FDA’s Guidances for Cell Therapy: Update on Seminars, Workshops and Public Reactions

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New Seminar on FDA’s Regulations for Stem Cell Therapy Development

If you are interested in learning more about the FDA’s current guidelines for the approval of stem cell therapies as well as the different guidances regulating to the various approval processes (INDs, BLAs etc.), then a new seminar might be of significant benefit.

FDA’s Regulation of Regenerative Medicine including Stem Cell Treatments, Tissue Engineering and Gene Therapies is a two-day seminar aimed at those involved, in some way, in the cell therapy development process – from regulatory, quality and compliance personnel to clinicians and researchers – that aims to present in detail the various guidances associated with the drug development and approval processes, but also design, GMP and GLP guidances, as well as product labeling and marketing.

The seminar will take place over two sessions: the first one is in Irvine, CA on July 14-15, and the second is in Newark, NJ from October 15-16, 2016.

The speaker at the seminars is Dr. Thomas J. Webster, Department Chair and Professor of Chemical Engineering at Northeastern University.

For more information and to register, follow this link.


FDA Postpones Public Hearing on Draft Guidances and Adds Scientific Workshop

The FDA was to hold a public hearing for interested stakeholders to provide feedback on their recently-released Draft Guidances Relating to the Regulation of Human Cells, Tissues or Cellular or Tissue-Based Products. The hearing was to take place on April 13, 2016, at the FDA’s White Oak Campus in Silver Spring, Maryland.

The purpose for the hearing is to provide stakeholders an opportunity to discuss the FDA’s proposed regulation of HCT/Ps regarding homologous use, same surgical procedure exception, minimal manipulation, and adipose tissue.

Due to the high interest, the FDA has postponed the hearing to a date later this year.

However, ina ddition to the hearing, the FDA now plans to also add a scientific workshop to “gather information from manufacturers of cell based products, clinical researchers […] regarding the generation of scientific evidence to facilitate the development of safe and effective cell based therapeutics.”

More details on the hearing and workshop will be provided once known.


Bipartisan Policy Group Releases Cell Therapy Document

This Winter, the Bipartisan Policy Group (BPC) – a Washington, DC-based political think-tank promoting various agendas aimed at advancing areas of national interest –

unveiled a document, titled Advancing Regenerative Cellular Therapies, which aims to presents new pathways for the development of cellular therapies that would allow for the more rapid and clinically efficient translation of discoveries to viable commercial therapies.

The document proposes a number of significant recommendations to the FDA. The bulk of the new proposals are centered around a “conditional-approval” clause. In summary, their recommendations are:

• A proposal for the FDA to grant a time-limited, conditional approval for cell-based therapies based on preliminary clinical evidence of safety and efficacy, without Phase 3 trials;

• Allow for patients to be granted limited access to these conditionally-approved therapies

• Require the sponsor of the conditionally-approved therapies to submit a BLA within three years of receiving conditional approval or negotiate with the FDA an extended conditional period to collect  additional data

• Permit reimbursement during the conditional approval period

The entire document can be accessed here.

These proposals have been met with caution, and they have been called misguided and controversial elsewhere – particularly with respect to the “conditional approval” clause.

We will not comment on the validity of these proposals, but invite you to read the entire report and look for the industry’s reactions directly.



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