Exploring the ethics of patient involvement in developing drugs for rare diseases

The Ethics of Engagement in Patient-Focused Drug Development for Rare Diseases

NIH-funded research Stanford University · NIH-11039662

This study looks at how patients and their families are helping to shape new treatments for rare diseases, while also exploring the tricky ethical questions that come up when they work with researchers and drug companies, all to make sure that everyone benefits fairly from these new therapies.

Quick facts

Grant typeR01 grant
Study typeNIH-funded research
Funding institutionStanford University NIH-funded
Lab location1 site (Stanford, United States)
Project IDNIH-11039662 on NIH RePORTER

What this research studies

This research investigates how patient and family groups are increasingly influencing the development of new therapies for rare diseases. It focuses on the ethical challenges that arise when these groups collaborate with academic institutions and drug developers. By examining the competing values and interests in patient-focused drug development, the research aims to create tools that can help manage these ethical dilemmas. The goal is to ensure that patient engagement leads to effective and equitable drug development processes.

Who could benefit from this research

Good fit: Ideal candidates for this research are individuals and families affected by rare diseases who are engaged or interested in the drug development process.

Not a fit: Patients with common diseases or those not involved in patient-focused drug development may not benefit from this research.

Why it matters

Potential benefit: If successful, this research could enhance the ethical framework surrounding patient involvement in drug development, leading to more effective therapies for rare diseases.

How similar studies have performed: While patient engagement in drug development is becoming more common, the specific ethical challenges in this context are still being explored, making this research both relevant and timely.

Where this research is happening

Stanford, United States

Researchers

About this research

  1. This is an active NIH-funded research project — typically early-stage science, not a clinical trial accepting patient enrollment.
  2. Some NIH-funded labs run parallel clinical studies or seek volunteers for related work. To check, contact the principal investigator or institution listed above.
  3. For full project details, budget, and progress reports, visit the official NIH RePORTER page below.
Last reviewed 2026-06-13 by the Find a Trial editorial team. Information on this page is for educational purposes and is not medical advice. Always consult qualified healthcare professionals about clinical trial participation.