Real world evidence is expected to play a major role in the approval of future new medicines

New regulatory developments in both the US and Europe are promoting a radical change to accelerate the approval of new medications for rare disease. These include the 21st Century Cure Act in the US and the Adaptive Regulatory Pathway in Europe.

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While very exciting for rare disease drug development, the differences from traditional drug development require attention to multiple areas, including small patient populations, more occurrence in paediatric patients, and complicated presentation of disease.

These small populations make it both practically and ethically difficult to conduct multiple confirmatory efficacy trials. There may be no established treatment standards so the design of the registration studies may involve use of a RWE approach to observe changes in clinical status over time rather than comparisons of drug effect relative to a placebo or comparator therapy.

The study process also needs to recognize changes in the patient’s disease and lifestyle and adapt both the study design and methods to a more patient-focused approach. Many rare diseases have strong patient advocacy groups that can aid in the design and execution of rare disease registries to identify and engage rare disease patients as part of clinical development

Key speakers

Jim Carroll

Vice President, Real World Evidence Strategy & Analytics Commercialisation and Outcomes, ICON

Jim Carroll oversees the development of real world data (RWD) and technology based solutions to support sponsors who are seeking to expand labelling and market access, while staying ahead of the growing demand for evidentiary requirements. Jim has over 20 years’ CRO and pharmaceutical experience and joins ICON from inVentiv Health, where he led the formation of a new business unit focused on providing novel RWD-driven services. Jim previously worked at IMS Health (now IQVIA), where he led the development and commercialization of innovative RWD-based, global analytic solutions to improve decision outcomes across the biopharmaceutical product lifecycle.

William C. Maier, PhD

Chief Scientific Officer, Mapi, an ICON plc company

Dr. Maier has over 20 years of experience in drug development and commercialization at pharmaceutical companies in Europe, Canada, the United States and Asia. He works with pharmaceutical companies throughout the world to provide regulatory, strategic and scientific guidance on medical treatment development and commercialisation. He is an expert in Real World Evidence and has worked with companies on the design, conduct and approval of over 100 observational studies related to biotechnology, consumer products, pharmaceuticals, drug development and product commercialisation.