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HDSA Research Webinars: The FDA and Patient Engagement— What’s Next for the HD Community?

May 12th, 2016

Presenters

Dr. John Whyte, MD, MPH — Director of Professional Affairs and Stakeholder Engagement at the Federal Drug Administration (FDA)

Laurie Haughey — Health Communications Specialist, Professional Affairs and Stakeholder Engagement at the Federal Drug Administration (FDA)

Summary

The Federal Drug Administration (FDA) is a public health organization that evaluates the safety, efficacy, and security of human and veterinary drugs, biological products, medical devices, food products, cosmetics, and potentially toxic products. In line with this mission, the FDA regulates clinical trials and drug approval. There is currently no cure and limited treatments available for those with Huntington’s disease. As such, the FDA has a consistent presence in the HD community as researchers develop and test potential treatments that are subject to FDA regulation.

The central mission of the Department of Professional Affairs and Stakeholder Engagement at the FDA is to create a culture of engagement between the FDA, patients, advocates, and healthcare professionals. They recognize the challenge of navigating an organization the size of the FDA and hope to promote a culture of communication based on dialogue with stakeholders in addition to dissemination of information.

Towards a goal of true conversation, there are opportunities available for stakeholders to engage with the FDA, including but not limited to: participating in patient-focused drug development meetings, joining an advisory committee as a patient representative or public speaker, encouraging HD experts to work as FDA reviewers, organizing citizens petitions, and generating direct outreach via emails and letters.

However, limitations to engagement are often difficult to overcome and it is valuable for the HD community to be aware of inherent restrictions to information sharing. For example, although the FDA hopes to be as transparent as possible, legal restrictions around confidentiality for knowledge, opinions, identities, and sponsorship during drug development and review may prevent full disclosure. This significantly restricts the FDA’s ability to discuss specific information about a product or research study under review. In addition, the FDA must be consistent in its approaches so as to not show bias to any one company or organization, and therefore often must default to presentation of only the scientific facts.

The presentation closed with a reiteration of the values of the FDA as well as the department of Professional Affairs and Stakeholder Engagement and that when it comes to drug development, patient interests are at the heart of what the organization does. In addition, the presenters expressed a desire for the HD community to know that the FDA does not want to slow research or drug development progress, they in fact want to say yes to new treatment options. However, to protect patients’ and stakeholders’ well being, they must be confident that a certain drug’s benefits outweigh its risks before doing so. For further questions or a desire to become involved, the presenters provided their contact information and encouraged listeners to reach out to them.

Contact Information

Dr. John Whyte MD, MPH — Director of Professional Affairs and Stakeholder             Engagement at the Federal Drug Administration (FDA)

Email: John.Whyte@fda.hhs.gov

Phone: 301-796-7600

Laurie Haughey — Health Communications Specialist, Professional Affairs and Stakeholder Engagement at the Federal Drug Administration (FDA

Email: Laurie.Haughey@fda.hhs.gov

Phone: 240-402-6511

HDSA Research Webinars can be accessed here.