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The First FDA Rare Disease Patient Advocacy Day

Posted by | 10:13am on Monday, March 12, 2012

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. 

We are pleased to feature a guest blog post from Catherine Sohn. She has more than 20 years of pharmaceutical marketing and strategic product development experience with one of the world’s largest companies and is currently an independent consultant working with companies focused on life science and rare disease.

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.

About Wendy White

Since founding Siren Interactive in 1999, Wendy has been recognized as a thought leader at the intersection of niche pharma brands, patient empowerment and online marketing. Her vision for how the internet can facilitate interactions and provide crucial information that patients, caregivers and their healthcare providers previously struggled to find has propelled Siren to the forefront of relationship marketing for rare disorder therapies.

View other posts from Wendy

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  • http://twitter.com/MichaelDayton3 Michael Dayton

    As an advocate for rare diseases, as well as having a rare disease myself, I would like to pass along some helpful information about an interesting company, The Rare Genomics Institute. The Rare Genomics Institute is a non-profit organization that focuses on helping patients and families with rare/orphan diseases that might benefit from genome sequencing. Rare Genomics Institute helps patients with gaining access to genome sequencing services, support, and funding. Washington University School of Medicine’s Genomics and Pathology Services and the Rare Genomics Institute are planning to award grants for the sequencing of 99 exomes to rare disease advocacy groups. The grants will be free of charge to the rare diseases community! Anyone interested, should contact The Rare Genomics Institute right away. Interested applicants should submit letters of interest by April 2, 2012. If you are interested, visit the web site for this program at the Rare Genomics Institute site: http://www.raregenomics.org/rare99x
    Mike Dayton

Siren Interactive
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