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  1. Vaccines, Blood & Biologics

CY 2023 Report from the Director

 

Peter Marks

Calendar year 2023 contained many achievements that have had a positive effect on public health. Indeed, CBER made significant strides with the approval of multiple important therapeutic products and advanced important public health priorities in blood and tissue safety and approved several important vaccines.  Additionally, cell and gene therapies represent cutting-edge technologies that may offer the most effective means to treat illnesses and conditions that presently are addressed by few - or no other - treatment options and have become an important focus for our work.  With continuing advancements in science and technology supporting the development of innovative products CBER regulates, we expect to build on this momentum in 2024.

We remain committed to working closely with patients, researchers, technology developers, and sponsors, and to providing our current thinking and recommendations related to the data needed to support the clinical development and approval of safe and effective products. The groundwork related to additional support efforts for products to treat rare diseases is underway through programs such as the Support for Clinical Trials Advancing Rare Disease Therapeutics Pilot Program (START) and Rare Disease Endpoint Advancement Pilot Program (RDEA). We are also considering how we can work with sponsors to leverage data about platform technologies for cell and gene therapies and implement the statutory provisions related to the accelerated approval pathway in a manner that expedites the development of innovative, safe, and effective treatments for patients.

In addition, we believe that global harmonization efforts related to the evaluation and regulation of cell and gene therapy products can help facilitate more efficient clinical development for these products and potentially for other product categories too. Toward that end, FDA supports efforts toward global regulatory convergence and, ultimately, global harmonization of regulations. A collaborative effort with our global regulatory partners will continue to advance forward in 2024 with the Collaboration on Gene Therapies (CoGenT) Global Pilot.

A few examples of how in 2023 CBER continued to follow the path of our 2021–2025 Strategic Plan:

Facilitate the development and availability of safe and effective medical products through the integration of advances in science and technology.

CBER achieved a significant milestone with the approval of 23 biologics device applications, 20 biologics license applications and 17 biologics license application supplements. A list of the 2023 biological product approvals and supporting documentation is posted on CBER’s website.

Noteworthy approvals and device clearances include:

  • The first gene therapy treatment produced using genome editing was approved for use in patients with severe sickle cell disease for the treatment of sickle cell disease in patients 12 years and older with recurrent vaso-occlusive crises.
  • The approval of a recombinant protein product indicated for prophylactic or on-demand enzyme replacement therapy for patients with congenital thrombotic thrombocytopenic purpura.
  • The first chikungunya vaccine was approved for use in individuals 18 years of age and older who are at increased risk of exposure to chikungunya virus.
  • The first respiratory syncytial virus (RSV) vaccine approved for use was approved for the prevention of lower respiratory tract disease (LRTD) caused by RSV in individuals 60 years of age and older and the first vaccine approved for use in pregnant individuals to prevent LRTD and severe LRTD caused by RSV in infants from birth through 6 months of age.
  • The clearance of a 510(k) an anti-cytomegalovirus (CMV) in vitro immunoassay on a fully automated high throughput for the qualitative detection of antibodies to CMV in human serum and plasma.

In addition to the portfolio of product approvals this year, CBER's Office of Blood Research and Review (OBRR) and Office of Biostatistics and Pharmacovigilance (OBPV) issued updated guidance for assessing blood donor eligibility using a set of individualized risk-based questions to reduce the risk of transfusion transmitted HIV, standardizing questions for every donor, regardless of sexual orientation, or sex. The implementation of these recommendations represents a significant milestone for the agency and the LGBTQI+ community.

Engaging with patients and listening to their perspectives on their diseases and treatments is critical to helping the Center facilitate the development of medical products that make the biggest differences in patients’ lives. In 2023, CBER participated in 35 patient focused drug development meetings and patient listening sessions, in addition to four CBER led or co-led public meetings with patient groups. CBER made plans to redouble our commitment to helping bring advances to patients and to incorporating patient involvement in the center's regulatory work.

