The first FDA #RareDisease patient advocacy day

In Uncategorized by Caroline Hornby

rare disease patients and fda Siren Interactive kindly provided this guest blog post from their blog, SirenSong. It was written by Catherine Angell Sohn, Pharm.D. Dean’s Professor, University of the Sciences in Philadelphia and President, Sohn Health Strategies, LLC.

Having a personal passion for furthering the cause of bringing new medicines for rare diseases to the marketplace, there was no doubt that I would be in Silver Springs, MD on March 1 to attend the inaugural FDA Rare Disease Patient Advocacy Day. According to Gayatri R. Rao, MD, JD, acting director of the FDA’s Office of Orphan Products Development, the event was designed to create a dialogue among all stakeholders involved in rare disease product development, including the FDA, patients, healthcare practitioners, the NIH, patient advocates and the pharmaceutical industry.

Another event objective was to support solidarity within the field of rare diseases by engaging with the patient advocacy community and providing updates on the FDA’s role in product development. To highlight the FDA’s effort to help patients and caregivers learn how to become advocates for advancement in treatments, she described the little-known FDA Patient Representative Program, in which patients work with the FDA and sponsors throughout the product development process.

Participation by FDA Commissioner, Dr. Margaret Hamburg, and Deputy Commissioner, Dr. Stephen Spielberg, demonstrated the growing support for rare diseases within the FDA. Dr. Rao did an amazing job creating and managing the event. I was impressed with the fact that all branches of the FDA and the NIH participated, and that the day’s activities were also shared through a webcast to reach those patients and healthcare practitioners who were unable to attend. That exemplified the tone of openness and inclusiveness that was so pervasive in so many aspects of the event.

Communication, Collaboration and Engagement

As I reflect back on the sessions that I attended throughout the day, I find that there was a recurring theme with the convergence of communication, collaboration and engagement. Speaker after speaker emphasized that patient communication and collaboration are critical during the development process and that engagement is mandatory to help define the end points that are meaningful to patients.

It’s so important to have the input of a patient representative as part of the entire process—from the investigational new drug application (IND) to the new drug application (NDA). The FDA gains valuable perspective and input on the patient’s perception of the burden of their disease and their potential tolerance for risk and side effects of new medicines, especially when the disease is life-threatening. In some cases, it’s resulted in the addition of patient-specified parameters as endpoints to the standard criteria for response. This acknowledgement of a therapy’s effect on a patient’s quality of life is a much-needed departure from the traditional disease endpoints for the evaluation, and it enriches the data collected during clinical trials.

Patient advocacy groups have been better able to understand the process either by working with the FDA as an FDA Patient Representative or by working with a sponsor as a consultant on a clinical trial. Understandably, it’s not permitted for the same group to serve in both roles at the same time.

The FDA takes pride in their success working with patient advocates, sponsors and healthcare professionals, which has resulted in a significant increase in the number of FDA drug approvals since the Orphan Drug Act was enacted in 1983. Part of their approach includes hosting forums for roundtable discussions with rare disease patients, caregivers, healthcare practitioners, industry and academia to address the barriers to getting rare disease products to patients. Dr. Rao encouraged sponsors and patient advocacy groups to engage with her department at the FDA, and she noted that there is no formal process for initiating the dialogue. It’s possible to request a meeting with an open agenda.

In the past, companies have been able to benefit from the meeting by:

  • Sharing information on their drug pipeline
  • Gaining informal feedback and perspective
  • Establishing awareness for the rare diseases they are studying
  • Learning about advocacy groups that are interacting with the FDA

The mantra of communication, collaboration and engagement for Rare Disease Patient Advocacy Day appears to be gaining traction beyond this first-ever event, which may be due to the FDA’s endorsement of pharmaceutical companies working more with patient advocacy groups.

If you also attended the event, please comment below and share your thoughts.

For more information on orphan drugs and rare diseases, check out the World Orphan Drug Congress USA. Siren Interactive is a silver sponsor.