For patients suffering from rare diseases, the limited number of breakthrough treatments has hindered improvements to their quality of life. While most clinical trials have recruitment and retention challenges, rare disease trials encounter these in addition to unique challenges such as inherently small and geographically dispersed patient populations, more heterogeneity in diagnosis and treatment approaches, patient’s age and impact on the entire family, and other factors. Leanne Larson, SVP, Real-World Evidence and Access at Parexel, and Matthew Gordon, VP, Real-World Evidence Strategy at Parexel, discuss how real-world data/evidence and external control arms provide new hope for patients and developers. For example, with external control arms, researchers are now able to use real-world evidence (RWE) from existing medical records, databases, clinical trial data, etc. to speed up recruitment, an often time-consuming process to drug development. They also discuss the implications and opportunities from the FDA’s planned guidance on the use of RWE in clinical research and what this means for the industry and the rare disease community.
Leanne Larson, Senior Vice President, Real-World Evidence and Access, Parexel
Leanne brings over 25 years’ experience in healthcare, featuring extensive work in pharmaceutical product development and commercialization, and in healthcare technology and operational consulting. She is an industry leader in designing and leading observational / non-interventional studies and in advancing the science of outcomes research, and she publishes and speaks widely on a variety of topics in this area.