your health system’s strategic goals.

Pharmacist Analogy: The “Pharmacy Without Walls”

Think about the evolution of retail over the last 20 years. For decades, the model was the brick-and-mortar store. Then came Amazon.

1. The Traditional “Brick-and-Mortar” Pharmacy:
This is the traditional clinical trial site or the standard specialty pharmacy model. Everything happens within your four walls. Patients must come to you for prescriptions, counseling, and monitoring. Clinical trials require patients to physically visit the research center for every assessment and drug dispensing.

2. Decentralized Clinical Trials (DCTs) – The “Amazon Prime” for Research:
This is like Amazon deciding they don’t need physical stores everywhere. They leverage technology (the internet, logistics networks) to bring the “store” (the clinical trial) to the customer’s (patient’s) home.
Instead of coming to the research center, the patient uses telehealth for visits. Wearable sensors collect vital signs. Mobile apps collect patient-reported outcomes. And crucially, the investigational drug is shipped directly from a central pharmacy (often leveraging specialty pharmacy capabilities) to the patient’s home.
Your HSSP role shifts from being the only dispensing site to potentially becoming the central logistics hub and remote clinical monitor, managing drug accountability, patient training on devices, and data integration across hundreds of patients spread across the country.

3. Digital Therapeutics (DTx) – The “Product” Becomes Software:
This is like Amazon not just selling physical books, but also selling the Kindle and the e-books themselves. The “product” is now software.
A DTx is prescribed software that directly treats a condition. Instead of just dispensing a pill for insomnia, you might now “dispense” (provide access to) an FDA-approved Cognitive Behavioral Therapy app.
Your HSSP role expands dramatically. You’re not just managing drug inventory; you’re managing software licenses. You’re not just counseling on side effects; you’re troubleshooting app connectivity. You’re not just processing drug claims; you’re navigating entirely new billing codes and reimbursement pathways for software. You become the expert in evaluating, implementing, and managing software as a medical intervention.

Both DCTs and DTx represent a fundamental shift from a place-based model of care and research to a patient-centered, technology-enabled model. Your ability to adapt your skills and infrastructure to this “pharmacy without walls” is essential for future success.

25.3.2 Deep Dive: Decentralized Clinical Trials (DCTs)

Decentralized Clinical Trials are not an all-or-nothing concept. Most modern trials are actually hybrid DCTs, incorporating some decentralized elements while still retaining some site-based visits. The key is using technology to reduce the burden on patients and broaden access.

Why the Shift to DCTs?

  • Patient Recruitment & Retention: The #1 reason trials fail or are delayed. Requiring patients to travel hours for frequent visits is a massive barrier. DCTs allow patients anywhere to participate.
  • Diversity & Inclusion: Traditional site-based trials often over-enroll patients who live near major academic centers, who tend to be less diverse. DCTs can reach rural, minority, and less mobile populations.
  • Real-World Data: Collecting data via wearables and apps in the patient’s home environment may provide a more accurate picture of a drug’s effect than data collected during artificial site visits.
  • Efficiency & Cost: While the technology investment is significant, DCTs can potentially reduce the need for expensive physical site infrastructure and speed up enrollment, leading to faster drug development.
  • Pandemic Resilience: COVID-19 shut down traditional trials overnight. DCTs, already emerging, became essential for research continuity. The FDA issued guidance rapidly, accelerating adoption.

