RMAT Designation – Impact on Regenerative Medicine Sector
Gain a deeper understanding of the implications of the Regenerative Medicine Advanced Therapies (RMAT) designation and the significance of its support and advancement by the FDA in this webinar recording. Held in partnership with the Alliance for Regenerative Medicine (ARM), ARM, Informa Pharma Intelligence, and representative from Abeona, Athersys, and Enzyvant discuss the RMAT designation and its implications on the sector.
Michael Werner, Co-Founder and Senior Policy Counsel for ARM, and Robert Falb, Director of U.S. Policy and Advocacy, participated in BIO’s webinar on NTAP and advanced therapies on May 9. ARM believes that it is critical that the NTAP program be modernized to facilitate patient access to new and life-saving treatments.
CAR-T cell therapy is now approved in the U.S. The first product, Novartis’ Kymriah, is approved to treat pediatric and young adult patients with B-cell acute lymphoblastic lymphoma, and products for other oncological indications are hot on its heels. Join Informa Pharma Intelligence and ARM for an interactive webinar to explore what this historic approval means for the patient community, as well as how this sector will approach the scientific, clinical, policy and business issues surrounding CAR-T approvals.
ARM submitted a letter to FDA’s CBER Director, Dr. Peter Marks, concerning ARM’s recommendations on guiding principles that may be helpful to determining approaches to disease-specific guidances as well as finalizing the recently published diseases-specific guidances for gene therapy.
ARM submitted comments on the Department of Health and Human Services (HHS) Request for Information: Blueprint to Lower Drug Prices and Reduce Out-of-Pocket Costs, encouraging HHS to consider the potential value of cell and gene therapies to patients and society, and the need to enable new pricing and reimbursement approaches that can help make them available to patients.
ARM published its position in response to the proposal for a Regulation of the European Parliament and of the Council on health technology assessment and amending Directive 2011/24/EU (COM(2018) 51 final). ARM’s position statement recognizes the benefit of the proposed regulation, which would require joint clinical assessments to be carried out for many medicinal products undergoing the central marketing authorization. In the statement, ARM also makes three recommendations intended to ensure the success of the regulation in attaining the full impact of those benefits.
ARM submits comments to CMS requesting that the agency rescind the planned NCA on the basis that it was prematurely undertaken and because the NCA may have a significantly chilling effect on access to the approved therapies and curtail clinical and financial investment in these technologies. If CMS continues with the NCA, ARM has identified several issues which we encourage CMS to acknowledge and address.
ARM submitted its comments regarding the modification of Medicare’s New Technology Add-on Payment Program (NTAP) to reflect the potential added value of regenerative medicine products to the healthcare system. These comments were drafted in response to the proposed changes by the Centers for Medicare and Medicaid Services (CMS) to the Hospital Inpatient Prospective Payment System, issued August 2017.
ARM has submitted comments on the EMA’s “Guideline on Safety and Efficacy Follow-Up and Risk Management of Advanced Therapy Medicinal Products.” In addition to various requests for clarification throughout the guidance, ARM also suggested the creation of a separate document for pre-authorization safety expectations, or to clarify whether the recommendations apply to all ATMPs or only to specific ATMP types
ARM CEO Janet Lambert sent a letter to CBER Director Peter Marks in response to FDA Commissioner Gottlieb’s announcement that the agency will be developing disease-specific guidances for gene therapy, the first of which covers hemophilia treatments.
ARM submits comments in response to the Office of Inspector General’s request regarding the OIG’s “safe harbors,” activities that are deemed in advance to not violate the Anti-Kickback Statute and other laws. These provisions can complicate or limit pay-for-performance arrangements with CMS.