Home Topics Public Policy NVCA Weighs In on FDA Transparency and Treating Rare Diseases

23

Jul

2010

NVCA Weighs In on FDA Transparency and Treating Rare Diseases PDF Print E-mail

Kelly Sloane

Written by Kelly Slone   
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The NVCA offered a venture capital perspective in two critical areas this week as it relates to medical innovation, all on the heels of submitting two nominations for the newly created Patient Centered Outcomes Institute (PCORT) Board of Governors.

On July 20, the NVCA submitted our formal comments on FDA's Transparency Initiative Draft Proposal which provides recommendations on the FDA's public disclosure policies.  The draft proposal falls into seven categories (1) adverse event reports; (2) docket management practices; (3) enforcement priorities and actions; (4) import procedures; (5) inspections; (6) product application; (7) recalls; and (8) warning and untitled letters.  Our comment letter focused on concerns regarding several provisions that would especially harm emerging growth companies and impact venture capital investment.  These provisions include the disclosure of confidential, highly sensitive information related to product applications that does not achieve public health objectives, and will put companies at a competitive disadvantage. You can read our full comment letter here.

On July 21 at the request of Senator Mike Enzi (R-WY), the NVCA submitted a statement to the Senate Health, Education, Labor and Pensions (HELP) Committee for their hearing on "Treating Rare and Neglected Pediatric Diseases: Promoting the Development of New Treatments and Cures."   Our statement asserted that:

NVCA wants to promote greater investment in this important area.  However, the incentives for investment in rare diseases are at risk in a number of ways, and we must act now to ensure that the venture capital-backed innovation engine can continue to deliver important new therapies for rare diseases.  Among other issues, we must ensure that the regulatory environment is conducive to investment in this area.… It is essential that regulators recognize the unique challenges of developing treatments for rare diseases, and show appropriate flexibility in the application of regulatory standards, to promote the continued development of new therapies for rare diseases while continuing to ensure the safety and efficacy of new treatments.

Both of these documents follow our efforts to ensure that the medical innovation community is well represented in the crafting of public policy.  On June 30, the NVCA submitted two nominations for the newly created Patient Centered Outcomes Institute (PCORI) Board of Governors that was established as part of the new health reform law; Dr. Risa Stack, Partner, Kleiner Perkins Caufield & Byers and Dr. Paul Yock, Martha Meier Professor of Cardiovascular Medicine and Founding Co-Chair of the Department of Bioengineering at Stanford University.  The implementing legislation for PCORI requires the inclusion of three Board members from the pharmaceutical, medical device and diagnostic industries.  NVCA believes that one of the industry representatives should be focused on development of medical innovation and have experience working with early stage companies and novel medical products. Dr. Stack and Dr. Yock both have the credentials to represent these important areas.  Representative Eshoo, (D-CA) sent letters supporting both candidates as well. 

The NVCA appreciates the opportunity to engage in the discussion surrounding these issues that are so critical to ongoing medical innovation in the United States and looks forward to having a seat at the table as these and other issues progress.

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