Chapter 4 74 EPILOGUE Based on our research, we conclude that the benefits of drugs under development is highly variable. If there is benefit at all, the magnitude of that benefit equally variable. Some drugs provide marginal improvements, while other drugs may cure patients from their illnesses. Patients that opt to access drugs in development through expanded access do so in the hope of accessing life-saving drugs. As we have concluded in our research, the majority of drugs under development cannot live up to that expectation. Physicians and patients could be misled by overconfident positive results presented both by physician-researchers and medical writers, and in publicly accessible channels such as news media and social media. It is crucial to ensure that patients are adequately informed of the risks and benefits of accessing drugs through expanded access. However, this part is only a proxy for assessing the value of expanded access. In theory, if expanded access only occurred to the most effective drugs, the average ‘effectiveness’ of drugs is not a good estimate of the added value of expanded access. Unfortunately, actual numbers of patient access to individual drugs are confidential and known only to regulators and drug companies. Nevertheless, our paper in Part II ‘Results from expanded access programs: a review of academic literature’ will highlight that the scientific dissemination of expanded access programs does show disproportionate access to ‘successful’ drugs, as the most frequently described drugs in expanded access publications are nivolumab, ipilimumab, gefitinib, sofosbuvir, and sunitinib which are all regulatory approved drugs with substantial clinical benefit. Moreover, as can be see in Figure 1 in the Prelude, the (group) compassionate use programs initiated in the Netherlands were aimed at highly effective drugs, such as blinatumomab, which in our analysis revealed the highest QALY gain among all hematology drugs (Figure 4). Similarly, previous research has shown that expanded access increases as data accumulates when drugs advance through the stages of clinical development.3 Regulators are increasingly willing to allow access to experimental drugs as more data becomes available, physician may feel more confident in prescribing later-stage drugs, and medical companies see less risk in providing access to drugs where substantial data on safety and efficacy has been obtained. This natural process decreases the chances that patient would access ineffective drugs, and positively effects clinical merit of expanded access for individual patients. While it is crucial not to overstate the clinical value of drugs in development and expanded access itself, there may still be value in expanded access programs that go beyond individual patient perspectives and provide collective benefits. For example, patients can contribute to scientific research through the generation of data within expanded access programs.
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