Components of a DCT Model

A DCT leverages a toolkit of technologies and services:

Component Description Examples HSSP Relevance
Telehealth / Virtual Visits Replacing in-person site visits with video conferencing for consultations, adverse event assessment, etc. Zoom, Doxy.me, dedicated trial platforms. Potential role in remote patient counseling, adherence checks, AE monitoring.
Remote Patient Monitoring (RPM) Using wearables or connected devices to collect physiological data continuously or periodically from home. Smartwatches (ECG, HR), continuous glucose monitors (CGMs), Bluetooth blood pressure cuffs, spirometers. Integrating device data into patient management workflows, troubleshooting device issues, interpreting data for AEs.
eConsent Obtaining and documenting informed consent electronically, often via video and e-signatures. DocuSign, specialized eConsent platforms. Ensuring proper documentation, verifying patient understanding remotely.
ePRO / eCOA Electronic Patient-Reported Outcomes / Clinical Outcome Assessments collected via mobile apps or web portals. Symptom diaries, quality of life surveys, adherence logs completed on a smartphone app. Monitoring patient-reported data for efficacy signals or safety concerns, driving adherence interventions.
Local / Mobile Healthcare Providers Using local labs, imaging centers, or mobile nurses/phlebotomists to perform assessments closer to the patient’s home. Labcorp/Quest home draws, mobile nursing services for infusions or study procedures. Coordinating with local providers, ensuring protocol adherence outside the central site.
Direct-to-Patient (DtP) Investigational Product (IP) Shipment Shipping the study drug directly from a central pharmacy or depot to the patient’s home. Specialty couriers delivering temperature-controlled biologics, oral IP sent via mail. CORE HSSP ROLE: Managing logistics, ensuring temperature integrity, drug accountability, returns/destruction, patient training on self-administration.

The HSSP’s Evolving Role in DCTs: From Dispenser to Logistics & Data Hub

Your health system’s participation in DCTs presents a massive opportunity for the HSSP to expand its value beyond traditional dispensing. You are uniquely positioned to solve the logistical and clinical challenges inherent in this model.

HSSP as the DCT Implementation Partner: Key Roles

When your health system’s research department considers a DCT protocol, you should be at the table from Day 1. Here’s how you contribute:

  1. Feasibility Assessment (Pharmacy Perspective):
    • “Does the Investigational Product (IP) require complex compounding we can’t do remotely?”
    • “What are the storage/shipping requirements (cold chain, ambient)? Can we guarantee integrity to the patient’s home?”
    • “What are the drug accountability requirements? How will we track dispensing, returns, and destruction remotely?”
    • “Does the protocol require patient training on self-injection or device use? Can we do this effectively via video?”
    • “What state licensing requirements apply if we ship IP across state lines?”
  2. Patient Identification & Recruitment:
    • Leverage your access to the EMR and specialty pharmacy data to identify potentially eligible patients based on diagnosis, prior treatments, etc.
    • Act as a trusted point of contact to introduce the trial opportunity to patients already under your care.
  3. Investigational Drug Services (IDS) Logistics Hub:
    • Serve as the central pharmacy depot for receiving, storing, and shipping IP directly to patients according to protocol randomization and visit schedules.
    • Implement robust procedures for temperature monitoring during shipping (e.g., using validated shippers and temp monitors).
    • Develop clear workflows for managing drug returns, temperature excursions, and destruction of unused IP, all documented meticulously for FDA/sponsor audits.
    • Manage drug accountability logs electronically, tracking every dose from receipt to patient administration or return.
  4. Remote Patient Education & Support:
    • Develop video training modules and conduct live telehealth sessions to teach patients how to store, prepare, and self-administer IP.
    • Serve as the primary point of contact for patients with drug-related questions or device malfunctions.
    • Proactively monitor adherence through ePRO data or smart device technology (e.g., connected autoinjectors).
  5. Data Integration & Safety Monitoring:
    • Work with IT to integrate data feeds from RPM devices or ePRO apps into the patient’s EMR or a dedicated trial dashboard.
    • Monitor incoming patient data (e.g., vital signs, reported symptoms) for potential adverse events requiring escalation to the research team.
    • Ensure seamless communication between the HSSP team, the patient, the local providers (if any), and the central research site/sponsor.

