HHS Policy Shift Could Boost Role of Patient Advocates in Clinical Trials

PC: How do you see the potential impact of HHS's proposed policy change on patient advocacy efforts, particularly for rare and serious diseases?

Leuchter: If I step back and think about how critical it is to receive input, feedback, and partnership from patient advocates—their voice is truly essential—it’s hard to overstate. At Neurvati and GRIN, we’ve embraced that view since our inception in September 2021. Some of our earliest hires were individuals who’ve dedicated their careers to advocating for patients and making their voices heard in the drug development process.

Maintaining mechanisms that allow patient advocates to express their views and collaborate with all stakeholders in drug development is incredibly important. It’s not just about understanding the patient journey—it directly informs how we design clinical trials. Patient and caregiver insights shape everything from trial design and site selection to endpoint development and regulatory strategy. Their lived experiences help us create more thoughtful, patient-centric research from the ground up.

Full Interview Summary: The proposed policy change by the Department of Health and Human Services (HHS) that could reduce formal mechanisms for patient advocate input in regulatory discussions poses significant risks to drug development, especially for rare and serious diseases. Patient advocates are crucial in helping developers understand the lived experiences of patients and caregivers, especially in conditions with limited natural history data. Their insights inform decisions around clinical trial design, site and endpoint selection, and regulatory strategies. Organizations such as GRIN Therapeutics have prioritized patient advocacy from the outset, integrating advocates into their teams to ensure ongoing dialogue and collaboration throughout development.

Historically, public participation has been instrumental in shaping healthcare policy. For newly recognized or genetically defined diseases, patient advocates often provide the only frame of reference for symptom progression and caregiver burden. This input is invaluable not only to developers and regulators but also to payers making reimbursement decisions. High-cost, innovative therapies must demonstrate broad impact across disease symptoms to justify value, and advocates help articulate which symptoms matter most to patients. Their contributions help define clinical endpoints that better capture treatment effectiveness, supporting both regulatory approval and reimbursement.

If formal patient input mechanisms at the federal level were removed, drug developers would lose a vital perspective at a critical decision-making stage. While engagement can continue informally—through interviews, surveys, and other methods—losing the regulatory touchpoint would weaken the system. Nonetheless, developers can and should continue gathering robust patient and caregiver insights and proactively share them with regulators. In doing so, they help bridge any gaps left by policy changes and sustain meaningful engagement that ultimately benefits all stakeholders—patients, developers, payers, and regulators alike. This collaborative approach remains essential to ensuring the development of effective, impactful therapies for rare and serious conditions.