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P217: Analysis of Orphan Drugs (ODs) in the 21st Century by Type of Orphan, Approval Times and Facilitated Review Pathways (FRPs)

Poster Presenter

      Nicole Damour

      • Director
      • Sarepta Therapeutics
        United States


Evaluate approval timelines for ODs approved to treat a disease linked to a genetic mutation (GM) in context of all OD approvals and how approval timelines are impacted by FDA’s Facilitated Regulatory Pathways (FRPs).


Applications for 30 ODs submitted to FDA for 2011-2020 to treat a disease linked to a GM (OD-GM) were identified to evaluate the impact of FRPs. Analyses in this project focus on the initial approval of the listed drug.


Since 2011, 31 applications have been approved by FDA to treat rare diseases linked to a GM (30 drugs analyzed based on available data). All drugs that comprise this data set utilized at least one FRP during development or approval. Over the 20-year period in which INDs were opened (1998 to 2018) there are distinct trends that accelerate development and approval timelines. During this time, Phase1 clinical trials became more targeted to mutation-specific disease states. Over the 10-year period of NDA/BLA submissions (2011-2020), the mean approval time shortened slightly, but the variance in approval timeline narrowed by almost half.


In the past 10 years, the development of ODs has become increasingly precise in terms of their therapeutic target. There is increasing specificity in the design of clinical trials for rare disease patients with an identified genetic mutation. OD-GMs are increasing in diversity, from an early and overwhelming emphasis on oncology, to a wider range of therapeutic areas. Development and approval timelines for OD-GM are rapidly accelerating, due to the regulatory benefits linking the OD designation to the frequent use of FRPs, aligning sponsor and FDA objectives. FRPs accelerate development and approval timelines, with Breakthrough Therapy Designation and Priority Review appearing particularly effective. Every FRP offers an advantage to the sponsor since they increase the level of engagement with FDA, accelerate development or accelerate the approval process. Three of the four available FRPs (Fast Track, Breakthrough Therapy Designation, and Accelerated Approval) are assigned during development, fostering collaboration between sponsors and FDA. This mutual benefit clearly brings efficiency to clinical development. Priority Review, granted when the NDA or BLA is submitted to the FDA, generally effective at driving FDA to make an approval decision within 6 months.