5 Key Takeaways

• 1

  The Orphan Drug Act incentivizes the development of therapies for rare diseases by providing faster FDA reviews and lower regulatory costs.

• 2

  The FDA's Rare Disease Evidence Principles aim to expedite the review of therapies for very rare genetic diseases affecting fewer than 1,000 patients.

• 3

  Ophthalmic conditions like thyroid eye disease and keratoconus are often underdiagnosed, leading to missed treatment opportunities for patients.

• 4

  Effective screening for rare ophthalmic diseases can be achieved with simple tools, such as corneal sensitivity tests, which are often overlooked.

• 5

  Despite the availability of treatments, many patients with rare eye diseases remain untreated due to a lack of clinician awareness and training.