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W 15: Applications of Expanded Access/Compassionate Use Programs for Evidence Generation

Poster Presenter

      Marielle Bassel

      • Research Scientist II
      • UBC: An Express Scripts Company


Understand what evidence and value messages can be generated from chart review studies in expanded access patient populations and describe best practices for collecting data from these programs to inform real-world use, treatment effectiveness and clinical outcomes.


ORAL PRESENTATION SCHEDULED: Session 2A at 10:10 - 10:20 AM

Key attributes to the design and conduct of chart review studies in expanded access populations will be described including: streamlined site identification and enrollment and focused data collection and endpoints that can be addressed and disseminated.


Expanded access programs, also known as compassionate use and named patient programs provide peri-approval drug access to patients with limited available treatment options based on voluntary physician requests. These programs are conducted prior to market approval and offer an opportunity to understand the impact of investigational medications in a non-trial setting. While patients provide informed consent and are enrolled into these programs, these programs typically do not involve data collection beyond safety data (serious adverse event, adverse event of special interest). There is a wealth of information that can be gleaned from these programs and data collection would be the first evidence of how the drug is performing outside of the highly controlled clinical trial setting. Peri-approval chart review studies of patients in expanded access programs offer an important opportunity to inform clinical, health economic, and market access decisions by: characterizing patterns of use and associated treatment costs and understanding the clinical impact of investigational medications in a non-trial setting. Data on confounding variables possibly excluded in trials and outcomes from sicker populations can be evaluated.


With the increase in expanded access programs and the need to understand drug performance outside of clinical trials, chart review studies can be successfully undertaken to inform on early drug use in non-trial settings with respect to patient characteristics, treatment effectiveness and drug safety profile.

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