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’s comments provide an alternative proposal for the format and content of the annual summary which we feel better aligns with the roles and responsibilities and requirements under the Right-to-Try Act.
ARM calls for greater CBER representation in the assessment and urged the agency to ensure that the patient perspective is considered.
ARM reiterates our stance that germline genome editing is currently inappropriate for in-human use. In regards to somatic genome editing, ARM cautios the World Health Organization that in certain regions where effective governance methods are already in place, duplicative requirements could hamper innovation. Additionally, ARM agrees with the WHO’s concerns regarding unscrupulous clinics promoting unproven therapies, and also urged the organization to include informed consent in the governance document.
In comments submitted to the European Commission, ARM highlights the potential for the European Health Data Space (EHDS) to advance patient access to cell and gene therapies.
ARM responds to FDA’s draft guidance on individualized antisense oligonucleotides, requesting guidance on regulatory considerations to support first in human exposure/treatment. ARM also notes that this was a CDER draft guidance and requested “procedural guidance and regulatory considerations from CBER on cell and gene therapies for rare and very rare severely debilitating or life-threatening diseases, with no adequate therapies.”
ARM’s recommendations for the next PDUFA agreement include appropriate funding to ensure FDA as the necessary resources to recruit, train and retain CBER reviewers; increased resources to establish and implement strong, swift and consistent legal actions against unregulated stem cell clinics; improvements to the INTERACT meeting process; increased flexibility during CMC reviews of cell and gene therapies; a public meeting to focus on the unique issues associates with the use of Real World Evidence (RWE) for cell and gene therapies; and the issuance of a “COVID-19 lessons learned” report examining pandemic-related disruptions to standard process, policies and procedures and what modifications should be changes should be adopted moving ahead.