Panel, Conference Presentation
System Thinking to Drive Patient Engagement: Walking the Talk
Milken InstituteSusan Schaeffer, Julie Gerberding, Ivor Horn, Kathy Hudson, Bray Patrick-Lake, Janet Woodcock, Arthur Allen, Syra
- Patient-centered R&D and active patient engagement are projected to improve product development, care delivery, and health outcomes by leveraging patient expertise to reduce waste of time, money, and resources, with general industry, regulator, and patient agreement on these goals.
- The FDA is expected to integrate patient perspectives into regulatory decisions following 2012 and 2017 PDUFA negotiations, convene workshops, issue guidances under the 21st Century Cures Act, and develop reusable core patient-reported outcome measures to avoid reinventing data collection methods.
- Biopharmaceutical companies and patient organizations are anticipated to advance by creating chief patient officer roles since 2014, funding and conducting research across basic studies to complex platform trials, and reshaping regulation through pathways like breakthrough therapy designations.
- Corporate priorities include co-creation of clinical trials, improving patient experience, and ensuring affordable access to drugs, while health assistant platforms like Accolade expect to apply engagement research to serve over 1.5 million people through longitudinal relationships.
- Research networks like PCORnet 2.0 aim to embed patient engagement strategies across systems caring for over 100 million patients, with a focus on validating measures that reflect the lived experience of populations often excluded from trials.
- Early and frequent patient engagement is predicted to shorten clinical cycle times, yield more meaningful endpoints, and improve net present value by affecting risk, cost, time, and revenue, though delays in engagement can prevent protocol changes due to high costs.
- Challenges to implementation include the difficulty of translating scientific norms to effective partnerships, the need for mutual education between researchers and patients, and fears regarding infeasible patient requests or regulatory uncertainty that could slow processes and increase expenses.
- Regulatory flexibility is expected to increase regarding patient experience data, while European payers and tech assessors will likely drive further adoption through payment decisions, though the transition toward a partnership model rather than a paternalistic one is anticipated to be patchy.
- Internal benefits include a potential increase in employee morale and alignment with corporate purpose, provided companies maintain transparency regarding input and explain why specific patient requests cannot be accommodated.
- Future discussions are expected to address roadblocks to systemic engagement, the development of objective data-driven methods for interpreting patient input, and the necessity of proving that engagement at every step accelerates the delivery of cures.