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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.
Meet Our People
Marianne Cunnington
Vice PresidentDr. Cunnington has over 20 years of experience as a pharmaceutical industry epidemiologist. She specializes in the generation of real-world evidence (RWE) in support of the development and life cycle management of pharmaceutical and vaccine products, with a particular focus on regulatory risk management strategies and product benefit-risk assessments. Dr. Cunnington’s research expertise includes the design, negotiation, and execution of real-world components of risk management plans for new product submission to regulators in the US, UK, EU, Japan, and South Korea, including studies of post-approval safety in the EU; post-marketing requirements and commitments in the US; and pregnancy registries. Prior to joining Analysis Group, she served in several senior epidemiology positions at pharmaceutical companies, including as the chair of GSK’s peer review forum for all pharmaceutical RWE projects; as a member of GSK’s Global Safety Board; as head of epidemiology at Novartis Vaccines; and as a managing board member of Innovative Medicines’ public-private partnership initiative to build an integrated European real-world data (RWD) network to monitor medication safety in pregnancy.