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CY 2024 Report from the Director

 

Dr. Peter Marks photograph

Throughout 2024, the Center for Biologics Evaluation and Research (CBER) built on the previous year’s momentum created by advancements in science and technology supporting the development of innovative products. CBER achieved significant accomplishments advancing health in this country and globally. CBER approved multiple products, including several that offer potential treatments for individuals with certain rare diseases. Additionally, the Center advanced important public health priorities in blood and tissue safety and approved several important preventative vaccines.  

CBER remained committed to working closely with patients, researchers, technology developers, and product developers, providing our current thinking and recommendations related to the data needed to support the clinical development and approval of safe and effective products. This included noteworthy advances in the area of rare disease. On May 29, 2024, the FDA notified selected participants of their acceptance into the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. Two months later, CBER announced jointly with the Center for Drug Evaluation and Research (CDER) the FDA Rare Disease Innovation Hub that will leverage cross-agency expertise with the goal of greatly enhancing the development of treatments for rare diseases, with a particular focus on products intended for smaller populations or for diseases where the natural history is variable and not fully understood. The development of therapies for these conditions can be particularly challenging. Throughout the year, the Rare Disease Endpoint Advancement (RDEA) Pilot Program continued to support novel endpoint efficacy development for drugs and biologics that treat rare diseases.  Additionally, the collaborative effort with our global regulatory partners will continue to advance in 2025 with the Collaboration on Gene Therapies (CoGenT) Global Pilot.

Strategic planning is essential for maintaining and further strengthening CBER’s strong support and focus to achieve our mission and vision. A few examples of how in 2024 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 significant milestones with the approval of 24 biological device applications, 17 biologics license applications and 26 biologics license application supplements. A list of the calendar year 2024 biological product approvals and supporting documentation is posted on CBER’s website. Numerous noteworthy supplement approvals reflect examples of the depth of continued updates to the use of previously approved products.

In September, CBER and CDER made available a draft Strategy Document which outlines the specific activities FDA intends to undertake to facilitate the use of innovative manufacturing technologies. Specifically, under the draft strategic plan FDA intends to undertake the following activities: continue to enhance the CBER Advanced Technologies Team (CATT) and CDER Emerging Technology Program (ETP) as a mechanism to support innovation; implement the Advanced Manufacturing Technologies Designation Program (AMTDP) in a manner that reflects feedback on eligibility criteria; continue to identify opportunities for international harmonization in support of advanced manufacturing; support and utilize ongoing initiatives for advanced manufacturing to address potential barriers; and support training in advanced manufacturing for FDA assessment staff.

CBER continued its focus on facilitating development of cell and gene therapies through numerous public town halls, webinars and a workshop along with the publication of several guidance documents.  The public meeting included important topics such as best practices for interactions with FDA, readiness for Chemistry, Manufacturing and Controls submissions for gene therapy BLAs, and the clinical use of integration site analysis (ISA) during long term follow-up following administration of gene therapies with integrating viral vectors.

In addition to the portfolio of product approvals this year, CBER continued its patient engagement efforts, with a focus on gene therapy and rare diseases. On September 20, 2024, and December 4, 2024, CBER held public listening meetings with patients with rare genetic diseases and their care partners. At these meetings CBER heard patient and care partner perspectives on the short-term and long-term risks of approved gene therapy products, information needed to make treatment decisions about approved gene therapies, participation in long-term post-market studies, and enrolling in a gene therapy clinical trial when a patient is in the presymptomatic or early stage of a rare genetic disease. On October 16, 2024, CBER and CDER, in collaboration with the Reagan-Udall Foundation for the FDA, held a public meeting to bring together rare disease patient advocates, academic researchers, regulated industry, and other key stakeholders to discuss how the Rare Disease Innovation Hub can best engage with members of the rare disease community and prioritize its work. In 2024, CBER participated in 35 patient focused drug development meetings and patient listening sessions, as we redoubled our commitment to helping bring advances to patients and to incorporating patient involvement in the center's regulatory work.

