Pharmaceutical Technology REGULATORY SOURCEBOOK MARCH 2021 9
plications. Some noteworthy guidance documents
include the following:
• Interpreting Sameness of Gene Therapy Products
Under the Orphan Drug Regulations; Guidance
for Industry
• Considerations for the Development of Human
Gene Therapy Products Incorporating Genome
Editing; Draft Guidance for Industry
• Considerations for the Development of Chimeric
Antigen Receptor (CAR) T Cell Therapies; Draft
Guidance for Industry.
COVID-19 guidance
The first quarter of 2021 saw the agency publish
guidance on a variety of subjects, with treatments
and products to address COVID-19 receiving a good
deal of the agency's attention. In March 2021, FDA
published guidance providing recommendations for
reporting and implementing changes to COVID-19
container closure system (CCS) components that con-
sist of glass vials and stoppers for parenteral sterile
drug products and biologics (4). The guidance dis-
cusses regulatory approaches to CMC changes and
common changes to glass vials and stoppers. The
guidance also discusses risk-based tools for imple-
menting CCS changes and comparability protocols.
FDA announced on Feb. 22, 2021 that it issued a
suite of guidance documents to address the emer-
gence of variants of SARS-CoV-2. The guidance
documents include an update to the October 2020
guidance on Emergency Use Authorization (EUA)
for vaccines to prevent COVID-19, guidance on the
impact of the variants on monoclonal antibody (mAb)
products and other treatments, and a guidance the
impact of the variants on COVID-19 tests (5). The
updated guidance, Emergency Use Authorization for
Vaccines to Prevent COVID-19, offers recommenda-
tions to vaccine developers regarding virus variants,
including developers who already received EUA for
their vaccine. The agency stated, in a press release, that
FDA-authorized vaccines are effective against the cur-
rent strains of SARS-CoV-2, but it may be necessary
to tailor vaccines should a new variant emerge in the
United States that is "moderately or fully resistant to
the antibody response elicited by the current genera-
tion of COVID-19 vaccines."
On Feb. 11, 2021, FDA published guidance that
gives recommendations to health care providers and
investigators on the use of COVID-19 convalescent
plasma or investigational convalescent plasma (6).
Recommendations are also provided regarding blood
collection. The document also discusses the agency's
interim compliance and enforcement policy for in-
vestigational new drug requirements for the use of
investigational convalescent plasma. The new guid-
ance, which supersedes guidance issued in January
2021 and previous versions, includes revised recom-
mendations for convalescent plasma donors. "The re-
visions address when individuals who have received
an investigational COVID-19 monoclonal antibody
therapy as a participant in a clinical trial, or received
an authorized or licensed COVID-19 monoclonal an-
tibody therapy, qualify as convalescent plasma donors,"
the guidance states.
References
1. J. Wechsler, PharmTech 45 (3) 2021.
2. CDER, CDER Guidance Agenda New & Revised Draft Guidance
Documents Planned for Publication in Calendar Year 2021 (Janu-
ary 2021).
3. CBER, Guidance Agenda: Guidance Documents CBER is Plan-
ning to Publish During Calendar Year 2021.
4. FDA, COVID-19 Container Closure System and Component
Changes: Glass Vials and Stoppers, Guidance for Industry (CBER,
CDER, March 2021).
5. FDA, "Coronavirus (COVID-19) Update: FDA Issues Policies to
Guide Medical Product Developers Addressing Virus Variants,"
Press Release, Feb. 22, 2021.
6. FDA, Investigational COVID-19 Convalescent Plasma, Guidance
for Industry (CBER, Feb. 11, 2021). PT
