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 contributed to the targeted stakeholder consultation on new genomic techniques to contribute to a European Commission study requested by the Council. ARM’s response provides an update on the progress in gene & gene modified cells therapies, including products based on genome editing; stresses the challenges posed by the GMO legislation in Europe; and proposes a reconsideration of whether the GMO legislation should apply for medicinal products.
The lack of a European Master File system creates a challenge for manufacturers of raw materials or specialised technologies as they are subject to requests and audits by ATMP developers to ensure that the information they present within the Clinical Trial Application or Marketing Authorisation Application is accurate and that the product is of sufficient quality.
ARM supports the issuance of guidance on this gene therapy sameness in regards to orphan drug exclusivity as it “would help incentivize new gene therapy products…as well as facilitate patient access to new therapies by providing clarity on FDA’s thinking…”
While ARM welcomes the Roadmap’s recognition of the wider industry’s contribution to the EU economy and its focus on ensuring patients’ access to safe, state-of-the-art therapies, ARM wants to ensure a continued pathway for the EU to benefit from innovative and life-changing advanced therapy medicinal products (ATMPs). More information is available here.
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.
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.