ARM is the sector’s lead advocate for clear, predictable, and harmonized regulatory and review pathways for regenerative medicine and advanced therapy products in the U.S. and Europe.
Clear and predictable approval process regulation ensures product developers are able to understand the data and other requirements needed for approval and navigate the regulatory process without unnecessary delays or uncertainty.
Similarly, internationally harmonized regulation – the creation of globally consistent regulatory policies – facilitates the rapid and efficient development and introduction of regenerative medicine products across multiple markets worldwide, to the benefit of both manufacturers and patients.
Review existing scientific standards and initiatives to identify key regulatory issues and to provide information and support to product developers and regulatory agencies.
Advocate for improved communication between product developers and regulatory bodies throughout the development pipeline.
Present at public meetings held by regulatory bodies to discuss best practices for translating scientific discoveries into innovative products for patients.
Submit comments on the implications of proposed regulations for the regenerative medicine sector.
Work with legislators and other policymakers to maintain and modify as appropriate the regulatory framework for regenerative medicine and advanced therapies.
ARM was instrumental in the development of the regenerative medicine provisions in the 21st Century Cures Act, including the establishment of the Regenerative Medicine and Advanced Therapy (RMAT) designation, intended to expedite the approval process for safe and effective advanced therapies for severe or life-threatening conditions. ARM is continuing to work with the FDA to ensure that a clear RMAT framework is in place, enabling designations for multiple cell and gene therapy products.
ARM works with the FDA, EMA, and other regulatory bodies to shape new guidance for therapies, such as the FDA’s disease-specific guidance for gene therapy targeting high-need diseases and disorders.
ARM submitted comments on FDA’s draft guidance for industry, “Rare Diseases: Common Issues in Drug Development.”
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’s comments to the EMA in this questionnaire are intended to provide recommendations for ATMP-specific goals and strategies for inclusion in the EMA’s Regulatory Science to 2025 document.
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.
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 recommends that the agency issue separate guidance specifically for rare and serious diseases with unmet medical needs and expressed concern “that the draft guidance does not acknowledge the regulatory flexibility for serious conditions.”