by Michael Firgens
Drug discovery and the translation of research findings into new therapies require significant investments and resources by biopharmaceutical companies. Analyses across all therapeutic areas indicate that the development of a new medicine, from target identification through marketing authorization, takes over 12 years and often much longer (DiMasi 2010).
An attractive alternate development strategy is the repurposing of known drugs. The basic idea works as follows: Marketed drugs are approved for certain uses and have been tested in humans, so detailed information is available on their pharmacology, formulation and potential toxicity. The repurposing builds on these available data, previous research and development efforts, which facilitate the identification, design and development of new therapies and can speed up the review by agencies and, if approved, the integration of the product into the healthcare system.
A comparison between conventional development strategies (i.e. for novel drugs and generics) and the repurposing development approach is presented in Figure 1. A more detailed view on the development process for novel and repurposed drugs is shown in Figure 2. In the literature, the estimated benefits of the repurposing development strategy slightly differs depending on the product type and target indication, however, it is obvious that drug repurposing is less risky, cheaper and faster than the development of novel drugs.
Repurposing of established drugs can be defined as “studying drugs that are already approved to treat one disease or condition to see if they are safe and effective for treating other diseases” (National Center for Advancing Translational Sciences).
All repurposing programs contain two key elements:
- taking existing scientific or medical knowledge and technology that is “approved” for human use in one disease or condition; and
- applying this knowledge and technology to another disease or condition.
Table 1 summarizes various examples of repurposed drugs for small molecules and biologicals. The opportunities of drug repurposing have been also discovered for the creation of totally new treatment regimens, e.g. combination therapies in oncology (Figure 3) (Bertolini 2015).
The concept of drug repurposing is known to regulators, and applicable regulatory frameworks have been defined in the EU and the US (Table 2). As for the development and registration of new molecule entities (NMEs), similar incentives exist for repurposed drugs, e.g. for orphan drugs or pediatric development programs. Combining the advantages of the drug repurposing approach with a sound development and regulatory strategy (avoiding pitfalls) provides enormous opportunities for biopharmaceutical companies.
Biopharma Excellence is experienced in the generation of tailor-made integrated development strategies from development to approval. If you wish to learn more how to successfully implement a repurposing strategy for your development program, please contact us.
- Ashburn, T. T., & Thor, K. B. (2004). Drug repositioning: identifying and developing new uses for existing drugs. Nature Reviews Drug Discovery, 3(8), 673–683.
- Bertolini, F., Sukhatme, V. P., & Bouche, G. (2015). Drug repurposing in oncology—patient
- DiMasi, J. A., Feldman, L., Seckler, A., & Wilson, A. (2010). Trends in Risks Associated With New Drug Development: Success Rates for Investigational Drugs. Clinical Pharmacology & Therapeutics, 87(3), 272–277.
- Papakrivos, J. (2011). Regulatory Strategy for the Development of Known Drugs in New Therapeutic Areas.