Drug repurposing involves identifying FDA-approved drugs that may be effective for diseases they’re not currently approved for. We believe that this is a much more efficient process than new drug research and development for rare diseases like Castleman disease.
Here are the facts:
- Currently, there are ~2,500 FDA approved drugs that treat 25% of the ~10,000 human diseases
- No treatments exist for over 7,000 diseases, many of which are rare and deadly
- Developing a new drug can cost $2-3 billion an can take 13-15 years before impacting patients
- Many rare diseases are heterogeneous and/or idiopathic.
By definition, rare diseases do not affect a large patient population and often do not present enough of a commercial incentive for pharmaceutical companies to develop new treatments.
“Drug repurposing empowers the community affected by a rare disease to take action to find cures”
Given the insufficient interest for new drug development, drug repurposing provides a potential solution. The basic idea of drug repurposing is that many diseases share similar defects and thus may benefit from the same treatments. Once a treatment has been FDA-approved and shown to be safe and effective for at least one disease, it is more likely to be safe and potentially effective in a new disease area than new, untested compounds. Research is required to identify and match potential treatments to specific conditions. This approach is often much faster and cheaper than new drug development.
Thus, redirecting more funding and research towards maximizing the utility of existing treatments, rather than focusing solely on the expensive, time-consuming and risky development of novel drugs, is potentially one of the fastest and most effective ways to get treatments to patients with rare diseases.