Tutorial: Reviewing a DCT Protocol for Pharmacy Feasibility

Imagine you receive a draft DCT protocol for a new subcutaneous biologic for Multiple Sclerosis. Here is your HSSP checklist for the pharmacy feasibility review:

  • IP Details:
    • Formulation: Liquid in pre-filled syringe (PFS)? Autoinjector? Lyophilized powder requiring reconstitution?
    • Dosage: Fixed dose? Weight-based?
    • Storage: Refrigerated (2-8°C)? Frozen? Room temperature? Excursion allowances?
    • Administration: Subcutaneous? Intramuscular? Self-administered or requires healthcare professional (HCP)?
  • DtP Logistics:
    • Shipment Frequency: Monthly? Every 3 months?
    • Packaging: Sponsor providing validated shippers and temp monitors, or must pharmacy develop?
    • Delivery: Courier requirements? Signature required? Can it be left on porch? Contingency for missed deliveries?
    • State Lines: Will you be shipping outside your state? Do you have non-resident pharmacy licenses for those states?
  • Drug Accountability:
    • Dispensing Logs: What specific data points must be captured? How is randomization confirmed?
    • Return Process: How should patients return unused/empty devices? Temperature requirements for returns?
    • Destruction: On-site destruction protocol? Need for witness? Documentation requirements?
  • Patient Training:
    • Device: Standard PFS/Autoinjector or novel device requiring extensive training?
    • Training Materials: Provided by sponsor or need to be developed? Video? Live telehealth required?
    • Competency Assessment: How is patient’s ability to self-administer documented?
  • Technology Integration:
    • ePRO/Apps: Is patient required to use an app? Who provides tech support?
    • RPM Devices: Are wearables involved? Who manages device provisioning and data monitoring?
    • Communication Platform: How will HSSP communicate with patient and research site? Secure messaging? Dedicated platform?
  • Budget & Resources:
    • Pharmacy Time: How many FTE hours estimated for setup, dispensing, shipping, training, monitoring per patient?
    • Supplies: Cost of shippers, monitors, ancillary supplies (sharps containers, alcohol swabs)?
    • Technology: Any specific software or hardware needed?

By systematically reviewing these points, you can provide the research team with a clear “go/no-go” recommendation from the pharmacy perspective, identify potential challenges early, and accurately budget the resources needed for successful execution. Your proactive involvement here prevents major operational headaches down the line.

25.3.3 Deep Dive: Digital Therapeutics (DTx)

Shift gears. We are moving from optimizing the *delivery* of traditional drugs (DCTs) to managing *software* as the drug itself. Digital Therapeutics (DTx) are a distinct category, rigorously tested and FDA-authorized, that require a completely new mindset and skillset from the HSSP.

What DTx Are (and Are Not)

The digital health landscape is crowded. It’s crucial to differentiate DTx from the thousands of unregulated “wellness apps.”

Category Definition Regulation Claims Examples HSSP Relevance
Wellness Apps Apps promoting general health, fitness, lifestyle (e.g., calorie counters, meditation guides). Generally Unregulated by FDA. Cannot claim to treat or diagnose specific diseases. MyFitnessPal, Calm, Fitbit app. Low. Generally outside pharmacy scope unless integrated into a broader health plan wellness program.
Digital Health / Monitoring Apps Apps that track health data, may provide feedback or connect to providers, but don’t directly deliver therapy. May be regulated as medical devices if making diagnostic claims. Variable (FDA oversight depends on risk and claims). May claim to help “manage” a condition or track data, but not “treat.” Connected CGM apps, remote BP monitoring platforms, symptom trackers for MS. Moderate. Data may integrate with HSSP workflows for monitoring adherence or AEs.
Digital Therapeutics (DTx) Software intended to treat, manage, or prevent a disease or disorder. Subject to rigorous clinical testing and FDA authorization (e.g., 510(k), De Novo). FDA Regulated as Software as a Medical Device (SaMD). Can make treatment claims based on proven clinical evidence. reSET (substance use disorder), EndeavorRx (pediatric ADHD), Mahana IBS (irritable bowel syndrome). High. Prescribed interventions requiring HSSP expertise in evaluation, access, reimbursement, patient onboarding, and monitoring.

