News
Analysis Group Researchers Support FDA BLA Approval of a Regenerative Medicine Product Using Novel External Benchmarking Statistical Method
Analysis Group researchers played a pivotal role in devising a novel statistical approach to compare our client’s single-arm clinical trials to literature-derived external benchmarks, leading to a successful bid for US Food and Drug Administration (FDA) approval of a regenerative medicine product using bioengineered human tissue technology.
Due to its potential to address a significant unmet medical need, this treatment received FDA Priority Review and was designated as a priority product by the US Department of Defense. In addition, it received the FDA’s regenerative medicine advanced therapy (RMAT) designation, reserved for cutting-edge and life-saving innovations in cell therapy and tissue engineering.
As part of this novel treatment’s Priority Review application, researchers evaluated its safety and efficacy in a prospective, single-arm, multicenter clinical trial. Additionally, real-world evidence (RWE) was gathered from patients with wartime injuries, with whom the treatment was deployed under a humanitarian use program. Since these studies did not include a direct comparison group, an Analysis Group team led by Chief Epidemiologist and Managing Principal Mei Sheng Duh and Vice Presidents Priyanka Bobbili and Marianne Cunnington assisted our client in designing an external literature-based comparator, derived from conducting meta-analysis of the existing literature on synthetic comparator products, with a confidence interval-based benchmarking method to support the assessment of the clinical performance of the treatment.
The results are presented in an article published in JAMA Surgery in November 2024.
Associated People
Marianne Cunnington
Managing PrincipalDr. Cunnington specializes in regulatory epidemiology, including the design and execution of real-world post-marketing safety and effectiveness studies, as well as real-world studies to support the approval of new products through innovative regulatory pathways. Her research expertise includes design, negotiation, and execution of real-world components of risk management plans for new product submissions with regulators across multiple geographies (US, UK, Europe, Japan, South Korea); post-approval safety studies in the EU; post-marketing requirement and commitment studies in the US; and pregnancy registries. Prior to joining Analysis Group, Dr. Cunnington spent more than 20 years as a pharmaceutical industry epidemiologist focusing on the generation of real-world evidence (RWE) to support the development and life cycle management of vaccines and pharmacological therapies. Dr. Cunnington held several senior epidemiology positions within Novartis and GlaxoSmithKline (GSK). At GSK, she chaired the peer review forum for all pharmaceutical RWE projects, provided epidemiological expertise for the company’s Global Safety Board, and was a managing board member of the Innovative Medicines Initiative’s public-private partnership, building an integrated European real-world data network to monitor medication safety in pregnancy. Dr. Cunnington serves on the board of the International Society for Pharmacoepidemiology as an elected representative of European industry and service providers.