Slide 1: February 28. Rare Disease Day. Rare diseases are defined as a disease that affects 200,000 or fewer people. Some of the most rare diseases or disorders include mesothelioma, Lambert-Eaton myasthenia syndrome (LEMS), and progeria.

Slide 2: The Orphan Drug Act of 1983 incentivizes the development of drugs to treat rare diseases. Before this law, 7,000 rare diseases were identified but only 5% had a treatment. The Orphan Drug Act also includes: Orphan Product Grants Program to fund product development, tax credits for clinical testing, waivers for prescription drug user fees, and potential market exclusivity following approval.

Slide 3: Where are they now?

Progeria. In 2020, FDA approved the first treatment to reduce the risk of mortality from progeria.

Mesothelioma. Since its Phase III clinical trial in 2003, cisplatin-pemtrexed combination treatment became the standard of care treatment for mesothelioma.

LEMS. In 2018 Firdapse (amifarpridine) became the first FDA approved treatment for LEMS.

Today, there are over 560 treatments in development for various rare diseases.

References

  • “Rare Disease by the Numbers.” Innovation.org Link.
  • “The History of Mesothelioma.” Asbestos.com Link.
  • “FDA Approves Drug for Progeria, a Rare Disease Causing Rapid Aging in Children.” Ajmc.com Link.
  • “FDA Approves First Treatment for Lambert-Eaton Myasthenia Syndrome , a Rare Autoimmune Disorder.” FDA.gov Link.
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