On April 30, 2010 a Notice of Public Hearing was published in the Federal Register. According to the notice, “The Food and Drug Administration (FDA) is announcing a public hearing regarding the Agency’s regulation of drugs, biological products, and devices (e.g., therapies and diagnostics) for the treatment, diagnosis, and/or management of rare diseases.” There will be two days available to provide testimony: June 29 and June 30, 2010.
When: Tuesday June 29 & Wednesday June 30, 2010 9am to 5pm ET
Where: 10903 New Hampshire Ave., Bldg 31, Rm 1503, Silver Spring, MD 20993
Oral Presentation: Written/Electronic Requests to Paras M Patel by May 31, 2010–
Phone: 301–796–8660, FAX: 301–847–8621
e-mail: OPDAR @fda.hhs.gov
Note: If you cannot attend the hearing, written/electronic comments will be accepted through August 31, 2010. Transcripts of the hearing will be available within 45 days.
I am interested in hearing what you might share at the Public Hearing. Please let me know via email if you would like me to testify on your behalf – I would like to gather some of your stories, comments, recommendations, etc. and provide a consolidated report to the FDA. Here’s some information directly from the Federal Register regarding the issue topics the FDA is particularly interested in receiving feedback on:
1. Orphan drug marketing applications are reviewed under the same review process and statutory standards regarding demonstration of safety, effectiveness, and product quality as drugs for patients with nonorphan diseases or conditions. FDA is sensitive to the unique needs of patients with rare diseases as it makes approval decisions regarding the overall risk benefit profile of therapies for the particular patient population for which they are being considered. Please comment on whether this practice has adequately addressed the needs of patients with rare diseases.
2. FDA designates a medical device as an HUD designed to treat or diagnose a rare disease—defined in this instance as a disease affecting or manifesting in fewer than 4,000 patients per year. Please comment on whether this practice has adequately addressed the needs of patients with rare diseases. Please also comment and provide your rationale on whether 4,000 patients constitutes an appropriate population size for an HUD determination. If improvements are suggested, please provide specific examples/suggestions for any recommended changes.
3. Current regulations for the approval of an HUD through the HDE pathway require that the application have a ‘‘description of the device and a discussion of the scientific rationale for the use of the device for the rare disease or condition’’ and ‘‘an explanation of why the probable benefit to health from the use of the device outweighs the risk of injury or illness from its use, taking into account the probable risks and benefits of currently available devices or alternative forms of treatment’’ (21 CFR 814.102 and 814.104). Please comment if you believe that these standards remain appropriate for the approval of devices for rare diseases under the HDE mechanism; please also comment whether a more precise definition of probable benefit is needed.
4. Have current processes for rare disease stakeholders to communicate with FDA regarding rare disease article development been useful? How could these processes be improved? Please provide specific examples/suggestions for any recommended changes.
Comments and feedback would be useful by COB Wednesday, May 19, 2010. However, because of the quick turnaround time I welcome your comments by COB Thursday, May 27, 2010.
Thanks in advance for all of your support and effort! ~ Howard
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Tomorrow night, Tuesday, May 18, 2010, the National Organization for Rare Disorders (NORD) will be hosting its annual gala – the Partners in Progress 2010 Gala – in Washington DC. The event will be emceed by Home Improvement and The West Wing actress Patricia Richardson, who’s father died of progressive supranuclear palsy (PSP), a little known but disabling brain disease. At the event NORD will be honoring Dr. Sami Said, a State University of New York (SUNY) professor, with the Partners in Progress Award. They will also be honoring Dr. Roscoe Brady of the National Institutes of Health (NIH), who developed the first effective treatment for Gaucher disease, with the Lifetime Achievement Award. Registration