Unlocking the value of expanded access ethical, statistical, and policy considerations

Patients with life-threatening or seriously debilitating diseases who cannot benefit from registered therapies or trial participation, may seek access to unapproved drugs through 'expanded access' programs. Expanded access programs offer a legal, regulated pathway to access otherwise unavailable medicine for patients in dire need.

The value of these programs, both for current and future patients, remains undetermined. In this thesis, we laid the groundwork by quantifying the clinical value of accessing medicines in development for current patients. Furthermore, future patients may benefit from data collection in expanded access programs, as we show that regulators, reimbursement bodies, and researchers increasingly use said data.

The increasing use of data from expanded access programs raises statistical concerns. We have developed a method to dynamically incorporate information from expanded access programs into trial analyses, by combining work from causal inference and bayesian dynamic borrowing.

As these programs can be viewed as controversial, we devoted a large portion of our thesis to discussing the ethics of expanded access programs. The evolution of expanded access programs—from primarily serving as a treatment avenue to becoming a research prospect—necessitates a revision of the existing policies and requires further ethical and statistical exploration.