CBER Plots 6 Gene Therapy Draft Guidances in 2018

Regulatory NewsRegulatory News | 22 January 2018 |  By 

The US Food and Drug Administration's (FDA) Center for Biologics Evaluation and Research on Monday unveiled the draft guidance documents it plans to publish in 2018, which will include as many as six documents on gene therapy.

All of the new draft guidance on gene therapy would be new for the agency and CBER said they are likely to include:
  • Testing of Retroviral Vector-Based Gene Therapy Products for Replication Competent Retrovirus during Product Manufacture and Patient Follow-up; Draft Guidance for Industry
  • Observing Subjects Who Received Human Gene Therapy Products for Delayed Adverse Events; Draft Guidance for Industry
  • Chemistry, Manufacturing, and Control (CMC) Information for Human Gene Therapy Investigational New Drug Applications (INDs); Draft Guidance for Industry
  • Gene Therapy for the Treatment of Hemophilia; Draft Guidance for Industry and “2 additional clinical draft guidance documents on the development of gene therapy products.”
Other new draft guidance planned for 2018 includes revised recommendations on reducing the risk of Zika virus transmission by blood and blood components and further testing of donations that are reactive on a screening test for antibodies to hepatitis C virus.

The total number of guidance documents planned for 2018 is on par with 2017.

New Standard Operating Policy and Procedure

In addition to the guidance plans, CBER also released two standard operating policy and procedure (SOPP) documents on the formal dispute resolution process and providing the public with notice of an order approving or denying a Premarket Approval Application (PMA) or Humanitarian Device Exemption (HDE) application.

The SOPP on disputes applies to those arising from unresolved disagreements between sponsors and CBER review staff regarding a sponsor’s pre-submission, investigational, marketing, or postmarking submission. But it does not apply to disputes that arise from scientific disagreements regarding the review of sponsor submissions.

Guidance Agenda: Guidance Documents CBER is Planning to Publish During Calendar Year 2018


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