By Anne McNickle, VP – Research & Communications, Catalyst

Can you believe 2023 is the 40th anniversary of the Orphan Drug Act (ODA)? That is some time lapse! In this blog, we want to highlight a few of the events we think will be special. As you likely know, President Reagan signed the ODA into law on Jan. 4, 1983, thus establishing the development incentives that created the orphan drug industry. In the 40 years since that time, the rare disease treatment landscape has undergone a massive change.

The ODA “completely changed the face of therapeutics for rare disorders,” FDA notes.

For example, since enactment of ODA, the number of orphan drugs approved by FDA has increased by over 1,500% – from just 38 to more than 600 treatments, according to the Rare Disease Company Coalition. For the past 3 years, products for rare diseases have accounted for just over half of the novel drugs approved by CDER; most recently, in 2022 they accounted for 54%. And the number of orphan drug designations (ODDs) granted by FDA “nearly tripled between the decades of the 2000s and 2010s” according to a 2022 paper by FDA’s Kathleen Miller, Office of Orphan Products Development, et al.

Rare Disease Week, which runs Feb. 27 – March 3, 2023, encompasses a broad range of activities spanning federal agencies, Capitol Hill and advocacy organizations. This year some of the key events include:

  • FDA – Feb. 27: FDA’s Rare Disease Day will feature speakers including Commissioner Robert Califf, Office of Orphan Products Development head Sandra Retzky, Principle Deputy Commissioner Janet Woodcock and many others. FDA’s theme for the meeting is “Intersections with Rare diseases—a Patient Focused Event” and will cover topics such as “clinical trial challenges with small patient populations focusing on children and improving diversity” and FDA initiatives to advance product development for rare diseases.
  • NIH – Feb. 28: NIH’s Rare Disease Day seeks to raise awareness about rare diseases, the people affected, and NIH collaborations that address scientific challenges and advance research for new treatments.
  • Capitol Hill/Everylife Foundation – March 1-2: Events on the Hill will include several hosted by EveryLife Foundation’s Rare Disease Legislative Advocates, for example:
    • A briefing for the Rare Disease Congressional Caucus
    • Meetings with individual Members – Rare disease advocates from across the US will advocate for policy changes directly to their Members of Congress

In addition, NORD will celebrate rare disease day Feb. 28 as part of an international effort coordinated by EURORDIS, a coalition of rare disease organizations across Europe, to raise awareness and spur change for the 300 million people worldwide living with a rare disease.

To all of our clients, allies and mentors who focus on meeting the needs of the rare disease community, and you know who you are, the Catalyst Healthcare Consulting team truly appreciates the work that you do and your dedication.  Thank you!