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Legislators in the USA have been discussing reforms to reduce the high cost of brand-name drugs, which are much higher in the USA than in other industrialized countries. One solution is to actively negotiate prices based on drugs’ clinical benefits. We discuss two important complexities from such an approach: drugs that have been approved for multiple indications and as part of a combination regimen.
Recent FDA draft guidance for sponsors of oncology clinical trials encourages enrolment of patients with incurable cancer and no potential for prolonged and/or near-normal survival, regardless of whether they have received existing treatment options. This guidance constitutes a substantial departure from current standards, with potentially profound implications for trial participants as well as drug regulation and reimbursement.
The treatments oncologists deliver are generally based on evidence from large randomized controlled trials, consistent with practice guidelines, and congruent with the treatments selected by our peers. In this Comment, we use two scenarios to highlight the discomfort clinicians might feel when they are outliers from the guideline-recommended standard of care.