The Center for Biologics Evaluation and Research (CBER) is a division within the U.S. Food and Drug Administration (FDA). Its mission is to protect and enhance public health by ensuring the safety, purity, potency, and effectiveness of biological products. This oversight covers products for preventing, diagnosing, and treating human diseases, conditions, or injuries. CBER also safeguards the public against emerging infectious diseases and potential bioterrorism threats.
Products Approved by CBER
CBER regulates a diverse range of biological products, which differ from chemically synthesized drugs as they are derived from living sources like humans, animals, or microorganisms. These products are manufactured using biotechnology. CBER’s regulatory authority extends to several categories, including:
Vaccines, which stimulate an immune response to protect against specific diseases.
Blood and blood components, such as whole blood, plasma, and red blood cells.
Plasma-derived products, including immune globulins and clotting factors.
Gene therapy products, which treat diseases by modifying or introducing genetic material into a patient’s cells.
Cellular therapy products, involving the administration of cells for therapeutic effects, often in regenerative medicine.
Human cells, tissues, and cellular and tissue-based products (HCT/Ps), intended for implantation, transplantation, infusion, or transfer into a human recipient.
Allergenic extracts, used for diagnosing and treating allergies.
Antitoxins and venoms, used to neutralize toxins or venoms from animal bites or stings.
Some medical devices associated with the collection, processing, and administration of blood and tissue products.
Navigating the CBER Approval Process
The journey to CBER approval begins with preclinical research, where a biological product is studied in laboratory and animal models to assess its potential safety and efficacy. This initial stage gathers data on the product’s chemistry, manufacturing, and controls, as well as its pharmacological effects and potential toxicity. The goal is to determine if the product is safe enough to proceed to human testing.
If preclinical data show promise, the next step involves submitting an Investigational New Drug (IND) application to CBER. This application is a request for authorization to administer the investigational biological product to humans in clinical trials. CBER reviews the IND to ascertain if the product is reasonably safe for human testing before allowing clinical trials to commence, often within 30 days of submission if no issues are raised.
Clinical trials are conducted in phases, each designed to gather specific information. Phase I trials typically involve a small group of healthy volunteers to evaluate the product’s safety, dosage range, and how it is metabolized and excreted. Phase II trials enroll a larger group of patients with the target condition to assess effectiveness and further evaluate safety. Phase III trials are large-scale studies involving hundreds to thousands of patients, comparing the investigational product to existing treatments or a placebo to confirm effectiveness and monitor for adverse reactions.
Upon successful completion of clinical trials, a manufacturer submits a Biologics License Application (BLA) to CBER. The BLA is a request for permission to introduce the biological product into interstate commerce. This application includes all preclinical and clinical data, detailed information on manufacturing processes, product characteristics, and proposed labeling.
CBER reviews the BLA with a multidisciplinary team of experts, including physicians, biologists, chemists, and statisticians. This review evaluates the product’s safety, purity, and potency, ensuring it meets established standards and that benefits outweigh risks for its intended use. Following approval, ongoing post-market surveillance monitors the product’s safety and effectiveness once available to the public.
Why CBER Approvals Matter
CBER’s regulatory oversight plays a role in public health and safety by establishing standards for biological products. The approval process ensures these complex therapies are evaluated for safety, effectiveness, and manufacturing quality before reaching patients. This evaluation helps prevent harmful or ineffective products from entering the market.
CBER’s review builds public trust in medical treatments. When a biological product receives CBER approval, it signals that the product has undergone scientific scrutiny and meets regulatory benchmarks. This assurance allows patients and healthcare providers to have confidence in the therapies they use.
CBER approvals encourage innovation within the biotechnology sector. By setting clear requirements for product development and data submission, CBER guides manufacturers toward producing safe and effective biologics. This regulatory framework supports the advancement of new therapies while maintaining public health safeguards, balancing innovation with patient protection.