CDER, CBER, and the Oncology Center of Excellence recently published a final guidance document titled “Considerations for the Use of Real-World Data and Real-World Evidence to Support Regulatory Decision-Making for Drug and Biological Products” as another part of its real-world evidence (“RWE”) Program. This finalized …
In May 2023, CDER and CBER published a draft guidance titled “Pediatric Drug Development: Regulatory Considerations – Complying with the Pediatric Research Equity Act [“PREA”] and Qualifying for Pediatric Exclusivity Under the Best Pharmaceuticals for Children Act [“BPCA”]” (the “Draft Guidance”). This Draft Guidance, in …
On May 2nd, FDA released a new draft guidance with recommendations for decentralized clinical trials (DCTs) for drugs, biologics, and devices. A DCT is a clinical trial where some or all of the trial-related activities, such as administration of the investigational product, data collection, or …
The Draft Guidance In February 2023, CDER, CBER, and the Oncology Center of Excellence published a draft guidance titled “Considerations for the Design and Conduct of Externally Controlled Trials for Drug and Biological Products” (the “Draft Guidance”) to provide recommendations to those considering the use of …
Hyman, Phelps & McNamara, P.C.’s Mark Tobolowsky co-authored the peer-reviewed article “Microdystrophin Expression as a Surrogate Endpoint for Duchenne Muscular Dystrophy Clinical Trials” in the recently published edition of Human Gene Therapy. The article arose out of work conducted by the Pathway Development Consortium, a …
Incorporating patient and caregiver experiences into every phase of drug development has become increasingly prioritized during both development and review (see, e.g., previous coverage here). Who better than people living with a condition to inform drug companies, physicians, academics, and the FDA on what it …
Amongst the many provisions that Congress included in the recent Food and Drug Omnibus Reform Act (“FDORA”) were two subtle changes – one change to the Federal Food, Drug, & Cosmetic Act’s (“FD&C Act”) requirements for advancing an investigational new drug into clinical trials and …
Last month, Congress took a big step towards improving clinical trial diversity by requiring sponsors of most drug and device clinical studies to submit a diversity action plan when they submit key trial documents to the Food and Drug Administration (FDA). This requirement, enacted under …
On December 7, 2022, FDA’s Center for Biologics Evaluation and Research (CBER) and the Office of Tissues and Advanced Therapies (OTAT) held a town hall to answer questions related to cell therapy and tissue-engineered products chemistry, manufacturing, and controls (CMC). The purpose of these town …
Hosted by American Conference Institute, the FDA Boot Camp returns for its 40th iteration with the continued intent of providing an essential working knowledge of core FDA concepts, and real-world examples that will help you to excel in your everyday practices. This year’s conference co-chairs include …
On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes. …
On September 30, 2022, President Biden signed into law the Continuing Appropriations and Ukraine Supplemental Appropriations Act, 2023, Division F of which is titled the “FDA User Fee Reauthorization Act of 2022” (“FUFRA”). In addition to reauthorizing for an additional five fiscal years—Fiscal Years 2023-2027—several …
On October 26, 2022, US Pharmacopeia (USP) Biologics Stakeholder Forum held a workshop on “Collaborating to solve CMC challenges and support efficient development of lentiviral-mediated CART cell therapies.” Panelists discussed CMC challenges for CART therapies, including potency assay development, analytical method transfer, and demonstrating comparability. …
FDA recently published the final guidance document “Comparability Protocols for Postapproval Changes to the Chemistry, Manufacturing, and Controls Information in an NDA, ANDA, or BLA.” This final guidance provides recommendations to original applicants and holders of approved applications for human drugs and certain biological products …
During last year’s 2021 Pharmaceutical Quality Symposium, CDER described the development of a regulatory framework called FRAME –Framework for Regulatory Advanced Manufacturing Evaluation, to accommodate innovation expected in the next 5-10 years with a focus on 4 technologies: End to End Manufacturing, Distributed Manufacturing, Point-Of-Care, …
