Part IV ♠ 218 PROLOGUE Views on expanded access policy differ among jurisdictions. Where some countries lack welldefined pathways, others may have multiple established routes to access unapproved medicine. In the United States, the Food and Drug Administration simultaneously regulates market approval and expanded access for all states.1 In the European Union, the European Medicines Agency approves products via a centralized procedure, but expanded access legislation is drafted on a national basis by individual member states.2 This has led to a proliferation of national pathways. In France, under certain conditions, medical companies can receive reimbursement for expanded access medicine, leading to a thriving market in unlicensed drugs.3 In Belgium, pharmaceutical companies must pay a fee to initiate a program and subsequently provide the drug for free.4 Regarding data collection, the United Kingdom offers integrative health technology assessment alongside expanded access through the Early Access to Medicines (EAMS) scheme,5 whereas Austria specifically forbids the systematic collection of data through their (single-patient) expanded access pathway.6 While Italy, Spain, and Belgium produce the largest number of publications per capita, Czechia, Slovakia, and Bulgaria have virtually no output on expanded access programs. The lack of harmonization in Europe has been subject of debate as the complexity of expanded access pathways increases.7 Policies and legislation regarding expanded access primarily have the same intent: providing treatment options to patients. However, the freedom to define expanded access per member state has led to a patchwork of national access pathways that vary in terms of regulatory requirements, procedures, and timelines, not to mention linguistic barriers. As such, these multitudes of polices may deter rather than expedite treatment options to patients in need. This lack of cohesion not only complicates matters for pharmaceutical companies, healthcare providers, and patients, but also hampers the generation of real-world evidence, which we have shown can be important for regulatory or reimbursement decisions, or in the dissemination of results developed through said programs. As a result, there is a growing need for compatible compassionate use legislation and European harmonization to balance access with evidence generation. In this part, we will delve into concerns regarding the discrepancies in regulations, which may result in issues related to patient equity, and we will suggest policy enhancements to address these concerns. Subsequently, we will present a comprehensive analysis of the various viewpoints on expanded access as a data generation approach, with a particular focus on rare disease medicine, and explore potential strategies to integrate these different ideas. Lastly, we will evaluate expanded access as a means to conduct research from an ethical perspective.
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