Despite pandemic, CBER hit FY20 milestones

| 09 February 2021 |  By 

Peter Marks, MD, PhD

The US Food and Drug Administration’s Center for Biologics Evaluation and Research (FDA’s CBER) met productivity milestones and continued product safety and biostatistics work, despite the pandemic disruptions of 2020. An annual report highlighting the fiscal year’s work, released 4 February, was accompanied by remarks from CBER director Peter Marks, MD, PhD.
Work plans for 2020 were upended as the COVID-19 pandemic swept the globe, said Marks. “This response has involved every product office and every support office across the Center. It involved rapidly developing policy, drafting needed guidance documents, and providing advice on and evaluating Investigational New Drug applications for hundreds of medical products targeting treatment or prophylaxis.”
Not least among CBER’s efforts has been work related to development and evaluation of vaccines against COVID-19. The office issued two vaccine product development guidances and closed out the calendar year by issuing emergency use authorizations (EUAs) for the two messenger RNA (mRNA) vaccines sponsored by Pfizer-BioNTech and Moderna. These authorizations came just one week apart, on 11 and 18 December, noted Marks, calling the year-end work a “remarkable achievement.”
In August, CBER also issued an EUA for convalescent plasma to treat hospitalized patients with COVID-19; other COVID-19 directed work included collaboration in the cross-agency efforts of the Coronavirus Treatment Acceleration Program (CTAP). Additionally, noted the report, FDA continues to collaborate with industry and other government agencies to make SARS-CoV-2 human immune globulin available for investigations into therapeutics. CBER also helped facilitate Mayo Clinic’s COVID-19 convalescent plasma expanded access program that eventually delivered the treatment to about 100,000 patients.
Still, Marks said, CBER took important steps forward in non-pandemic areas, amid the transition to working from home and all-virtual meeting formats. Marks pointed to the five novel biologics licensed by CBER; among these were Palforzia [Peanut (Arachis hypogaea) Allergen Powder-dnfp, Aimmune], the oral immunotherapy for peanut allergy, and Tecartus (brexucabtagene autoleucel, Kite), the CD19-directed genetically modified autologous T cell immunotherapy for adults with relapsed or refractory mantle cell lymphoma.
Taken together with the licensing of three blood grouping reagents, “These approvals are part of the overall success CBER had in FY 2020 in achieving or exceeding expectations” as laid out in the Prescription Drug User Fee Act VI (PDUFA VI)and Medical Device User Fee Amendments (MDUFA), noted Marks.
At the end of CBER’s 2020 fiscal year, CBER had received one standard review and six priority review biological license applications (BLAs); one has been approved and all other reviews are expected to be completed by goal dates. Seven PDUFA BLA original applications came to CBER in FY 2019; the two standard review timeline applications have been approved, as have three of the priority review applications. The other two priority review BLAs received Complete Response Letters (CRLs).
In terms of MDUFA goals, CBER reached or exceeded its performance targets for 510(k) submissions, real-time premarket assessment (PMA) supplements, and PMA 180-day supplements.
In its mission to meet deliverables set forth under the 21st Century Cures Act, CBER continued its support of the Complex Innovative Designs pilot program and continued its work in the areas of advanced manufacturing and regenerative medicine and advanced therapies.
“As part of its effort to facilitate the development and approval of safe and effective products, CBER works to identify stakeholder priorities and incorporate improved approaches to both research and regulatory science,” noted the agency in the report. The newly established Advanced Technologies Team (CATT), wrote CBER, has a goal of promoting “a more robust manufacturing process with fewer interruptions in production, fewer product failures, and greater assurance that the biologic products will meet clinical performance expectations.” The advanced technolgoies program awarded several grants, continued interface with developers of advanced therapies, and continued its support of extramural projects looking into improvements to advanced manufacturing techniques.
In addition to the significant new products called out by Marks, the report outlines additional activities of CBER’s individual offices. The Office of Vaccines Research and Review, for example, also approved a new meningococcal conjugate vaccine and a new adjuvanted influenza A vaccine.
In addition to its review of Tecartus, the Office of Tissues and Advanced Therapies approved a new recombinant Factor VIIa product for individuals with hemophilia A and B who have inhibitors. The Office of Blood Research and Review also approved a genotyping assay to detect HIV-1 genomic mutations and a new multiplex assay for HIV types 1 and 2 and Treponema pallidum, which causes syphilis.
CBER made contributions to agency-wide efforts to enhance engagement with patients, including webinar presentations and helping to plan and present a public workshop on patient-focused drug development.
During 2020, CBER officials made liberal use of warning letters and untitled letters to help forestall unapproved use of such biological products as exosome, adipose, and umbilical cord products, as well as an unauthorized vaccine. However, CBER noted in the report, FDA has generally been exercising enforcement discretion for certain uses of human cells, tissues and cellular and tissue-based products.
The report details the public meetings held by CBER during the course of the year, including collaborative international meetings, workshops for developers, and several meetings of the Vaccines and Related Biological Products Advisory Committee.
CBER’s Office of Biostatistics and Epidemiology continued enhancements of its post-market surveillance infrastructure including the Biologics Effectiveness and Safety (BEST) system, which the office tested with a vaccine study. Other surveillance efforts included ongoing monitoring of transfusion-transmitted infections and work with the Centers for Medicare and Medicaid Services to track vaccine safety for older adults.

The report closes by citing 10 representative research publications authored or co-authored by CBER staff; the work ranges across a variety of scientific fields from COVID-19-related work to adenovirus vector research to epidemiological research into influenza vaccine effectiveness.
CBER FY20 annual report


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Tags: coronavirus, FDA, US

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