Prelude 14 The multiple stakeholders in expanded access Patients predominantly hope to benefit from accessing experimental treatments.29 However, this access is only clinically valuable if the experimental treatments themselves offer advantages. Whether investigational treatments, and thereby expanded access, meet these clinical expectations is unclear. In this thesis, we explore the clinical value of medicine that may be obtained through expanded access. Apart from patients, expanded access involves a range of stakeholders with varying incentives. Physicians strive to offer optimal treatment options for their patients,30 companies seek to demonstrate ‘compassion’, enhance patient/physician engagement, and uphold their reputation,31 and regulators aim to provide greater access to novel therapies. Simultaneously, expanded access presents several drawbacks. Patients should not be exposed to unsafe or ineffective treatments, and physicians face an increased workload and may encounter both safety and ethical concerns when prescribing unapproved medicine.32–34 Companies must bear the cost providing experimental medicine and must dedicate ample resources to navigate differing regulatory pathways, while regulators must balance the needs of current patients without hindering clinical development and thereby endanger broader access to future patients.4 This thesis examines the practical and ethical dilemmas that can arise from such conflicting interests. Expanded access programs are primarily designed to provide treatment, and as a result, data of patients that participate in expanded access programs are often overlooked.35 In fact, some jurisdictions specifically prohibit the collection of data other than safety events in this setting.22 Nonetheless, there has been growing attention to the potential for expanded access programs to generate evidence. In this way, patients could altruistically contribute to the overall assessment of safety and effectiveness of new medicine, physicians could cooperatively participate in research that informs future clinical decision-making, companies could collect data to support regulatory approvals, reimbursement decisions, or publications, and regulators obtain a broader view of how novel treatments work outside of clinical trial patients. In this thesis, we delve deeper into the value of expanded access as a means to generate evidence. Generating evidence through expanded access Generating evidence through expanded access pathways has become a growing area of interest,36 with anecdotal evidence suggesting that data collected from such programs can be used to inform publications, reimbursement appraisals, and regulatory decision-making.37 Some countries have even integrated the evaluation of treatments under expanded access pathways in regulatory and
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