Research Projects
roadmap project



Earlier this year, the Castleman Disease Collaborative Network reached out to the SSRA with a request to participate in a project meant to develop a well thought out plan rare disease organizations could implement to resolve the roadblocks patient organizations face in trying to gain approval for repurposing of drugs currently on the market for therapeutic use in unrelated conditions.

One major problem encountered by small rare disease population groups is the FDA requirement of a double-blind study. Too often, there are not enough participants to produce the data required. This is a problem superficial siderosis research faces with deferiprone. Until a double-blind clinical trial is completed, the insurance coverage will continue to be problematic. While we may have more than enough volunteers to participate in a clinical study, no one wants to be in a placebo group. FDA regulations need to be sensitive to the needs of small patient populations.

About the Project

Over 7000 rare diseases and disorders affect approximately 30,000,000 individuals in the U.S.A., and 95% of these rare diseases do not have a single Food and Drug Administration-approved therapy. Since the incentives for new drug development for rare diseases are limited, drug repurposing provides a promising way to identify effective treatments for rare diseases faster and cheaper than developing new drugs. Still, many challenges exist in the drug repurposing space, namely:

  1. lack of a consensus of the roles that various stakeholders play (patient advocacy organizations, researchers, physicians, government, pharmaceutical companies, etc.)
  2. lack of a “roadmap” of how rare disease organizations should go about pursuing drug repurposing,
  3. lack of support (data, finances, resources, guidelines, etc.) during the drug repurposing process.

The Repurposing Of All Drugs, Mapping All Paths (ROADMAP) project proposes to fill some of these knowledge gaps through document analysis, surveys, and interviews grounded in a participatory design approach. Ultimately, this project aims to answer some fundamental questions about the experience of drug repurposing for various stakeholders and to design a solution to some of the challenges rare disease organizations are facing through the creation of an interactive tool.

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