As more regenerative medicine products approach clinical testing and commercialization, seize this opportunity to hear detailed insights and industry best practices from preeminent sector experts. Gain a deeper understanding of the many manufacturing and industrialization hurdles unique to bringing durable and even curative therapies to patients in need.
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.
BIO and ARM recommend establishing a DRG for Fiscal Year 2021 for CAR T-cell therapy cases to provide appropriate reimbursement for treatment and protect access to care for Medicare beneficiaries. It’s critical that this be developed for FY 2021 before the new technology add-on payment (NTAP) expiration for currently available CAR T-cell therapies occurs on September 30, 2020, which would result in a significant drop in provider reimbursement nationwide that could impact patient access to CAR T.
While ARM understands the intent of the new standards, we are very concerned about the precedent of having the CON Commission create a new standard of eligibility of a site of care that wishes to provide cutting edge cellular immunotherapies. ARM believes that the current U.S. Food and Drug Administration (FDA) label requirements and the Risk Evaluation and Mitigation (REMS) for each cellular therapy provides the appropriate safety criteria and standards. The additional requirements put forth by the CON Commissions have the potential to negatively impact patient access.
The HTA evaluation issues raised in the ICER press release raise critical concerns for ARM members. ARM requests that ICER augment efforts to use Real World Evidence; expand and revise voting structure to capture important potential other benefits and contextual considerations not captured in the cost-effectiveness analysis; and create a new process for re-assessing the emergence of new evidence. ARM also highlighted areas of concern, including the premature timing of review, cost-effectiveness threshold ranges, the discounted approach to cost-effectiveness modeling, imprecise evidence ratings, and the use of the German HTA system prior to an international HTA standard being set.
ARM believes that while there are some positive elements in ICER’s proposal, overall, the value assessment methods are inadequate to fully reflect the long-term value of these transformative therapies.
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.