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.
And 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ARM submitted comments on FDA’s draft guidance for industry, “Rare Diseases: Common Issues in Drug Development.”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’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 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 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 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 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.