Registration: https://diaglobal.zoom.us/webinar/register/WN_6wPKTvBUSQqTYU6Ae-a0Lg
The DIA Statistics and Data Science Community is pleased to announce a New Webinar on 1 October 2021, 10:00 - 12:00ET.
The Statistical Considerations for Rare Disease Clinical Trials webinar will see presentations from the following experts:
Anita Zaidi (FDA)
Xiang Yin (Medidata)
Qing Liu (QRmedSci)
Ben Saville (Berry Consultants)
Ming-Dauh Wang (Bayer)
Abstract:
Drug development in rare diseases presents many challenges as well as opportunities for innovation and adoption of novel approaches. The substantial evidence of effectiveness has generally been interpreted as two adequate and well-controlled trials. This requirement could also be met by a single trial plus confirmatory evidence which may be more feasible in the rare disease population. However, the limited population of patients and limited availability of natural history information often hinders the ability to design an adequate and well controlled trial. Some rare diseases primarily (or exclusively) affect children and, thus, additional ethical considerations of enrolling children in clinical investigations come into play and need to be considered carefully. Lastly, selecting and validating clinical endpoints for demonstration of drug efficacy is often exceedingly difficult for various reasons and the use of biomarkers can become important but also includes complex considerations as a substitute to clinical evidence of efficacy.
We do hope you can join us for this exciting event.
Registration: https://diaglobal.zoom.us/webinar/register/WN_6wPKTvBUSQqTYU6Ae-a0Lg
--------------------
https://diaglobal.zoom.us/webinar/register/WN_6wPKTvBUSQqTYU6Ae-a0Lg
https://diaglobal.zoom.us/webinar/register/WN_6wPKTvBUSQqTYU6Ae-a0Lg