CBER Plots New Draft Guidance in 2020 on Genome Editing, CAR-T Therapies

Regulatory NewsRegulatory News | 14 February 2020 |  By 

The FDA’s Center for Biologics Evaluation and Research (CBER) unveiled its 2020 guidance list on Friday, with new drafts coming on several increasingly crowded areas for drug development.

Three new drafts this year will likely draw lots of comments from industry and other stakeholders. They deal with considerations for the development of Chimeric Antigen Receptor (CAR) T-cell therapies, considerations for the development of human gene therapies incorporating genome editing, and one on human gene therapies for neurodegenerative diseases.

FDA late last month also finalized six guidance documents on gene therapies and released one new draft for comment on interpreting the sameness of gene therapies under the orphan drug regulations.

Other new draft guidance from CBER in 2020 will focus on blood pressure and pulse donor eligibility requirements, as well as alternative procedures for cold-stored platelets intended for transfusion.

Final guidance expected this year will deal with a draft from last September on interacting with FDA on complex and innovative trial designs for biologics and a draft from December 2017 on chemistry, manufacturing, and controls changes to an approved biologic.

A final guidance on the automated collection of platelets is also expected and CBER says, “We intend to issue a Level 2 guidance to revise existing recommendations to address statistical sampling plans for process validation.”

Other final guidance expected in 2020 includes one on “Revised Recommendations for Biological Product Deviation Reporting for Blood and Plasma Establishments” and one on, “Testing for Biotin Interference in In Vitro Diagnostic Devices," drafted last June.

CBER Guidance Agenda for 2020


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