The Rare Disease Revolution

Jan 25, 2018

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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. Real World Evidence (RWE) is expected to play a major role in the approval of future new medicines in these new drug approval pathways.

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 pediatric 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.



William C. Maier, PhD, Chief Scientific Officer, Mapi Group, an ICON plc. company

Jim Carroll, Vice President, Real World Evidence, ICON plc.


Date and Time:

Live: Thursday, 25 Jan., 2018 | 11 am EST | 10 am CST | 1600 GMT | 1700 CET

After the final airing of the webcast on 25 Jan., 2018 it will be available on demand until 25 Jan., 2019. 

Sponsor: ICON plc.

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