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Using Real World Evidence for Externally Controlled Trials in Rare Disease Drug Development: Insights from ISPOR Europe

Key insights in managing the complexities of external control arms (ECAs) in rare disease clinical trials, garnered from ISPOR and ICPE participation and in partnership with the FIORD Work Project.

In the realm of rare disease research, the traditional pathway of utilizing randomized controlled trials (RCTs) to gather evidence faces significant ethical and practical hurdles. The rarity of patient populations often renders such trials unfeasible or, in some cases, unethical. This challenge has led to the exploration of innovative trial designs that leverage external control arms (ECAs) to contextualize the findings of single-arm studies.

As the pharmaceutical industry seeks to navigate these complexities, the integration of real-world secondary data sources to provide comparative evidence has become increasingly prevalent. These efforts aim to support applications to regulatory bodies and health technology assessment (HTA) agencies, ensuring that new therapies can reach those in need with robust backing.

Download the white paper below to learn insights into the use of ECAs in the advancement of rare disease treatment and the evolving landscape of clinical research.

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