Around 7,000 orphan diseases have been described by physicians, since the days of yore through the 19th century. With more knowledge in biology and genetics, their classification has dramatically improved, even in recent years. For a long period of time, given the size of the population, investments from the industry were scarce with limited repurposing of drugs. Under pressure from patients, caregivers, and families, the economic and political landscape has undergone drastic change in the last 25 years. The FDA expanded patent protection for Orphan Drug Designations (ODD) to 7 years post-approval (Orphan Drug Act 1983). Similarly, the Japanese and Chinese drug authorization bodies and EMA incentivized ODD (The Orphan Regulation 1999, The Act on Securing Quality, Efficacy and Safety of Pharmaceuticals, Medical Devices, Regenerative and Cellular Therapy Products, Gene Therapy Products, and Cosmetics 1960, and the Rare Disease List, respectively). France established its first Plan Alliance Maladies Rares in year 2005 and was the instigator for Orphanet in 1997. These changes in policies driven by patient advocates fueled many applications with better economic rewards for promoters. Nowadays, in some indications, the competition is as intense as it is for very prevalent conditions. As a result, differentiation and cure at sight are now more of an expectation in a commoditized market where ODD alone are not enough to gain patient access or reimbursement.
We believe that in this current ocean of high unmet needs, there are still many opportunities for the industry and patients.