Regulatory Updates


The American Society of Gene & Cell Therapy monitors regulatory activity in the United States that may affect the gene and cell therapy field. The information on this page provides ASGCT members and stakeholders with updates on the release of FDA guidance documents related to the field, as well as other relevant regulatory developments.

FDA Guidance Documents

Regulatory News

FDA Guidance Documents

FDA Issues Guidance Documents to Enhance Interactions With Product Developers

Updated: Decmeber 18, 2017

The FDA announced the availability of two guidance documents on December 28 that aim to facilitate early, thorough interaction between the FDA and product developers. The following final guidance supports communication between those submitting investigational new drug applications (INDs) and the FDA’s review staff at critical junctures in development, to maximize efficiency of the process:

Best Practices for Communication Between IND Sponsors
and FDA During Drug Development

The following draft guidance provides recommendations for efficient, consistent procedures for the timely and effective conduct of formal meetings between the FDA and those developing new drug or biologic products. This guidance has a 90-day comment period: 

Formal Meetings Between the Food and Drug Administration
and Sponsors or Applicants of PDUFA Products

FDA CBER Issues Draft Guidance on Standards

Updated: December 19, 2017

The FDA CBER issued a draft guidance that includes information on how sponsors can utilize standards to facilitate product development and FDA acceptance of use of standards. Comments may be submitted until March 19, 2018:

Standards Development and the Use of Standards in
Regulatory Submissions Reviewed in the Center for
Biologics Evaluation and Research

FDA Framework for Regenerative Medicine

Updated: November 16, 2017

The FDA announced a comprehensive regenerative medicine policy framework in November, outlined in two finalized guidance documents, and two draft guidance documents. The first final guidance document clarifies the determination of whether HCT/Ps are subject to FDA’s premarket review requirements:

Regulatory Considerations for Human Cell, Tissues,
and Cellular and Tissue-Based Products: Minimal
Manipulation and Homologous Use

The second final guidance clarifies the qualifications for an exception from the established regulations for HCT/Ps that are removed from and implanted in the same individual within the same surgical procedure and remain in their original form:

Same Surgical Procedure Exception: Questions and
Answers Regarding the Scope of the Exception

The first draft guidance document describes several expedited programs that are available to sponsors of regenerative medicine therapies, and information about the requirements for, and benefits of, the new RMAT designation program that was created by the 21st Century Cures Act. This guidance has a 90-day comment period:

Expedited Programs for Regenerative Medicine
Therapies for Serious Conditions
 

The second draft guidance, published as a requirement of the 21st Century Cures Act, provides the agency’s current thinking about the evaluation of devices used in the recovery, isolation and delivery of RMATs.  This guidance has a 90-day comment period:

Evaluation of Devices Used with Regenerative
Medicine Advanced Therapies

Regulatory News

ASGCT a Stakeholder at Cell Therapy Liaison Meeting

Updated: January 30, 2018

The International Society for Cellular Therapy annually holds meetings between the FDA Center for Biologics Evaluation and Research and over 20 invited stakeholder organizations. ASGCT had two representatives at the Cell Therapy Liaison Meeting in Bethesda, MD. These meetings enable the cell therapy community to discuss specific issues of concern with CBER and provide updates on recent developments in field. A meeting summary will be posted here when available.

FDA CBER Issues 2018 Guidance Agenda

Updated: January 19, 2018

The FDA Center for Biologic Evaluation and Research (CBER) issued a statement of the guidance documents that it intends to issue in 2018, which includes up to six guidance documents on gene therapy. All of the gene therapy topics would be new guidance documents, and will likely include:

  • Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus During Product Manufacture and Patient Follow-Up
  • Observing Subjects Who Received Gene Therapy Products for Delayed Adverse Events
  • Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug (IND) Applications
  • Gene Therapy for the Treatment of Hemophilia

The agency also intends to issue two additional clinical draft guidance documents on the development of gene therapy products.

FDA Orphan Products Grants Program Funding of Rare Disease Natural History Studies

Updated: October 10, 2017

For the first time, on October 7 the FDA awarded six new research grants for natural history studies in rare diseases to conduct rare disease natural history studies, through its Orphan Products Grants Program. The FDA is providing a total of $6.3 million over the next five years to fund four natural history studies and, through a partnership with NCATS Therapeutics for Rare and Neglected Diseases program, the FDA received $3.5 million to be combined with FDA funding to fund an additional two studies.

Such research can inform clinical trial development, and may lead to the use of natural history models to augment or replace placebo arms in studies of therapies for very rare diseases, for which trial recruitment can be difficult and for which withholding treatment may pose ethical concerns.

The grants support research on Friedreich’s ataxia; pregnancy and lactation-associated osteoporosis; sarcoidosis; sickle cell anemia to determine biomarkers of endothelial function changes in chronic kidney disease; Angelman syndrome; and myotonic dystrophy type 1 to determine biomarkers and clinical endpoints.  

Government Relations Publications

Read recent ASGCT publications including the white paper on gene editing and our response to the National Center for Advancing Translational Sciences request for information.

Legislative Updates

Learn about recent legislative issues that ASGCT is monitoring or taking action on and read more about regularoty changes that effect the field of gene and cell therapy. 

Recent Collaborative Government Affairs Actions

Learn about ASGCT's most recent legislative actions and join our collaborative efforts. 

2018
21st Annual Meeting
May 16 - 19 | Chicago
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