Page 12 - HFA Dateline 2020-2021 Special Issue

A LOOK AT FDA’S GENE AND

CELL THERAPY FRAMEWORK

and its Impact on New Hemophilia

Treatments in 2021 and Beyond

BY ANGELA N. JOHNSON, PH.D., RAC, CPGP
SENIOR DIRECTOR OF REGULATORY AFFAIRS AT SIGILON THERAPEUTICS IN CAMBRIDGE,
MASSACHUSETTS, AND REGULATORY STRATEGY LECTURER AT NORTHEASTERN UNIVERSITY IN BOSTON

More cell and gene therapy as genetically modified factor- of guidance documents as a
products are being developed producing cells do not involve playbook of how to do it in most,
and entering clinical trials each changes in patient genetic but not all, cases.
year. The U.S. Food and Drug material and may allow better
Administration plays a key role control of dosing as well as Guidance frameworks are groups
in overseeing drug development, redosing. But many challenges of related guidance documents,
including providing guidance and uncertainties face researchers such as those relating to CGT. New
and receiving investigational new and drug developers. To help set guidance creation is an important
drug applications or requests best practices across the industry, part of FDA’s ongoing mission to
to start a new clinical trial FDA guidance frameworks play expedite innovations that make
submitted by drug developers. an important role in safe and medical products more effective,
efficient development of CGT safer and more affordable.
In 2021, there are more than products. Guidance documents are written
1,000 cell and gene therapy and published according to a
clinical trials, including more WHAT ARE FDA GUIDANCE process called Good Guidance
than a dozen in hemophilia. FDA FRAMEWORKS? Practice, which describes how
announced it expects more than To help the companies FDA staff will bring together
200 new requests to start clinical developing new drugs, FDA expert and public feedback in
trials based on gene and cell publishes recommendations guidance recommendations.
technologies each year. By called guidance documents.
2025, FDA expects 10 to Unlike the laws passed GROWING EXPECTATIONS FOR
CGT IN HEMOPHILIA
20 new CGT treatments by Congress or formal FDA’s first draft guidance
will be approved annually. regulations, guidance document dedicated to
documents contain FDA
Gene therapy treatments expectations and current early development of CGT
for hemophilia have shown scientific thinking not required clinical trials — Guidance for
potential to eliminate the need by law. If we think of regulations Industry: Considerations for
for prophylactic factor infusions as what the law requires drug the Design of Early-Phase
and injections. Unlike gene developers to do, we can think Clinical Trials of Cellular and
therapy, newer technologies such Gene Therapy Products — was

12 DATELINE FEDERATION < www.hemophiliafed.org