System Thinking to Drive Patient Engagement: Walking the Talk

Key Participants and Organizational Roles:

• Susan Schaefer Good (Moderator): Founding President/CEO of Patients Academy for Research Advocacy; former editor of Biocentury.
• Julie Gerberding: Executive Vice President and Chief Patient Officer at Merck; former CDC Director.
• Ivor Horn: Chief Medical Officer at Accolade (concierge health advocacy).
• Kathy Hudson: Founding CEO of the People-Centered Research Foundation (PCRF); involved in PCORnet.
• Bray Patrick Lake: Director of Stakeholder Engagement at Duke Clinical Research Institute (DCRI); Senior Fellow at Faster Cures.
• Janet Woodcock: Director of the Center for Drug Evaluation and Research (CDER) at the FDA.

Regulatory Milestones and Policy Shifts:

• PDUFA (Prescription Drug User Fee Act): Patients were active participants in the 2012 and 2017 reauthorizations; FDA committed to seeking patient input and integrating perspectives into regulatory decisions.
• 21st Century Cures Act: Mandated public workshops on patient-focused drug development and the issuance of guidances to make patient experience data integral to product development.
• Breakthrough Therapy Designation: Initially conceived by Friends of Cancer Research to accelerate development for serious conditions.
• FDA Guidance for Duchenne Muscular Dystrophy: Initially drafted by Parent Project Muscular Dystrophy.
• Cures Act Requirements: Now mandates that patient data be included in benefit-risk discussions during drug evaluations.

Industry Structural Changes:

• Chief Patient Officer (CPO): The first CPO role was created in 2014 at Merck; subsequent adoption by other biopharmaceutical companies to reorganize teams for patient-centered practices.
• Patient-Led Research: Organizations like the Multiple Myeloma Research Foundation now fund and conduct large complex platform clinical trials (e.g., the COMPASS study).
• Employer-Hired Advocacy: Accolade operates as a concierge advocacy service hired by self-insured employers to help 1.5 million+ individuals navigate benefits and care.

Evidence on Engagement Efficacy:

• Clinical Trial Amendments: Data from the Tufts Center for Study of Drug Development indicates average Phase 3 trials undergo four amendments, costing approximately $500,000 and delaying implementation by eight weeks each; early patient engagement can prevent such amendments.
• Value Metrics: Patient engagement levers (Duke Clinical Research Institute model) map to improved net present value by reducing risk, cost, and time to market while enhancing revenue potential.
• Historical Discrepancies: FDA reviewers historically admitted to 25 years of practice without fully understanding the patient's disease burden or treatment suffering (e.g., "I never knew").

Strategic Priorities and Best Practices:

• Timing of Engagement: Participants emphasized the need to engage patients before protocol design (IND/NDA stages) rather than after decisions are made to avoid costly rework.
• Measurement Focus: Shift from clinician-reported outcomes (e.g., joint counts) to Patient-Reported Outcome Measures (PROMs) that reflect lived experience (e.g., stiffness duration, ability to function).
• Inclusion and Diversity: Significant challenge remains in ensuring engagement includes populations with high disease burden who lack access to trials, advocacy networks, or digital literacy.
• Regulatory Uncertainty: A primary barrier for sponsors; fears exist that patient input may expand the scope of required studies, though FDA guidance aims to increase certainty.

Challenges and Disagreements:

• Cultural Friction: Scientists and sponsors often struggle to shift from a paternalistic "expert knows best" mindset to a partnership model; timelines and grant deadlines often conflict with patient availability.
• Input Utilization: Sponsors face pressure to act on patient feedback; when requests are deemed infeasible, there is a risk of disengagement if transparency is lacking.
• Representativeness: Small focus groups in "patient-focused drug development" meetings struggle to provide generalizable data without robust scientific methodology for aggregation.
• Payer Alignment: Current clinical trials lack comparative effectiveness perspectives; patients and payers often lack visibility into alternative treatment options and affordability before trials conclude.
• FDA Implementation: Internal consistency at the FDA varies due to the organization's size and the need for a "cognitive revolution" among staff accustomed to traditional medical hierarchies.

Forward-Looking Statements and Future Directions:

• Standardization: FDA and industry are working to develop a core set of reusable patient-reported outcome measures to prevent "reinventing the wheel" for every disease area.
• Systemic Integration: The conversation is shifting toward "benefit-risk templates" that formally integrate patient experience data into FDA decision-making.
• Trial Design Evolution: Future research will move toward "master protocols" and "platform trials" focused on improving disease management rather than testing single-hypothesis interventions.
• Employee Morale: Pharmaceutical organizations report that patient engagement initiatives significantly boost employee morale by reconnecting scientists with their "north star" purpose.
• Global Influence: European health technology assessors (HTAs) increasingly require patient-centric data for reimbursement decisions, creating external incentives for US companies.

Specific Regulatory Examples Cited:

• Psoriasis/Atopic Dermatitis: FDA shifted approval stances significantly after patient meetings highlighted the devastating impact of these conditions on quality of life.
• Tysabri (Multiple Sclerosis): Patient advocacy input was critical in the decision to reintroduce the drug to the market after a previous recall due to a rare viral infection.
• CML Drug (Chronic Myelogenous Leukemia): FDA altered release mechanisms for a drug requiring immediate cessation to avoid death, citing patient feedback that the drug was essential despite vascular risks.