The LGDA is proud to have contributed to the recently published ROADMAP (Repurposing of All Drugs, Mapping All Paths) study, which provides a comprehensive overview of rare disease drug repurposing efforts in the United States. This study, published in JAMA Network Open, offers valuable insights into the strategies and challenges faced by rare disease nonprofit organizations (RDNPs) in their pursuit of effective treatments.
Key Findings from the ROADMAP Study
- Broad Participation: Out of 698 potential U.S.-based RDNPs, 147 participated in the survey, with 58 organizations actively supporting drug repurposing projects.
- Repurposing Success Rates: Among 94 drugs in various stages of repurposing, 23 achieved success criteria, including 5 obtaining FDA approval and 18 demonstrating off-label use with subjective benefits.
- Factors Influencing Success: The study identified that nonprofit-supported patient recruitment into trials and the provision of nonfinancial research support were significantly associated with successful outcomes.
- Five-Stage Framework: Interviews with RDNP leaders led to the development of a five-stage framework for drug repurposing: (1) enabling drug repurposing, (2) identifying a drug therapy, (3) validating a drug therapy, (4) clinical use and testing, and (5) reaching an optimal endpoint for clinical practice.
LGDA’s Contribution
The LGDA actively participated in the ROADMAP study by sharing the survey with its community and engaging in interviews. Our involvement provided valuable data and perspectives that enriched the study's findings. By contributing to this research, the LGDA continues its commitment to advancing treatment options for those affected by lymphangiomatosis and Gorham’s disease.
Moving Forward
The insights from the ROADMAP study underscore the critical role that RDNPs like the LGDA play in the drug repurposing landscape. By facilitating patient recruitment, providing research support, and sharing community experiences, organizations can significantly impact the development of therapies for rare diseases.
We encourage our community members, researchers, and stakeholders to read the full study to better understand the collaborative efforts driving progress in rare disease treatment.
Access the full study here: ROADMAP Study in JAMA Network Open
Together, we can continue to make strides in finding effective treatments for rare diseases.
