Risk-sharing agreements, under which payers and pharmaceutical manufacturers agree to link payment for drugs to health outcomes achieved, rather than the volume of products used, offer an appealing payment model for pharmaceuticals. Although such agreements have been widely touted, the experience to date mainly demonstrates how hard they are to implement. Barriers include high implementation costs, measurement challenges, and the absence of a suitable data infrastructure. Risk-sharing arrangements could gain traction in the United States as payers and product manufacturers acquire experience with the concept and as measurement techniques and information systems improve. For the foreseeable future, they are likely to remain the exception as drug companies pursue payment models unconnected to data collection or performance assessment.

The rapid development of immuno-oncology (I-O) therapies for multiple types of cancer has transformed the cancer treatment landscape and brightened the long-term outlook for many patients with advanced cancer. Responding to ongoing efforts to generate value assessments for novel therapies, multiple stakeholders have been considering the question of “What makes I-O transformative?” Evaluating the distinct features and attributes of these therapies, and better characterizing how patients experience them, will inform such assessments. This paper defines ways in which treatment with I-O is different from other therapies. It also proposes key aspects and attributes of I-O therapies that should be considered in any assessment of their value and seeks to address evidence gaps in existing value frameworks given the unique properties of patient outcomes with I-O therapy. The paper concludes with a “data needs catalogue” (DNC) predicated on the belief that multiple key, unique elements that are necessary to fully characterize the value of I-O therapies are not routinely or robustly measured in current clinical practice or reimbursement databases and are infrequently captured in existing research studies. A better characterization of the benefit of I-O treatment will allow a more thorough assessment of its benefits and provide a template for the design and prioritization of future clinical trials and a roadmap for healthcare insurers to optimize coverage for patients with cancers eligible for I-O therapy.

Contemporary clinical effectiveness and cost estimates show an initial surgical bypass is the most cost-effective alternative to local wound care alone for CLI with tissue loss and can be supported even in a cost-averse health care environment.

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This research was funded by Biogen, which provided an unrestricted research grant to the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center. Biogen and Sobi reviewed and provided feedback on the manuscript. The authors had full editorial control of the manuscript and provided final approval of all content. The authors report no conflict of interest regarding the material discussed in this article. Neumann and Chambers are employed at the Center for the Evaluation of Value and Risk in Health at Tufts Medical Center. Thorat was an employee of Center for Evaluation Value and Risk in Health, Tufts Medical Center when the analyses were carried out. Chambers has participated on advisory boards for Sanofi and Astellas Pharma.

We analyzed specialty drug coverage decisions issued by the largest US commercial health plans to examine variation in coverage and the consistency of those decisions with indications approved by the Food and Drug Administration (FDA). Across 3,417 decisions, 16 percent of the 302 drug-indication pairs were covered the same way by all of the health plans, and 48 percent were covered the same way by 75 percent of the plans. Specifically, 52 percent of the decisions were consistent with the FDA label, 9 percent less restrictive, 2 percent mixed (less restrictive in some ways but more restrictive in others), and 33 percent more restrictive, while 5 percent of the pairs were not covered. Health plans restricted coverage of drugs indicated for cancer less often than they did coverage of drugs indicated for other diseases. Using multivariate regression, we found that several drug-related factors were associated with less restrictive coverage, including indications for orphan diseases or pediatric populations, absence of safety warnings, time on the market, lack of alternatives, and expedited FDA review. Variations in coverage have implications for patients' access to treatment and health system costs.

Medicare legislation mandates that the program not pay for items and services that are not "reasonable and necessary" - terms that are open to interpretation and easily politicized. In today's fiscal environment, "reasonable and necessary" warrants a closer look.

CMS is covering a number of interventions that do not appear to be cost-effective, suggesting that resources could be allocated more efficiently. Although the authors identified several instances where cost-effectiveness evidence was cited in NCDs, they found no clear evidence of an implicit threshold.