Celebrating the Orphan Drug Act’s 35th Anniversary: HP&M Attorneys Author Proposal for Building an FDA Rare Disease Center of Excellence in Advance of EveryLife Foundation Scientific Workshop

September 12, 2018By James E. Valentine & Frank J. Sasinowski

As we celebrate the 35th Anniversary of the Orphan Drug Act (see our 30th anniversary post here), periodic consideration of opportunities to reform and refine the approach to rare disease medical product regulation is warranted – similar to the review that occurred 10 years ago, which resulted in the establishment of the CDER Rare Diseases Program and first FDA public hearing on orphan drugs in June 2010.

In that spirit, Hyman, Phelps & McNamara, P.C.’s Frank Sasinowski (who serves as Vice Chair of the EveryLife Foundation Board of Directors) and James Valentine co-authored a proposal for building an FDA Rare Disease Center of Excellence (COE). The proposal will serve as a discussion document for the upcoming EveryLife Foundation 10th Annual Scientific Workshop. The proposal comes on the heals of the 21st Century Cures Act which provides FDA authority to establish COEs, as well as recent successes with the first COE in oncology.

The onus for this proposal, as well as the Scientific Workshop, is that because of the Orphan Drug Act, which provides incentives to make developing drugs for small numbers of patients financially viable, there has been increased investment in the research and development of medical products (drugs, biologics, and medical devices) to prevent and treat rare diseases, also known as orphan conditions. However, this influx has created unique regulatory challenges for FDA in providing regulatory oversight and in conducting review of marketing applications given that the development of products for these conditions present many unique challenges. There is no separate, lower or lesser legal or regulatory standard for approval of orphan products, so researchers and product developers and FDA alike must confront these issues throughout all phases of development and employ creative approaches to product development and review must be employed.

Given the unique challenges and, therefore, the unique expertise needed to advance the development and review of products for rare diseases, a Rare Disease COE would provide the necessary infrastructure to allow centers and offices across FDA to consistently and efficiently review novel products for these conditions. The proposed Rare Disease COE would involve a combination of three overarching organizational changes at FDA:

  1. Establishment of a COE organizational unit within the Office of Medical Products and Tobacco with cross-Center responsibilities
  2. Establishment of a Deputy Director for Rare Diseases within each review office/division across CDER, CBER, and CDRH
  3. Establishment of a Rare Disease Advisory Committee

For more information on the details on the structure, function, and regulatory responsibilities of this Rare Disease COE, you can view the proposal here.

The EveryLife Foundation’s Scientific Workshop, which takes place this Thursday, September 13th from 8:30 am until 4:00 pm has a robust agenda that brings together regulators, patient advocates, academia, industry, and other stakeholders to discuss both progress and continued challenges in the development and review of medical products for rare diseases. The day will culminate with a presentation by Mr. Sasinowski on this proposal for a Rare Disease COE, which will be followed by a panel discussion consisting of:

  • Rich Moscicki, MD, Executive Vice President, PhRMA
  • Paul Melmeyer, Director of Federal Policy, NORD
  • Lucas Kempf, MD, Associate Director Rare Diseases Program, FDA
  • Celia Witten, MD, PhD, Deputy Director, FDA CBER
  • Alan Beggs, PhD, Director, The Manton Center for Orphan Disease Research

The workshop agenda can be found here, and you can register for the webcast is available here.

We hope this proposal and the workshop will initiate a dialogue about such possibilities to jumpstart brainstorming that may result in increased visibility for rare disease therapies and other related developments (e.g., a practical way to enhance and augment the prominence of surrogates and Accelerated Approval).