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Race to Cure Rare Diseases

Last year, Scientist announced our winners for the Rare Disease Challenge, as part of our efforts to raise awareness for rare diseases. We know how alone many rare disease patients may feel, especially when big pharma may not seem like they are focusing on these rare diseases. Although each rare disease may only affect a small population, all together they affect 30 million Americans, or nearly 1 in 10 people.1 Additionally, most of the drugs currently being released or in clinical trials are targeted towards common diseases. Clearly there is an important market for the opportunity for new drug discovery!

Nearly one in 10 Americans are affected by rare diseases.1

However in recent years, there has been a rise in the research of rare diseases. This is likely due to the increased awareness through patient advocacy groups and through the increase in social media attention.2 This has resulted in more rare disease research in both academic institutions and in industry. While many common diseases research may have a lot of competition, rare disease researchers are able to focus on their research without fear of getting scooped. The downside to this is often the feeling of isolation in academic circles without the benefit of possible collaborators that can add to the research of a particular disease. Another downside is the struggle with securing funding in this research space, as many federally funded grants are typically given to broad impact disease areas. Despite this, researchers have been able to secure foundation grants and design their federal grant applications to explore research that aids not only the rare disease but also draws parallels to a common disease.

Additionally, because of the pharmaceutical innovation crisis, biotech companies are realizing that they can successfully cash in on the orphan drug designation. Genzyme is one famous biotech company that has been able to demonstrate how researching rare diseases can be profitable. Some other companies following in Genzyme’s footsteps include Actelion Pharmaceuticals, BioMarin, and Alexion Pharmaceuticals. These companies have directed R&D funding towards rare disease therapies, showing that rare disease research not only can potentially make a profit but it doesn’t need to be limited to academic research.

In fact, with the variety of rare diseases resulting in a large number of potential therapies and drugs waiting to be discovered, the start-up potential is ripe for rare disease research. The rise of CROs can only help the various stages of the development of rare disease therapies, from high throughput screening to clinical research. While rare disease research still has its challenges, the increase in awareness and resources has increased innovation in this area. Hopefully biotech companies can use such resources like Scientist and make an impact in the cures for rare diseases!

  2. Ledford H. Disease Research: Rare insights. Nature 505, 443-445 (2014). doi:10.1038/nj7483-443a