Postlude 250 benefits across disease areas, expanding beyond hematology and oncology. The Quality-Adjusted Life Year, or QALY, is a metric frequently used in cost-effectiveness research to study the added benefits of pharmaceuticals that are under evaluation for reimbursement in the United Kingdom. We concluded that the average clinical merit of novel drugs in development is modest at best. From these results, it may appear that the likelihood of deriving substantial clinical gains from expanded access programs is limited. However, the practical reality may be more positive, as physicians and patients may be more inclined to participate in expanded access programs, and companies are more willing to initiate said programs for more effective drugs than for the average drug, resulting in a higher chance of deriving a clinically positive outcome for current patients. Indeed, we will see this line of reasoning confirmed in Part II. Additionally, and of particular interest in this thesis, there may be benefits reaped through the analysis of data of current expanded access patients to inform the clinical practice of future patients. ♦ Knowledge generation through expanded access programs formed the basis of our subsequent research. When we started to write this thesis, information on the use of evidence from expanded access programs was predominantly anecdotal. Mandatory pharmacovigilance surveilled the occurrence of safety events in expanded access programs, some of which eventually informed drug labelling. The use of efficacy outcomes was undocumented – impossible or even considered unethical to some. We created algorithms to enable the systematic analysis of a large volume of health policy documents, at times employing advanced techniques like optical character recognition to convert historic images into textual data. These automated approaches enabled us to search through vast bodies of documents available online from regulators such as the US FDA, the EU EMA, and cost-effectiveness research institutes such as NICE in the UK. We provided the first systematic overviews of the use of expanded access data in Part II. To make these results accessible to the general public, we created online research explainer videos and animations to accompany our publicly available code1. We discovered that the use of data from expanded access programs has increased over time, not only by regulators, but expanding to reimbursement bodies and researchers. Data collection is no longer limited to safety parameters, but has expanded to encompass efficacy outcomes, patientreported outcomes, and even healthcare cost. It is evident that the utilization of expanded access has evolved past the realm of anecdote, to assume a substantive, and possibly even systematic 1 https://github.com/TobiasPolak
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