Chapter 10 222 We commend the initiators of the DRUG Access Protocol for their efforts to combine earlier access to medicine with structured data collection.9 Although this is a novel program in the Netherlands, similar programs covering compassionate use, evidence generation, and reimbursement are already in effect in England (Early Access to Medicines Scheme) and in France (L’Accès Précoce).10,11 The benefits of the DRUG Access Protocol in providing conditional reimbursement of registered drugs and thereby creating access are evident. However, the effects of the protocol in the setting of compassionate use (typically free of charge) requires further exploration. First, the current set-up of access to compassionate use in Europe has led to a patchwork of national access pathways. The DRUG Access Protocol could further complicate the process of obtaining access to compassionate use, by introducing a novel national pathway specifically for oncology. Pharmaceutical companies without local presence or sufficient resources may prefer to provide access in countries with easier access pathways, which raises issues of equity in patient access. With the harmonization of clinical trials through the Clinical Trial Regulation and health technology assessments through the EUnetHTA initiative, we believe the need grows for compatible compassionate use legislation, rather than further diversifying pathways. Second, the DRUG Access Protocol poses additional hurdles and workload to oncologists and companies as participation does not guarantee regulatory approval for compassionate use. Because this protocol is a voluntary, cooperative initiative and not a legally mandated pathway, regulatory approval still has to be obtained separately. This workload may deter rather than expedite access, especially for patients and oncologists in less specialized centers. The benefits of additional evidence generation may not outweigh the extra paperwork and research strains imposed on patients and physicians. In a broader context, the changing nature of compassionate use programs to provide ‘research’ rather than ‘treatment’ has been a growing source of concern among bioethicists.12 In recent years there has been an increased interest in compassionate use programs to generate evidence on safety and efficacy that supports trial results. The Compassionate Use Guidelines of the European Medicines Agency from 2007,2 which do not mention the collection of efficacy data, seem out of date. We hope a guideline revision will clarify the value of compassionate use as real-world evidence, shed light on the concerns of equity to access raised above, stimulate harmonization of access pathways, and incorporate the experiences from the DRUG Access Protocol.
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