Patient Participation in Clinical Trials of Oncology Drugs and Biologics Preceding Approval by the US Food and Drug Administration
The study provides a crucial understanding of the role and impact of patient contributions in oncology drug development, focusing on their association with clinically impactful drug outcomes. Successful oncology drugs required an average of 12,217 patients in pre-license trials, with higher numbers for drugs meeting ASCO-VF standards. This highlights the significant patient involvement needed for regulatory licensure compared to unsuccessful efforts. The study underscores the moral implications, noting that patients undertake substantial risks for future patient care, often without direct personal gain. It raises ethical considerations, as more than two-thirds of participants contributed to non-approved efforts within 8 years.
Identifying research activities that maximize patient contributions can lead to more efficient drug development, with preliminary findings suggesting biomarker enrichment trials and immunotherapy drugs may require fewer patients. Policy implications include designing policies that support morally efficient drug development, such as evaluating the Pediatric Exclusivity Provision based on patient investment metrics. In summary, optimizing patient contributions respects their sacrifices and enhances research efficiencies, guiding future policy adjustments and research priorities towards a patient-centered approach.
Recent studies estimate the median cost of bringing a new drug to market at $985.3 million for all therapeutic agents and $793.4 million for oncology drugs. For every 100 drugs entered into phase 1 clinical testing in oncology, fewer than 10 will ultimately receive regulatory approval. A minority of approved cancer drugs provide substantial clinical benefit.
However, cost estimates and molecule success rates do not reflect the full range of inputs societies commit to pharmaceutical research. Drug development requires that patient study participants agree to submit to screening, forgo standard care, receive experimental medicines, and undergo research procedures. Drug development also requires that physicians and patients invest substantial cognitive resources in explaining and understanding study protocols. Previous studies of patient enrollment have shown that a median of 1708 patients participated in clinical trials per European Medicines Agency new molecular entity approval; a median of 2316 patients were studied per US Food and Drug Administration (FDA) approval of a precision medicine oncology drug.
However, these studies restricted their analyses to approved drugs and thus do not account for the large numbers of patients participating in unsuccessful drug development efforts. Estimates that include all patient study participants can make more visible the degree to which private drug development efforts build on public volunteerism. They can also help to identify research activities that more efficiently use this public endowment. The aim of this study was to estimate the number of patient study participants needed to obtain a first FDA approval for a cancer therapeutic.
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