This webinar is intended to provide members of the press in Europe with an overview of the ATMP landscape in Europe, including major financings, the clinical pipeline, and the potential for these therapies to significantly improve the standard of care for many patients suffering from devastating diseases and disorders. The webinar also provides a brief introduction to ARM and ARM’s role in improving the landscape for ATMPs in Europe and globally.
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.
ARM released a Therapeutic Developers’ Statement of Principles, setting forth a bioethical framework for the use of gene editing in therapeutic applications. The statement, developed by ARM’s Gene Editing Task Force and signed by 13 therapeutic developers using gene editing technologies, specifies five key principles for the ethical use of gene editing and genetic modification. The statement includes a common commitment against germline genome modification unless and until ethical and potential safety questions with respect to germline gene editing are adequately addressed.
ARM believes that the proposed standard for the Certificate of Need Program requiring that any facility to receive both the Commission’s approval and a third-party accreditation from the Foundation for the Accreditation of Cellular Therapy (FACT) in order to deliver CAR-T therapies to patients in Michigan will be an onerous barrier to access, create an unnecessary financial burden on health care facilities across the state, and limit the sites of care from offering cellular therapies to patients.
ARM welcomes the discussion paper on the use of patient disease registries for regulatory purposes and believes this initiative can be instrumental in the adoption of standards for Real World Evidence (RWE). Registries represent one of the main tools to document long-term safety and efficacy effects and generate RWE; therefore, they are particularly important for ATMPs. ARM’s recommendations are intended to ensure that these registries align with the needs of RWE infrastructures and long-term follow-up to improve access to ATMPs.
ARM appreciates that CMS has discussed a few options to improve the current NTAP and overall MS-DRG system with a focus on creating a methodology and a system that balances appropriate access and cost effective care to new and innovative therapies. ARM suggests that CMS adjust its CAR T specific reimbursement policies to utilize actual drug acquisition costs, rather than marked up charges in NTAP and outlier payment calculations for fiscal year (FY) 2020; finalize policies for FY 2020 that create the infrastructure to move toward an appropriately valued CAR T MS-DRG in future fiscal years; recognize certain FDA approval designations for drugs as dispositive for newness and substantial clinical improvement; and establish a more frequent NTAP process. ARM also disagrees with CMS’ proposal to change the severity level designation for 13 ICD-10-CM diagnosis codes from categories I21 and I22 from a MCC to a CC.
While ARM is supportive of the OIG’s efforts to reexamine the applicable regulatory safe harbors currently in place under the anti-kickback statute, we are concerned that the proposed change would serve to also exclude arrangements involving the application of price concessions based on value.
ARM commends the FDA for the revision of the draft guidance on common issues in rare disease drug development. We recognize that the standard for safety and efficacy for drug approval does not differ between orphan products for rare diseases in comparison to the standards for drug approval for products for common diseases. However, there is need for regulatory flexibility to support drug development for rare diseases. We request the Agency to consider how the guidance can leverage even more comprehensively the flexibility afforded by the FDA in the context of drug development in rare diseases.
It is critical for CMS to develop and implement policies and programs that support beneficiary access to new technologies when they are deemed most clinically appropriate and stimulate their continued development. This is particularly true for regenerative medicine and other advanced therapies that hold the promise of durably treating and potentially even curing chronic or life-threatening diseases. In light of this goal, ARM asks CMS to ensure Medicare patients who can benefit from CAR T therapies are covered under the NCD, allow CAR T to be administered in certified, trained, experienced facilities and not limit access to hospitals, and certify a registry to be in place by May 17 to ensure continued access to CAR T by Medicare beneficiaries.