05-9011. Biological Products for Treatment of Rare Plasma Protein Disorders; Public Workshop

AGENCY:

Food and Drug Administration, HHS.

ACTION:

Notice of public workshop.

SUMMARY:

The Food and Drug Administration (FDA) Office of Public Health and Science in the Department of Health and Human Services, are announcing a public workshop entitled “Biological Products for Treatment of Rare Plasma Protein Disorders.” The purpose of the workshop is to discuss the scientific and regulatory challenges encountered during the development of biological products used to treat rare plasma protein disorders. The workshop also will include a discussion about options that could be used to facilitate future product development.

Date and Time: The 2-day public workshop will be held on June 13 and June 14, 2005, from 8:30 a.m. to 5 p.m.

Location: The public workshop will be held at the National Institutes of Health, Lister Hill Auditorium, Building 38A, 8600 Rockville Pike, Bethesda, MD 20894.

Contact Person: Rhonda Dawson, Center for Biologics Evaluation and Research (HFM-302), Food and Drug Administration, 1401 Rockville Pike, suite 200N, Rockville, MD 20852-1448, 301-827-3514, FAX: [phone], email: [e-mail].

Registration: There is no registration fee for the workshop. Registration by May 30, 2005, is recommended due to limited seating. There will be onsite registration, on a space available basis, the first day of the workshop, beginning at 7:15 a.m. If you need special accommodations due to a disability, please contact Rhonda Dawson at least 7 days in advance of the workshop.

SUPPLEMENTARY INFORMATION:

FDA and Office of Public Health and Science in the Department of Health and Human Services are co-sponsoring a 2-day public workshop entitled “Biological Products for Treatment of Rare Plasma Protein Disorders.” The opening session of the workshop will include presentations from national and international regulatory officials, patient groups, health care providers, and manufacturers concerning the need for therapeutic products to treat plasma protein disorders that may affect small patient populations, and the obstacles to developing these products. The second session of the workshop will include discussions about regulatory issues affecting industry, including trial designs, statistical considerations, orphan drug provisions, product development, and case studies. The last session of the workshop will include presentations and discussions on other relevant topics, including the availability and possible use of patient registries, research support, reimbursement, potentials for international harmonization, modifying clinical trial design, and facilitating future product development.

FDA will post the agenda for this public workshop, when finalized, on CBER's Web sites at http://www.fda.gov/​cber/​scireg.htm and http://www.fda.gov/​cber/​minutes/​workshop-min.htm.

Transcripts: Transcripts of the meeting may be requested in writing from the Freedom of Information Office (FOI), (HFI-35), Food and Drug Administration, 5600 Fishers Lane, rm. 12A-16, Rockville, MD 20857, approximately 15 working days after the workshop at a cost of 10 cents per page. The transcript will also be placed on the FDA Web site at http://www.fda.gov/​cber/​minutes/​workshop-min.htm.

Dated: April 29, 2005.

Jeffrey Shuren,

Assistant Commissioner for Policy.