Examples of DTx Relevant to Specialty Pharmacy

DTx are emerging across numerous specialty areas:

  • Psychiatry/Neurology: Cognitive Behavioral Therapy (CBT) apps for depression/anxiety (often co-prescribed with SSRIs), ADHD attention training games, potential apps for MS symptom management or cognitive rehab post-stroke.
  • Substance Use Disorders: Apps providing contingency management and therapy alongside medication-assisted treatment (MAT).
  • Gastroenterology: CBT-based apps for Irritable Bowel Syndrome (IBS).
  • Endocrinology: Apps integrated with CGMs providing automated insulin dosing recommendations (regulated as device control software).
  • Oncology: Emerging apps for managing chemotherapy side effects (e.g., nausea, fatigue) or providing psychosocial support.
  • Cardiology: Apps for cardiac rehabilitation or managing heart failure.

The HSSP’s Role in Integrating DTx: The New Formulary Frontier

Imagine a provider wants to prescribe “Somryst,” an FDA-authorized DTx delivering CBT for chronic insomnia. Where does this prescription go? How does the patient get access? How does the provider (or the health system) get paid? These are the questions you, the HSSP, will be tasked with answering.

HSSP Master Checklist for DTx Integration

Integrating the first DTx into your health system is a major project. You will likely lead or co-lead this effort. Here is your roadmap:

  1. Clinical Evaluation (P&T Committee):
    • “What is the evidence?” Review the pivotal trials submitted to the FDA. Is the benefit clinically meaningful? How does it compare to standard of care (drugs, traditional therapy)?
    • “What is the indication?” Is it narrow or broad? First-line or refractory? Adjunct or monotherapy?
    • “What are the risks?” Data privacy/security? Potential for misuse? Software glitches? Equity concerns (requires smartphone/internet)?
    • Your Role: Prepare the P&T monograph, present the evidence, make a formulary recommendation (just like any drug).
  2. Prescribing & Fulfillment Workflow:
    • “How is it prescribed?” Does it need a specific order in the EMR? Can any provider prescribe, or only specialists?
    • “How does the patient get access?” Does the prescription go to the HSSP? Does the manufacturer’s hub handle it? Is there an “access code” generated?
    • “Who ‘dispenses’ it?” Does the HSSP provide the access code and onboarding instructions? Is it entirely handled by the DTx company?
    • Your Role: Design the EMR order. Define the fulfillment pathway (HSSP vs. Hub). Create patient education materials.
  3. Reimbursement & Billing (The Black Box):
    • “Is there a billing code?” Some DTx have unique HCPCS codes (e.g., A-codes, K-codes). Others rely on remote patient monitoring (RPM) CPT codes or general telehealth codes.
    • “Who covers it?” Is it on the Pharmacy Benefit (via PBM)? Medical Benefit (via health plan)? Is it carved out? Is there *any* payer coverage yet? (Often, no).
    • “How does the provider/system get paid?” Fee-for-service based on code? Included in a value-based care bundle? Is there a direct contract with the manufacturer?
    • Your Role: Work with Revenue Cycle, Managed Care, and Finance to understand (or create!) the reimbursement strategy. This is often the biggest barrier.
  4. Patient Onboarding & Support:
    • “Who teaches the patient how to use the app?” The HSSP team? A dedicated DTx support specialist? The manufacturer’s hub?
    • “Who handles tech support?” App crashes? Login issues? Device compatibility?
    • Your Role: Develop patient training resources. Establish clear lines of responsibility for technical vs. clinical support.
  5. Data Integration & Monitoring:
    • “Does the DTx generate data?” Adherence logs? Symptom scores? Physiologic data?
    • “Where does the data go?” Does it flow back into the EMR? Is there a separate provider portal? Who monitors it?
    • “How is progress assessed?” How does the provider know if the DTx is working?
    • Your Role: Work with IT/Informatics to establish data flow (if possible). Define pharmacist’s role in monitoring DTx data for safety, efficacy, and adherence, similar to managing drug therapy.

