CMC & Manufacturing

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ARM identifies barriers to the manufacturing and scale up of regenerative medicines and works with its multi-stakeholder audience to develop and implement solutions.

Overcoming Challenges in Manufacturing

Over the past decade, advanced therapies have transitioned from academic dream to a clinical reality. Though this field has the potential to dramatically transform the health care landscape and treat patients who have in the past had limited or no treatment options, the shifting treatment paradigm also presents development and marketing hurdles which are different from traditional therapies.

These therapies pose unique challenges for manufacturing and scale up, including shorter shelf lives, greater temperature sensitivities, and increased complexity and cost related to purity and identity testing; producing sufficient supply of starting materials, such as cell lines or viral vectors; autologous manufacturing processes for many products, which requires material from the patient to be collected and modified prior administration; and Chemistry, Manufacturing and Controls (CMC) requirements which are not adequately tailored to the specific needs of regenerative medicines or which create and duplicative processes for testing, reporting, and clinical submissions from country to country.

To ensure broad patient access to these transformative therapies in a timely manner, sector stakeholders must now convene to identify and address these challenges.

ARM's Role in Manufacturing

  • Assess all FDA, EMA and related CMC guidance relevant to cell and gene therapy and make recommendations where needed to promote a positive environment for manufacturing these products.
  • Promote international convergence of regulation and guidance by identifying inconsistency and developing proposals for adoption by regulatory agencies.
  • Identify best practices for establishing robust manufacturing processes and quality control.
  • Educate the rapidly expanding regenerative medicine workforce via workshops, white papers, and webinars.
  • Collaborate with regenerative medicine stakeholder groups and recognized standards organizations to encourage harmonization in manufacturing and CMC standards and guidances.

 

ARM’s IN VIVO Article, Published July 2019
“Manufacturing Cures: Infrastructure Challenges Facing Cell and Gene Therapy Developers”
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ARM's Current Areas of Focus

  • Projects A-Gene and A-Cell

    ARM is currently developing the “A-Gene” and “A-Cell” projects, intended to create a case study-based reference guide on the best practices for the development of gene therapies and cell therapies, respectively. An expert team, including representation from leading commercial and academic groups, will address key topics in the development and manufacturing of cell and gene therapies, including comparability, critical quality attributes, the product life cycle, the development and use of standards, regulatory implications, and others. The resulting white papers will help advance the principles of Quality by Design (QbD) during the manufacture of gene- and cell-based therapeutics.

  • Manufacturing Workshop Series

    ARM’s Manufacturing Workshop Series is intended to convene sector stakeholders, including ARM members, FDA and EMA officials, and others in order to discuss issues relevant to the manufacturing of regenerative medicines, including comparability, international frameworks, and starting & ancillary materials.

    • On May 31, 2019, ARM and the US Pharmacopeia (USP) co-hosted a workshop titled “Comparability in Cell and Gene Therapies” in response to a running series of challenges in understanding Comparability for next generation therapeutics. The workshop attracted 120+ participants. A final report and summary from the workshop is available here.

Submitted Comments

December 2018

March 2018