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’s comments encourage further clarification of the use of real world evidence (RWE) in satisfying post-approval requirements for products with RMAT designation. ARM also believes that the RWE framework document should take incorporate special considerations to address issues specific to the use of RWE for RMAT products, including the high unmet medical need many of these products are designed to address, as well as the potential for small patient populations due to the number of RMAT products designed to address rare and ultra-rare diseases.
ARM filed comments in response to a Trump Administration proposal which would change the current Medicare Part B reimbursement model, which is based on a manufacturer’s average sale price (ASP) in the United States in combination with an add-on payment, to one that would adopt an International Pricing Index. While commending the effort to align value with reimbursement, ARM believes that the proposal will fall short of this goal and harm patient access to regenerative medicines.
ARM expressed support for the draft guidance recommendations and the structure of the in general. ARM also requested further clarity in certain sections of the guidance, including the recommended information needed in order for sponsors to request a pre-IND meeting and the expectations for background material for a standard pre-IND meeting.
ARM submitted comments on the FDA’s six draft guidances for gene therapy, released in July 2018. The guidance documents are intended to build on a framework for regenerative medicine released last November. Three are focused on disease areas (hemophilia, retinal disorders, and rare diseases) while the others outline standards for manufacturing, testing, and long-term follow-up. ARM’s comments include a number of recommendations for each of the draft guidances, including: the use of a consistent definition of gene therapy throughout the six guidances; a new version of the Common Technical Document with examples for gene therapy; cross-referencing between the guidance documents; and other issues.
ARM appreciates CMS’ statements related to the similarity between data satisfying FDA designations and data satisfying substantial clinical improvement. This clarity will help future applicants understand which types of data can serve as the foundation for the satisfying the substantial clinical improvement criterion. In addition, ARM urges CMS to change the payment rate for certain classes of NTAP recipients. ARM is concerned that without changes to the current reimbursement methodology for CAR-T therapies and other regenerative and cell therapies that ARM represents, the NTAP will not satisfy the congressional intent of the NTAP program, which is to adequately reflect the estimated average cost of such service or technology.
ARM prepared a letter to the European Commission on GMO requirements for gene therapies, asking that they consider additional proposals to facilitate and streamline the approval process with GMO authorities. These proposals included a more streamlined approach to assessment of ATMPs containing or consisting of GMOs; additional guidance on interpretation of GMO/GMM Directives (Directive 2001/18/EC and Directive 2009/41/EC) in context of ATMPs; increased transparency of discussions on streamlining the EU GMO framework for ATMPs; and multi-stakeholder workshop on GMO aspects of clinical trials involving ATMPs.
ARM submitted comments to the Department of Health and Human Services’ Office of Inspector General in response to an RFI concerning the Anti-Kickback Statute and value-based payment models. The current structure of the Anti-Kickback Statute creates regulatory and legal barriers to federal implementation of value-based payment models for innovative, one-time and durable therapies. ARM encourages the OIG to consider the cost of initial assessments, the potentially limited geographic access to regenerative medicine products for patients, and the need for continuing evaluation when developing safe harbors.
ARM provided comments to the NIH in response to the proposed changes to the NIH guidelines for research involving recombinant or synthetic nucleic acid molecules. ARM supports the removal of duplicative review and data reporting requirements as previously required by NIH guidelines.
ARM provided comments on the CMS requests for information on promoting interoperability and electronic health care information, price transparency, and leveraging authority for the Competitive Acquisition Program (CAP) for Part B drugs and biologicals for a potential CMS innovation center model.
ARM submitted comments in response to the public consultation on the EMA draft qualification opinion on the cellular therapy module of the European Society for Blood & Marrow Transplantation (EBMT) Registry.
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.