Rare Diseases Remain Underserved, FDA & Aleon Increase Focus on Orphan Drug Designations

January 2023

Rare diseases are defined in the US as diseases or conditions that affect fewer than 200,000 individuals. The relatively small patient population has historically meant therapies are harder to come by. Pharmaceutical companies were focused on more prevalent diseases given the larger number of patients in need, and trials for rare diseases were more costly to run, even finding patients was significantly more difficult. The FDA has taken great efforts to correct this problem as roughly 7,000 rare diseases affect more than 30 million people throughout the US. One major step the FDA took was the introduction of the Orphan Drug Designation program following the Orphan Drug act of 1983, a step quickly replicated by countries around the world. The policies are different in each country, but generally they provide incentives (tax breaks, exclusive marketing rights, etc. The US Marketing Application fee can be waived which currently costs more than $3 million USD) for companies to develop therapies targeting rare diseases, and since then the number of new therapies for rare diseases has increased dramatically. From 1967 to 1983 the FDA approved 34 drugs that would have qualified for the Orphan Drug Program. From 1983 to 2020 the FDA has granted more than 4,700 Orphan Drug Designations and approved more than 700 new treatments. Last year alone 26 of CDER’s 50 new drug approvals were for rare diseases. https://www.fda.gov/news-events/fda-voices/cder-continues-make-rare-diseases-priority-drug-approvals-and-programming-speed-therapeutic. However, many rare diseases are still lacking treatment. The roughly 700 approved therapies mean that 90% of the rare diseases in the US still do not have approved treatments. It is therefore no surprise that CDER is increasing focus on rare disease treatments with the introduction of the Rare Disease Cures Accelerator-Data and Analytics Platform in 2018, the Standard Core Clinical Outcome Assessment Grant Program in 2019, CDER’s Accelerating Rare Disease Cures Program and the Action Plan for Neurodegenerative Disease including Amyotrophic Lateral Sclerosis (ALS) in 2022, and the upcoming Rare Disease Endpoint Advancement Pilot program in 2023. CDER and the FDA are committed to seeing more therapies for rare diseases in the coming years, and we at Aleon Pharma International are dedicated to supporting sponsors seeking Orphan Drug Designations. Aleon provides regulatory strategy during the process with up-to-date regulatory affairs knowledge and experience demonstrated by years of professional relationships with health authorities. Following best practices based on experience and past FDA feedback, Aleon develops a thorough internal review checklist to capture key items during the request preparation and review periods to ensure high-quality submissions. As a result, our sponsors gain approval even in challenging situations like Orphan subset or early stage development with only in vitro data available. Supporting sponsors through an Orphan Drug Designation application is essential to getting these treatments into the hands of patients as soon as possible. Aleon is proud to have helped with more than twenty successful ODD designations in the US, and in 2022 we are hard at work with sponsors on more designations in the coming months. If you are considering an Orphan Drug Designation request to FDA, please consult us today.