Ethics of Engaging Patient Groups in Drug Development for Rare Diseases
Official title: The Ethics of Engagement in Patient-Focused Drug Development for Rare Diseases
Funded at Medical College of Wisconsin · NIH-11530731 · Data updated 2026-11-12
This grant asks how patient and family groups can ethically help shape rare-disease drug development from idea to market, with input from patients, families, doctors, and researchers.
Quick facts
| Grant type | R01 grant |
|---|---|
| Study type | NIH-funded research |
| Funding institution | Medical College of Wisconsin NIH-funded |
| Lab location | 1 site (Milwaukee, United States) |
| Project start | February 1, 2025 |
| Project end | January 31, 2029 |
| Funding year | 2026 |
| Award (this year) | $319,927 |
| NIH project number | 7R01HG014059-03 |
| Project ID | NIH-11530731 on NIH RePORTER |
Who could benefit from this research
✓Could help
Patients with a rare disease and their families who participate in PFDD discussions or are part of patient groups partnering with researchers.
✕Less likely to help
General patients not involved in PFDD partnerships may not directly benefit from these specific tools and guidelines.
What this research studies
This project looks at the ethical challenges that arise when patient-focused drug development involves patients and families in collaboration with researchers and industry. It uses collaborative methods to identify concerns, values, and expectations, and to create practical tools to manage these issues. The aim is to make PFDD partnerships more trustworthy and effective so that rare-disease therapies progress more smoothly to patients who need them.
Why it matters
Potential benefit: If successful, the work could provide practical guidelines and tools to ethically manage PFDD collaborations, potentially speeding access to safe, patient-centered therapies.
How similar studies have gone: Previous PFDD work has shown that stakeholder collaboration can drive research, though ethical challenges remain a common to-be-addressed area.
Where this research is happening
-
Medical College of Wisconsin
Milwaukee, United States Active
Researchers
- Principal investigator: Halley, Meghan — Medical College of Wisconsin
- Study coordinator: Halley, Meghan
Related research
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Encore: speeding up finding new uses for existing drugs to help kids with rare diseases
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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.