Regulatory

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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.

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 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.

  • 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.

    March 29, 2018

    ARM CEO Janet Lambert sent a letter to CBER Director Peter Marks in response to FDA Commissioner Gottlieb’s announcement that the agency will be developing disease-specific guidances for gene therapy, the first of which covers hemophilia treatments.


    March 2018
    ARM submitted comments on FDA’s draft guidance for industry, “Expedited Programs for Regenerative Medicine Therapies for Serious Conditions,”
  • 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.
    MARCH 2018
    ARM S&T and Regulatory Committees’ joint task force submitted comments to the FDA on its recent CMC and Standards guidance.
  • 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.