Tutorial: Navigating DTx Reimbursement Pathways

This is the Wild West. Unlike drugs, there is no single, established pathway for DTx reimbursement. You need to understand the potential avenues and their challenges.

Pathway How it Works Pros Cons / Challenges
Pharmacy Benefit (via PBM) DTx is assigned an NDC-like number, placed on formulary, adjudicated like a drug claim. Patient pays copay. Utilizes existing, familiar infrastructure. Clear patient cost-sharing. PBMs have been slow to adopt. Requires complex contracting. Doesn’t fit well for DTx needing clinical monitoring.
Medical Benefit (via Health Plan) DTx is billed using HCPCS or CPT codes, often requiring prior authorization. May fall under DME (Durable Medical Equipment) or specific therapy benefits. Allows for prior auth and medical necessity review. May align better with value-based care models. Lack of established codes. Coverage policies are highly variable and often non-existent. High patient deductibles/coinsurance may apply.
Remote Patient Monitoring (RPM) / Remote Therapeutic Monitoring (RTM) Codes Provider bills existing CPT codes for time spent onboarding patient, monitoring data, and adjusting care based on DTx output. Leverages existing billing codes. Focuses on the *service* provided around the DTx. Only covers provider time, not the cost of the DTx itself. Requires careful time tracking and documentation.
Direct Purchase / Cash Pay Patient pays out-of-pocket for a subscription or access code. Simple fulfillment. No payer hurdles. Major access barrier for most patients. Not a sustainable model for broad adoption.
Employer / Health System Contract Employer or health system pays a bulk license fee or per-member-per-month (PMPM) fee directly to the DTx manufacturer for access. Bypasses traditional payer complexity. Can be integrated into wellness or population health programs. Requires significant upfront investment/negotiation. May only cover specific employee/patient populations.

Your HSSP Role: You must become your system’s internal expert on these pathways. You will work with managed care to understand payer policies, with revenue cycle to implement billing processes, and with finance to analyze the cost-effectiveness of different models. You may even help negotiate direct contracts with DTx manufacturers.

25.3.4 Synergies and Future State: The HSSP at the Nexus

DCTs and DTx are not isolated trends; they are converging, and the HSSP sits directly at their nexus. Imagine a future state:

A patient with Parkinson’s Disease is enrolled in a hybrid DCT run through your health system.

  • They receive the investigational oral drug via DtP shipment managed by your HSSP team.
  • They wear a sensor (RPM device) that continuously monitors tremors and gait.
  • They use an FDA-approved DTx app on their phone that delivers virtual physical therapy exercises and collects daily symptom scores (ePRO).
  • Your HSSP team receives alerts from the RPM device indicating worsening tremors. You review the ePRO data showing decreased adherence to the DTx exercises. You conduct a telehealth visit with the patient, counsel them on adherence, review IP administration technique, assess for AEs, and coordinate a dose adjustment discussion with the Principal Investigator.

In this future state, you are not just a dispenser. You are a remote trial coordinator, a digital health coach, a data analyst, and a clinical interventionist, all leveraging technology to manage complex patients outside the traditional clinic walls. This requires a significant evolution of skills, including data literacy, comfort with digital health platforms, and expertise in remote communication strategies.

Conclusion: The decentralization of research and the digitization of therapeutics are redefining the boundaries of pharmacy practice. For the HSSP, this is not a threat, but an unprecedented opportunity. By embracing your role as a logistics expert for DCTs and the primary manager for DTx, you position yourself at the forefront of innovation, driving value for your patients and cementing the HSSP as an essential leader in the future of specialty care delivery.