For carcinoid and neuroendocrine tumor patients who would like their voices to be heard regarding the Food and Drug Administration’s (FDA) regulation of drugs, biologics, and devices for the diagnosis and treatment of rare diseases, you are invited to attend a public hearing on June 29 and 30 in Silver Spring, Maryland. The FDA’s goals is to “gain input from patients, industry, healthcare providers, academic researchers and others to help share the future work of FDA’s committee for rare diseases.” Did you know that 1 out of 10 Americans has a rare disease?
For FDA basics, click on the above illustration.
A representative of the National Organization for Rare Disorders (NORD) will speak at the meeting. Any representative of a patient organization who would like to connect with NORD regarding speaking at the hearing or submitting comments afterwards can contact Diane Dorman at NORD’s Washington Office (ddorman@rarediseases.org).
The public hearing will be held from 9 a.m. to 5 p.m. on Tuesday, June 29 and Wednesday, June 30 at 10903 New Hampshire Avenue, Building 31, Room 1503, Silver Spring, Maryland. The hearing is free and seating will be on a first-come, first-served basis. Attendees who do not wish to make an oral presentation do not need to register. Anyone wishing to make an oral presentation during the hearing must register by submitting a written or electronic request to Paras M. Patel by May 31, 2010. For those unable to attend the hearing, written or electronic comments will be accepted afterward until August 31.
Established in March 2010, the FDA committee for rare diseases considers how the agency reviews data from non-clinical studies and clinical trials, and makes decisions about marketing authorization and post-marketing surveillance for products being developed for the rare disease patient population.
The hearing will provide an opportunity for advocates for patients with rare diseases, healthcare providers, the pharmaceutical industry and other interested individuals “to relate their experience with, concerns about, and suggestions for the way FDA regulates the scientific evaluation of, marketing authorization for, and post-marketing surveillance of products for rare diseases.”
Specific questions to be considered include:
- Should orphan drug marketing applications continue to be reviewed under the same process, and with the same standards, as non-orphan products?
- Do the current processes regarding HUDs (Humanitarian Use Devices) adequately address the needs of rare disease patients?
- Are current standards for approval of devices for rare diseases under the HDE (Humanitarian Device Exemption) mechanism appropriate?
- Have current processes for rare disease stakeholders to communicate with FDA been useful?
For information about how to submit comments or requests for oral presentations, and for additional information about the hearing, read the Federal Register notice.
Questions? Contact: Paras M. Patel, Food and Drug Administration, 10903 New Hampshire Avenue, Building 32, Room 5271, Silver Spring, MD 20993-0002; (301) 796-8660 or fax (301) 847- 8621; OPDAR@fda.hhs.gov.