CBER  issued important safety communications to consumers, patients and healthcare providers including but not limited to the following topics:  amniotic fluid eyedrops; a required boxed warning for T cell malignancies following treatment with BCMA-Directed or CD19-Directed autologous chimeric antigen receptor (CAR) T cell immunotherapies; use of unapproved HIV blood sample self-collection kits; and modifications to the Risk Evaluation and Mitigation Strategy (REMS) for these CAR T cell immunotherapies to minimize the burden of complying with the REMS on the healthcare delivery system. Additionally, CBER provided information for blood establishments and tissue establishments regarding the Oropouche virus along with reminding the public that FDA has not approved “young plasma” for any medical conditions or to provide other health and wellness benefits. Information for blood establishments and transfusion services for strengthening their cybersecurity practices to prevent and mitigate cybersecurity incidents that could affect the availability and safety of blood and blood components for transfusion or further manufacture was also made available on FDA’s web site.  

Guidance development and publication continues to offer CBER’s current thinking on important product design, manufacturing and testing of many regulated products and in 2024, 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’s Office of Therapeutic Products (OTP) hosted eight events to share information about the review of regulated products and to bring together important stakeholders to discuss the development and advancement of products, including CAR-T cells, regenerative medicine, gene therapy, and provide a forum for clinical trial participants to learn about finding a support team as they take part in a study. OTP hosted two listening sessions for patients and their care partners perspectives on gene therapy for rare diseases.

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

The science that underpins the safety, efficacy, and quality of new biological products is constantly evolving. The combination of bench research and regulatory expertise enables CBER scientists to tackle product development roadblocks in the lab, design important new approaches and tools to speed up product development, review product submissions very efficiently and effectively, make regulatory decisions informed by state-of-the-art science, and develop sound evidence-based policy. CBER conducts scientific research of the highest quality and relevance that is integral to the Center's regulatory mission and public health portfolio, that is proactive and anticipates regulatory issues and public health needs, and that directly supports regulatory decision-making and policy development responsibilities.

CBER’s 60 laboratory research programs, directed by principal investigators who conduct both product-relevant research and review product-related information in regulatory submissions, published more than 120 papers in peer-reviewed journals in CY 2024. Moreover, our pharmacovigilance and statistical scientists provided over 40 published reports of safety and efficacy analyses related to use of CBER-regulated products, as well as reports that described improved methods to leverage clinical trial data.


Increase preparedness for emerging threats and promote global public health

In June 2024, the Vaccines and Related Biological Products Advisory Committee (VRBPAC) met to discuss and make recommendations on the selection of strains to be included in the 2024-2025 Formula for COVID-19 vaccines. Two months later, CBER approved and authorized the updated COVID-19 vaccines to more closely target currently circulating variants and provide better protection against serious consequences of COVID-19, including hospitalization and death. Additionally, in December, CBER approved COVID-19 Convalescent Plasma with high titers of anti-SARS-CoV-2 antibodies for the treatment of COVID-19 in patients with immunosuppressive disease or receiving immunosuppressive treatment.

CBER has reviewed over 217 EUA amendments to authorized COVID-19 vaccines and 174 BLA amendments for licensed COVID-19 vaccines. In CY 2024, eight of CBER’s laboratory research programs continue to include studies related to COVID-19, resulting in approximately 15 publications. The Center continues to monitor the safety of the COVID-19 vaccines and other COVID-19-related biological products through passive and active safety surveillance systems using state-of-the-art technologies, providing over 20 published reports of such analyses in CY 2024.

CBER remains vigilant monitoring H5N1 and continues preparations in the event that a vaccine response is required. In October 2024, the VRBPAC discussed and made recommendations on the strain selection for the influenza virus vaccines for the 2025 Southern hemisphere influenza season and pandemic preparedness for highly pathogenic avian influenza virus including considerations for vaccine composition for H5 vaccines.

Manage for strategic excellence and organizational accountability

Throughout CBER there has been work over the past year to make a variety of business processes more efficient. For example, CBER's Office of Therapeutic Products was reorganized into a super office in August of 2023 in order to improve discipline alignment, increase review capacity, and enhance expertise in cell and gene therapies; we implemented these changes throughout 2024. These structural changes have helped support the growing field of cell and gene therapies, with ten new cell and gene therapies for rare diseases approved since the reorganization, approximately twice the rate of previous years’ approvals.  

As CBER’s significant and enduring contributions to public health for over 120 years continues, we look forward to our ongoing work with sponsors, patients and others to help facilitate the timely development and availability of important products for patients with unmet medical needs while maintaining our strong focus on the Center’s mission.


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

 

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