The Problem:

There are approximately 10,000 known diseases, but only 2,500 of them are addressed by FDA-approved drugs. That leaves around 7,500 diseases without FDA approved treatment, many of which are rare diseases, diseases in pregnant women, children and neonates, as well as neglected tropical diseases and emerging/reemerging infectious diseases. Many existing drugs might work to treat these diseases, but often there are no commercial or regulatory incentives – or there are actual disincentives — to do the extensive clinical testing required to show they are effective.

The Solution:

In response to these challenges, the CURE Drug Repurposing Collaboratory (CDRC) was founded to explore whether already marketed drugs can be repurposed for diseases that are not commercially attractive. CDRC is strongly interested in capturing data from diverse populations, including pediatric patients and pregnant women, as well as under-served populations.

As part of this process, a roadmap will be developed for the efficient generation of evidence, in order for FDA to update labeling. This includes creating a path of potential scenarios, depending upon the context of the specific indication, unmet patient needs, the engagement of the drug sponsor or the first NDA holder and the status of current legislation. The data gathered through the CURE ID app can be used to inform the design of clinical trials, to evaluate the effectiveness and safety of a marketed therapy intended for repurposing, or to support treatment guideline development.

The path chosen will depend on the drug(s) in question (patented vs generic), the disease epidemiology and natural history (availability and procurement of sufficient data), and the existing legislative mechanisms (sponsor, payer, and/or patient support). Although many of the challenges of drug repurposing are global, regulators need to address these locally and within the existing regulatory systems. As regulators globally pilot programs, an ability to provide lessons learned and potentially share information to harmonize processes will enable pharma and manufacturers to better align by reducing complexity, lowering costs and ensuring safe and effective treatments for all patients.

The Impact:

The Collaboratory, led by Critical Path Institute (C-Path), works in a transparent, open forum, with a diverse set of global stakeholders including, but not limited to, clinicians, scientists, U.S. Health and Human Services (HHS) agencies, non-government organizations, foundations and societies in order to:

• • Evaluate drug leads through advanced analytics to identify candidates for repurposing as new treatments
  • Inform the design of clinical trials of existing marketed drugs for new indications
  • Generate real-world evidence for expanding drug labels
  • Provide a regulatory roadmap to advance drug repurposing and expedite the availability of safe and efficacious treatments for diseases with limited or no treatment options
  • Promote the CURE ID platform to enable the global health community to openly share patient treatment outcomes