Sustaining Regulatory Engagement Through Industry and Advocacy Collaboration

PC: In the absence of public participation at the federal level, how can industry leaders collaborate with advocacy groups to sustain meaningful engagement with regulators?

Leuchter: Experienced regulators are very attuned to how patients and caregivers live day to day with a given disease. That understanding is a key part of how they evaluate opportunities to improve patients' lives.

Whether or not there’s a formal mechanism for public dialogue, I believe regulators continue to value this perspective as they engage with drug developers. If you removed the official pathway for open dialogue but retained drug developers’ ability to engage with the patient community, I believe we could still channel that feedback to regulators in meaningful ways.

There are various mechanisms to do this. In our Phase I/II Honeycomb study, for example, we conducted interviews with caregivers to better understand their daily challenges. That qualitative input is crucial, and we’ve shared it with regulators.

The formal path for hearing from patient advocates is valuable and currently exists. But even if that were removed, developers would still have the tools and responsibility to gather this information, integrate it into trial design, and communicate it to regulators. This ongoing work can help mitigate any loss of a dedicated patient advocacy pathway, as long as multinational regulators continue to support these efforts.

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.