Regulatory & Legislative Priorities

As of August 2016:

Global reimbursement issues. Advancing specific proposals to enable market access and favorable reimbursement policies for gene and cell therapies and other regenerative medicine products.

  • Identifying potential policy and legal impediments to coverage and reimbursement
  • Conducting formal analysis of payment models to facilitate access and adoption
  • Outreach to U.S. CMS, private payers and EU HTA bodies and reimbursement agencies

Standards development. ARM and the members of the international regenerative medicine Standards Coordinating Body (SCB) steering committee are preparing an initial work plan for the group to address important near-term requirements for cell therapy, reference standards for in-vivo and ex-vivo gene therapies, tissue engineering and drug discovery. The group is also working with NIST and other federal agencies to finalize a public-private partnership model to support these activities.

Optimizing current regulatory and review pathways to ensure patient access to safe and efficacious regenerative medicine products. This includes:

  • ARM continues to work closely with FDA to identify opportunities for improvement and optimization.
  • A modified role for the NIH-Recombinant DNA Advisory Committee (RAC) to ensure the streamlined oversight of gene therapy clinical trials.

Combination products. ARM advocates for reforms to optimize the review process for combination products or other situations when more than one review center at FDA is involved in product evaluation and review.

EU GMP for ATMP consultation. The European Commission has published a new targeted multi-stakeholder consultation on the draft guidelines Good Manufacturing Practices specific to ATMPs. ARM is developing its response for submission in Q3 2016.

International regulatory convergence. ARM is working to establish and maintain a predictable and efficient regulatory review and approval process in the U.S. and EU to promote greater international harmonization.

Gene editing & related bioethics issues. ARM is working closely with the National Academy of Sciences (NAS), and has provided a detailed industry perspective on the state of commercialization of somatic cell gene editing technologies and the existing regulatory framework. This information will be included in the NAS upcoming consensus report to be released by EOY 2016.

PDUFA reauthorization. The current Prescription Drug User Fee Act (PDUFA) version V is set to expire September 2017. ARM has convened a working group to discuss ARM’s goals for the reauthorization and will provide a statement to be submitted at the upcoming mid-August public meeting.

FDA draft guidance on minimal manipulation. ARM will comment on and help to shape FDA’s draft guidance on minimal manipulation and homologous use on human cell and tissue products. ARM will also present at the FDA public meeting in September 2016.

National Academies Forum. ARM has been invited to be a member of the NAS Forum on Regenerative Medicine. The Forum will examine regulatory and reimbursement issues.