The oncologist
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Patients, clinicians, payers, and policymakers face an environment of significant evidentiary uncertainty as they attempt to achieve maximum value, or the greatest level of benefit possible at a given level of cost in their respective health care decisions. This is particularly true in the area of oncology, for which published evidence from clinical trials is often incongruent with real-world patient care, and a substantial portion of clinical use is for off-label indications that have not been systematically evaluated. It is this uncertainty in the knowledge of the clinical harms and benefits associated with oncology treatments that prevents postregulatory decision makers from making accurate assessments of the value of these treatments. ⋯ Food and Drug Administration, this situation is exacerbated in the area of oncology. This paper investigates the convergence of incentives and circumstances that lead to widespread uncertainty in oncology and proposes new paradigms for clinical research, including pragmatic clinical trials, methodological guidance, and coverage with evidence development. Each of these initiatives would support the design of clinical research that is more informative for postregulatory decision makers, and would therefore reduce uncertainty and provide greater confidence in conclusions about the value of these treatments.
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The U. S. Food and Drug Administration grants marketing approval for drug products based on a comprehensive review of safety and efficacy data. ⋯ Overall survival is the gold standard for a registration trial designed to gain marketing approval; however, additional endpoints have been used in the approval of oncology drugs. Advantages of specific endpoints are discussed, including the accuracy of an endpoint's measurement and its relation to clinical benefit. Surrogate endpoints may be acceptable for "accelerated" approval, with a sponsor commitment to provide evidence of clinical benefit in a subsequent trial.
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This paper examines the issue of prices, relative to value, for cancer drugs. The analysis focuses on the effects on manufacturer pricing incentives of insurance coverage, specifically, the effectiveness of patient cost sharing, incentives created by reimbursement rules for physician-dispensed drugs, and payer ability and incentives to negotiate discounts. For pharmacy-dispensed cancer drugs, both Medicare Part D prescription drug plans (PDPs) and private payers' pharmacy benefit managers are increasingly placing these drugs on specialty tiers that offer no leverage for negotiating discounts and imply often unaffordable cost sharing for patients who lack catastrophic coverage. ⋯ S. data. Making such outcomes-adjusted prices available in the U. S. would be helpful to physicians, payers, and patients and indirectly constrain pricing to align with value.
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Many new cancer drugs provide only limited benefits, but at very great cost, for example, $200,000-$300,000 per quality-adjusted life year produced. By most standards of value or cost-effectiveness, this does not represent good value. ⋯ I examine several equity considerations-priority to the worse off, aggregation and special priority to life extension, and the rule of rescue-and argue that none justifies greater priority for cancer treatment on the grounds of equity. Finally, I conclude by noting two recent policy changes that are in the wrong direction for achieving value in cancer care, and suggesting some small steps that could take us in the right direction.