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Regulatory

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.

Promoting Clear and Predictable Regulatory Pathways

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.

ARM's Role in the Regulatory Environment

Review & Identify

Review existing scientific standards and initiatives to identify key regulatory issues and to provide information and support to product developers and regulatory agencies.

Advocate & Improve

Advocate for improved communication between product developers and regulatory bodies throughout the development pipeline.

Present & Discuss

Present at public meetings held by regulatory bodies to discuss best practices for translating scientific discoveries into innovative products for patients.

Submit Comments

Submit comments on the implications of proposed regulations for the regenerative medicine sector.

Maintain & Modify

Work with legislators and other policymakers to maintain and modify as appropriate the regulatory framework for regenerative medicine and advanced therapies.

ARM's Current Areas of Focus

RMAT Designation

ARM was instrumental in the development of the regenerative medicine provisions in the 21^st 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.
Regulatory Guidances for 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.

april 2019

ARM submitted comments on FDA’s draft guidance for industry, “Rare Diseases: Common Issues in Drug Development.”

March 2018

ARM submitted comments on FDA’s framework document, “Framework for FDA’s Real-World Evidence Program.”
Review of CMC and Other Related Guidance

ARM is working with FDA to ensure that its CMC and other manufacturing guidance is updated to appropriately apply to cell and gene therapies.

December 2018

ARM S&T and Regulatory Committees’ joint task force submitted comments to the FDA on its recently released six draft guidances for gene therapy, including three CMC-focused guidances.
Pan-European Regulatory Convergence

ARM is working with national authorities throughout Europe to promote consistency and harmonization among national standards with regard to hospital exemption, GMO requirements, blood and cell tissue directives, and good manufacturing practices (GMP) for advanced therapy medicinal products.

MARCH 2018

ARM provides recommendations that support and complement the EMA’s & EC’s Action Plan on ATMPs. ARM’s proposals elaborate on the organizations’ initiatives, emphasizing areas of priority and aspects not covered in the original plan, with specifics for next steps, grouped into four main areas of focus: Research & Development; Regulatory Processes; Hospital Exemption Pathway; Funding, Investment, & Market Access.