Implications of Historical Control Studies for Rare Disease Products
Development programs in rare diseases are becoming more common and a major focus for small biotech and pharmaceutical companies. A crucial consideration in clinical study and protocol design in these indications is the choice of control group used to assess the efficacy of an intervention. Standard study designs and control are often not feasible due to limitations in the number of prospective patients as well as ethical considerations. Non-standard study designs, including studies relying on external historical control groups, can be used to evaluate the efficacy of an intervention. This webinar will discuss:
- Factors to consider when evaluating the use of an external control in a planned clinical study
- Appropriate analysis and interpretation of externally controlled studies
- The critical role of externally controlled studies in supporting marketing applications for orphan/rare disease products.
Kevin Barber, Ph.D.
Vice President, Regulatory Strategy & Submissions
Combining extensive strategic leadership, scientific knowledge, and regulatory expertise, Dr. Kevin Barber supports Rho’s clientele in the development and execution of integrated regulatory strategies and clinical development plans for drugs, biologics, and medical devices. With close to 25 years of experience in regulatory affairs and product development, Dr. Barber is a regulatory expert across all phases from preclinical through product launch and post-approval life cycle management. His significant experience moderating regulatory authority meetings and preparing and filing regulatory submissions includes more than 45 US INDs and more than 40 global marketing applications. Throughout his career, Dr. Barber has participated in…
Vice President, Regulatory Strategy & Biometrics
Joining the team in 2007, Rob Woolson brings 20 years of experience as an applied statistician to his current role as Rho’s Vice President of Regulatory Strategy & Biometrics. Mr. Woolson has an extensive background working with small biotech and pharmaceutical companies to successfully bridge and navigate the biometrics, CDISC data standards, and regulatory requirements of drug and biologics development. He has advised and supported sponsor companies through 12 marketing applications in several therapeutic areas, including playing a vital role in numerous FDA and Advisory Committee meetings. Much of Mr. Woolson’s work has focused on psychiatry-related product development, with an…