In the United States, rare diseases (RDs) are statutorily defined as conditions that impact 200,000 individuals or less. Currently, more than 30 million Americans are affected by a rare disease. At the same time, about 5% of rare diseases have a Food and Drug Administration (FDA)-approved treatment and less than 15% have at least one drug either in clinical development or that has demonstrated potential in treatment, diagnosis or prevention. In recent years, an emerging approach called “drug repurposing” has shown promise in accelerating the search for treatments targeting RDs. By repurposing existing drugs, researchers and manufacturers can save time, reduce costs, and potentially help patients with RDs gain access to therapies.

Understanding Drug Repurposing

Drug repurposing, also known as “drug repositioning” or “drug reprofiling,” involves finding new therapeutic uses for existing drugs outside of the scope of their originally intended or FDA-approved indication. Unlike “off label” use, repurposing of drugs involves seeking FDA approval for the additional indication.

Development of drugs in the RD category is often challenging due to multiple factors, such as small patient populations, difficulties in patient identification, disease heterogeneity, limited understanding of pathophysiology, and high drug development costs. Drug repurposing provides scientists, developers, and manufacturers with an opportunity to reduce time and costs of clinical development.

Through collaboration between academia, pharmaceutical manufacturers, regulatory bodies, and patient advocacy groups, drug repurposing can pool resources, and data to expedite the drug development process and offer additional treatment options to patients.

Stakeholders repurposing drugs for RD treatment have the following considerations when evaluating new uses for generics, brand-name drugs, and shelved compounds:

While drug repurposing has its benefits, it is not without challenges.

Figure 1. Headwinds and Tailwinds of Drug Repurposing in Rare Diseases

Deeper Dive

Drug repurposing offers potential new treatment pathways for patients with, although there are considerable barriers to implantation that stakeholders should be mindful of. By leveraging the knowledge, safety profiles, and regulatory pathways of already approved or investigational drugs, researchers may bypass some hurdles in the drug development process. While challenges exist, the continued exploration of drug repurposing in rare diseases holds significant potential to improve the lives of patients with RDs.

Avalere applies our expertise in portfolio repositioning and evidence generation planning to help stakeholders meet their business objectives through effective commercialization.  To learn more about addressing unmet needs in RDs through drug repurposing, connect with us.