We announced the innovative Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program with our colleagues in the Center for Drug Evaluation and Research (CDER), along with a Request for Information for stakeholders to provide feedback regarding critical scientific challenges and opportunities to advance the development of cellular and gene therapies designed for an individual or very small number of patients diagnosed with a rare disease. CBER, jointly with CDER, continued to participate in the ongoing Rare Disease Endpoint Advancement (RDEA) Pilot Program.

CBER provided important safety communications to consumers, patients and healthcare providers including CBER’s investigation of a serious risk of malignancy following treatment with BCMA- or CD19-directed autologous CAR T cell immunotherapies and, jointly with FDA’s Center for Food Safety and Applied Nutrition (CFSAN), warned of the use of probiotics in preterm infants.

Additional guidance development and publication continues to provide CBER’s current thinking on important product design, manufacturing and testing of many regulated products and in 2023, CBER published numerous draft and final guidance documents regarding blood and blood components, cellular and gene therapy, tissues, vaccines and many other important regulatory topics.

CBER held numerous advisory committee meetings to obtain advice on important and challenging product review and approval issues and we are grateful for the feedback from members of CBER's various advisory committees.

Conduct research to address challenges in the development and regulatory evaluation of medical products.

CBER’s intramural research program continues its rich history of outstanding scientific accomplishment. We strive to conduct world-class scientific research related to biological product development that aligns with CBER’s regulatory mission, directly informing regulatory decisions and policy while advancing public health. Topics of investigation range from applied studies that target specific needs of product classes to foundational studies that anticipate future treatments and developments. Our 65 research programs, directed by scientists who both conduct product-relevant research and review product-related information in regulatory submissions, published approximately 150 papers in peer-reviewed journals in 2023. Moreover, our pharmacovigilance and statistical scientists provided over 50 published reports of safety and efficacy analyses related to use of CBER-regulated products, as well as described improved methods to leverage clinical trial data.

Increase preparedness for emerging threats and promote global public health.

In April 2023, FDA amended the emergency use authorizations (EUAs) for the COVID-19 bivalent mRNA vaccines to simplify the vaccination schedule for most individuals. Following the end of the COVID-19 public health emergency on May 11th, CBER focused on continuing to provide safe and effective vaccines to safeguard against COVID-19, which continues to impact the population. CBER has reviewed over 2000 EUA amendments to authorize COVID-19 vaccines and conducted over 40 COVID-19 laboratory research studies, resulting in more than 200 publications. In addition, the Center continues to monitor the safety of the COVID-19 vaccines through passive and active safety surveillance systems using state-of-the-art technologies.

Manage for strategic excellence and organizational accountability.

CBER underwent several important organizational updates during calendar year 2023 to address the continued substantial growth in innovative, novel products and an ever-changing landscape of potential public health threats. Most notably, the Center stood-up the Office of Therapeutic Products to further our commitment to advancing the timely development and availability of cell and gene therapies. CBER also implemented structural changes in the Office of Blood Research and Review and the Office of Vaccines Research and Review, the Office of Biostatistics and Pharmacovigilance and the Office of Regulatory Operations.  These organizational changes established a structure designed to enable CBER’s workforce to focus on key priorities that protect public health, improve operational efficiency, and increase professional development opportunities.

CBER aims to make 2024 a breakout year for addressing key challenges in the development of cell and gene therapies, especially for rare disorders. The Center is strongly committed to furthering these efforts through increased communications under the START Pilot, increasing efficiency of the regulatory process through the CoGenT Global Pilot, facilitating the use of accelerated approval of gene therapy based on surrogate endpoints that are reasonably likely to predict clinical outcomes, and issuing guidance to be transparent about our current thinking. Additionally, we will continue partnering with CDER to conduct the Chemistry, Manufacturing, and Controls (CMC) Development and Readiness Pilot (CDRP) Program to facilitate CMC development of selected products under investigational new drug (IND) applications with expedited clinical development timeframes to help patients gain access to these products earlier.

We look forward to continuing to work with sponsors, patients and others to help facilitate the timely development and availability of important products for patients with unmet medical needs.

 

Peter Marks, M.D., Ph.D.
Director
Center for Biologics Evaluation and Research 

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