Regulatory

Recent Activity

ARM Urges NIH to Change Role of RAC Review of Gene Therapy Protocols - Read More Here >>

ARM Statement Regarding Potential Budget Cuts to the FDA and NIH

UK House of Lords Submission

Sweeping FDA Reforms Successfully Enacted

Regulatory Overview

A major priority for the Alliance is to assist in creating clear and predictable regulatory pathways that facilitate rapid approval of safe and effective regenerative medicine products.

The Alliance has a robust regulatory agenda designed to achieve this goal, and has developed a collaborative working relationship with FDA. Commencing in May 2010 at a meeting with Commissioner Hamburg, center directors and senior agency staff, the Alliance began a series of productive meetings and interactions with the agency, and will continue to have regular meetings with senior FDA officials in the coming weeks and months. Specific regulatory initiatives resulting from these exchanges include:

Standards

After conversations with regulatory officials, it was clear that a review and analysis of scientific standards initiatives was necessary. In response, the Alliance performed a thorough gap analysis of existing standards that was shared with FDA. The analysis focused on standards related to cell potency assay development and validation, which are crucial to regenerative medicine product development and regulatory approval. The Alliance additionally formed working groups to identify key regulatory issues as well as provide information and support to product developers and the agency.

Improved Communication

Improved communication between sponsors and FDA is critical to support regulatory approval of regenerative medicine products, as evidenced by the high number of cell therapy INDs put on hold. The Alliance is developing a paper for publication that will outline best practices for commercial sponsors to communicate with the FDA during clinical development, from pre-IND through post-market.

Presentations at FDA Public Meetings

The Alliance presented at multiple FDA-sponsored public meetings to discuss efforts and best practices for translating scientific discoveries into innovative products for patients.

Submissions

The Alliance regularly takes advantage of FDA requests for public comment on topics germane to regenerative medicine. To date, the Alliance has publicly addressed: unmet needs for clinical translation of cell-based therapies; the FDA-NIH Leadership Council; improving regulation and regulatory review; and parallel review of medical products.

International Regulatory Harmonization

Global consistency of regulatory policy, where possible, will facilitate more rapid and efficient introduction of regenerative medicine products. The Alliance has formed a working group to develop policies that will improve international regulatory harmonization.