Ethics of patient and family partnerships in rare disease drug development
Official title: The Ethics of Engagement in Patient-Focused Drug Development for Rare Diseases
Funded at Stanford University · NIH-11269163 · Data updated 2026-11-12
This project is learning how patient and family groups can work with researchers, drug developers, and regulators in rare diseases so those partnerships are fair, trustworthy, and helpful.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Stanford University NIH-funded |
| Lab location | 1 site (Stanford, United States) |
| Project start | February 1, 2025 |
| Project end | January 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $610,309 |
| NIH project number | 5R01HG014059-02 |
| Project ID | NIH-11269163 on NIH RePORTER |
Who could benefit from this research
✓Could help
Ideal participants include people with rare diseases, family members, leaders of patient and family groups, and professionals who have taken part in rare disease drug development efforts.
✕Less likely to help
People with conditions unrelated to rare diseases or those not willing to share their experiences with research partnerships are unlikely to receive direct benefit from participating.
What this research studies
As someone affected by a rare condition, this project asks patients, family groups, researchers, industry representatives, and regulators about their experiences and concerns in collaborative drug development. The team will conduct interviews, hold collaborative workshops, and review policies and case studies to map ethical challenges that arise in patient-focused drug development. Researchers will co-create practical tools and guidance with community partners to help manage conflicts of interest, data sharing, and expectations across the development pipeline. The aim is to make partnerships safer, more transparent, and more effective for patients and families.
Why it matters
Potential benefit: If successful, this work could strengthen trust and reduce ethical problems in patient-led drug development so rare disease communities can more effectively advance therapies.
How similar studies have gone: Previous ethics and engagement projects have produced useful guidance on research partnerships, but a focused, co-created approach tailored to rare disease patient-led development is relatively new.
Where this research is happening
-
Stanford University
Stanford, United States Active
Researchers
- Principal investigator: Halley, Meghan — Stanford University
- Study coordinator: Halley, Meghan
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About this research
- This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
- Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
- For full project details, budget, and progress reports, visit the official NIH RePORTER page.