HD Lighthouse Contributing Editor's Comment:
--
Posted to the HDL: 10 Mar 2006

Marsha Miller, Ph.D.

Steve Ireland and I are attending the eighth annual conference of the American Society of Experimental Neurotherapeutics in Washington, D.C. from March 9-11. I learned about this organization when I interviewed Dr. Kathleen Clarence-Smith who is the president of Prestwick Pharmaceuticals and also the president of ASENT.

This was the first year that HD families attended. In addition to Steve and me, Nancy Wexler from the HDF and Tim O’Neil, Lighthouse regular, attended. Two representatives from HD organizations also attended - Deb Lovecky from the HDSA and Carl Johnson of the Hereditary Disease Foundation.

Fifty people were expected but over a hundred were in attendance. We were welcomed by a delightful Dr. Clarence-Smith who stressed the importance of patient/family advocacy and predicted that we would look back at this gathering as history in the making.

The first speaker was Russell Katz, MD, who is the Director of the division of neuropharmacological drug products at the FDA. The title of his talk was “Role of Patients in Drug Development: An FDA perspective.” Office of Special Health Issues (OSHI) was established in1988 in the office of the commissioner. Its activities are to...

• Represent Patient concerns
• Conduct outreach and education
• Administer patient representative and consultants programs

He spoke about the Patient Representative and Patient Consultants programs. The Patient Representative Program...

• provides for patient representative at public advisory committee meetings.
• must be someone with direct connection to specific disease under consideration (either someone with the disease or a family member).
• must be someone with appropriate skills/background. Candidates are identified through contacts with patient advocacy groups or self-identification.

The Patient Consultant Program was established in 2001 to fill a need that was identified by OSHI/Oncology division. It has been recently expanded to include Parkinson’s Disease. The program involves extensive educational efforts by OSHI with yearly conferences and at least monthly phone calls.

Typically the patient consultant is involved at end of phase 2 meetings with sponsors. They are given complete access to relevant materials and asked for input on many issues, including informed consent, enrollment, retention, outcome measures. – patients can help shape the outcome expectations of the study.

In both the Patient Representative and the Patient Consultant programs, the individuals are considered special government employees which means that they are bound by confidentiality agreements and also must be cleared for conflicts of interest which takes some time and slows down the process. An alternative being considered is to have meetings to address generic issues. Another issues of concern are whether the representatives/consultants chosen are truly representative of the needs, concerns, and views of the patient population. A third concern is that they may have goals inconsistent with FDA regulations. For example, the FDA may be considering the approval of a certain drug on its merits while the representative’s view is that different types of drugs are needed.

Dr. Katz ended his talk with a quote from Dr. Margaret Mead, the famed anthropologist, “Never doubt that a small group of committed people can change the world; indeed, it is the only thing that has.”

Tracked on the Lighthouse:
ASENT

# # #

Source:

printer friendly version

 

	Read the HDAC/HDLighthouse Forum. Post your comments