Can Compassionate Use Programs Help You See into the Future? A Case Study that Says Yes.

Can Compassionate Use Programs Help You See into the Future? A Case Study that Says Yes.

Compassionate use programs provide access to investigational products on unsolicited physician requests for patients with serious or immediately life-threatening conditions. These patients either have limited treatment options or have exhausted all other standard therapies. Compassionate use programs not only provide patients an additional treatment option, but these programs also offer manufacturers an opportunity to approximate the future effectiveness of their drug outside of a clinical trial setting. Implementing chart review studies in compassionate use program populations permits the collection of pre-approval data which can be used to inform important economic evaluations, value dossiers, and drug safety assessments.

Case Study

UBC designed and implemented three multi-national chart review studies consisting of patients who were provided oral oncology therapies via compassionate use programs. The studies were initiated pre-approval following positive clinical trial findings. Site and patient selection was performed using compassionate use enrollment data, which allowed for a more rapid study start-up.

Prior to chart review initiation, ethical approval was sought on a local and/or national level in line with country regulatory requirements. The sample sizes varied from approximately 300-350 charts per study and countries included: Australia, Belgium, Greece, Ireland, Israel, Italy, Netherlands, New Zealand, Spain, Switzerland, and the United Kingdom.

We were able to collect the following data:

·         Patient demographics

·         Medical/disease/treatment history

·         Reasons for treatment discontinuation

·         Clinical benefit

·         Adverse and serious adverse events

·         Overall survival

·         Duration of treatment

·         Patterns of care

·         Drug dosing

·         Progression-free survival

·         Patterns of clinical progression


The site staff entered the collected data directly into a compliant electronic data capture system specifically designed for chart review studies. The data were cleaned at point of data entry in real-time, resulting in tailored international datasets.

Actionable Results

Periapproval chart review studies of patients in compassionate use programs can approximate real-world post-market use outside of the clinical trial setting by characterizing patterns of use and associated treatment costs. These studies provide manufacturers a peek into the future success of a drug, which can help inform clinical, health economic, and market access decisions.

UBC’s Value Demonstration team understands that chart review study design requires a balance between scientific rigor, practicality, and feasibility, specifically when used in compassionate use programs. Contact us to discuss how our team can use our expertise to help approximate your product’s post-market practice patterns and costs.

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