Understanding and improving value frameworks with real-world patient outcomes.

OBJECTIVES: To provide recommendations that will improve approaches to measuring the value of new medical technologies to patients. STUDY DESIGN: Informed discussion by experts after literature review. METHODS: A working group was formed, and participants discussed how value frameworks should incorporate key features important to patients in evaluating new medical technologies, particularly for chronic diseases. RESULTS: The working group suggests that new value frameworks should integrate real-world evidence to complement randomized controlled trials, incorporate the ways in which real-world behavior mediates outcomes, and explicitly discuss how therapies affect real-world equity and disparities in care. CONCLUSIONS: Collective stakeholders that include key decision makers within our healthcare system need to recognize the importance of implementing real-world evidence and devote resources to further research into the chronic disease areas in which the impact of human behavior is amplified by the duration of disease and treatment.

The social value of childhood vaccination in the United States.

OBJECTIVES: To determine the lifetime social value of using the guideline-recommended vaccines for children born in the United States in 2009. STUDY DESIGN: This study utilized an economic model with parameter values sourced from clinical and observational data, as well as the literature. METHODS: The model quantified the health effects of routine vaccination for 14 diseases in terms of quality-adjusted life-years (QALYs) saved. The health effects were then valued by applying an economic value of a QALY. Producers' profits were estimated using data on vaccine prices, profit margins, and the number of vaccines administrated in the 2009 US birth cohort. The costs of producing the vaccines were subtracted from the value of the health effects to yield the total social value of vaccination. The producers' and consumers' shares of this social value were calculated. Sensitivity analyses were conducted to determine how results depend on underlying parameter assumptions. RESULTS: Estimates indicated that vaccination of this cohort will save 1.2 million QALYs, relative to no vaccination. Of those health gains, 88% stemmed from reduced mortality and 12% from reduced morbidity. We estimated a social value of $184.1 billion from these gains, of which $3.4 billion accrues to manufacturers as profits, while $180.7 billion accrues to the rest of society. In sensitivity analysis, the total social value ranged from $40 billion to $675 billion, and the manufacturers' share ranged from 0.3% to 11.5%. CONCLUSIONS: Policy makers should account for this social value when considering policies affecting incentives to vaccinate and develop new vaccines.

Translating medical effectiveness research into policy: lessons from the California Health Benefits Review Program.

CONTEXT: Legislatures and executive branch agencies in the United States and other nations are increasingly using reviews of the medical literature to inform health policy decisions. To clarify these efforts to give policymakers evidence of medical effectiveness, this article discusses the California Health Benefits Review Program (CHBRP). This program, based at the University of California, analyzes the medical effectiveness of health insurance benefit mandate bills for the California legislature, as well as their impact on cost and public health. METHODS: This article is based on the authors' experience reviewing benefit mandate bills for CHBRP and findings from evaluations of the program. General observations are illustrated with examples from CHBRP's reports. Information about efforts to incorporate evidence into health policymaking in other states and nations was obtained through a review of published literature. FINDINGS: CHBRP produces reports that California legislators, legislative staff, and other major stakeholders value and use routinely in deliberations about benefit mandate bills. Where available, the program relies on previously published meta-analyses and systematic reviews to streamline the review of the medical literature. Faculty and staff responsible for the medical effectiveness sections of CHBRP's reports have learned four major lessons over the course of the program's six-year history: the need to (1) recognize the limitations of the medical literature, (2) anticipate the need to inform legislators about the complexity of evidence, (3) have realistic expectations regarding the impact of medical effectiveness reviews, and (4) understand the consequences of the reactive nature of mandated benefit reviews. CONCLUSIONS: CHBRP has demonstrated that it is possible to produce useful reviews of the medical literature within the tight time constraints of the legislative process. The program's reports have provided state legislators with independent analyses that allow them to move beyond sifting through conflicting information from proponents and opponents to consider difficult policy choices and their implications.

Litigating the science of breast cancer treatment.

Beginning in the late 1980s, many health insurers refused to cover high-dose chemotherapy with autologous bone marrow transplant (HDC/ABMT) for high-risk and metastatic breast cancer patients. Insurers denied coverage because there was no persuasive evidence of clinical effectiveness. In response, many women sued to compel coverage. After years of litigation and the expenditure of approximately $3 billion, randomized clinical trials (RCTs) showed that the procedure was no more effective and possibly more harmful than conventional therapy. To understand whether and how litigation contributed to the diffusion of the procedure, we conducted a series of case studies that examine the litigation tactics and strategies used by defense and plaintiffs' counsel. Despite the fact that HDC/ABMT lacked proven scientific effectiveness, insurance defense attorneys were unable to stop the procedure's diffusion. Plaintiffs' attorneys had a much easier and more sympathetic story to tell and were able to exploit vulnerabilities facing the defense.

Evaluating medical effectiveness for the california health benefits review program.

An important aspect of the mandate assessments requested by the California legislature is a review of the scientific and medical literature on the medical effectiveness of the proposed health insurance benefit mandate. Although such a review bears many similarities to effectiveness reviews that might be undertaken for publication as research studies, several important differences arise from the requirements of the California legislation. Our reviews are intended to assist the legislators in deciding whether to support a specific mandate to modify health insurance benefits in a particular way. Thus, our assessments focus on how the scientific literature bears on the proposed mandate, which may involve a complicated chain of potential effects leading from altered coverage to ultimate impact on health. Evidence may be available for only some of the links in the chain. Furthermore, not all the evidence may be directly applicable to the diverse population of California or the subpopulation affected by the mandate. The mandate reviews, including the medical effectiveness analyses, may be used in a potentially contentious decision making setting. The legislative calendar requires that they need to be timely, yet they must be as valid, credible, and based on the best information available as possible. The focus on applicability also implies the need for informed, technical decisions concerning the relevance of the articles for the report, and these decisions need to be made as transparent as possible. These goals and constraints yield an approach that differs somewhat from an investigator-initiated review of the literature.

Independent medical review of health plan coverage denials: early trends.

Concerns among patients and physicians that health plans would deny coverage for medically necessary services has brought about legislation mandating independent medical review (IMR) of denied services. Among 1,400 cases submitted in California, the most common areas for dispute were cancer care, endocrine/metabolic care (especially for obesity), orthopedic care, and neurological disorders. Surgery and pharmacy services constituted 52 percent of cases. In 58 percent of cases, IMR upheld the health plan's decision, while in 33 percent of cases the health plan's decision was overturned, which suggests that external IMR provides additional patient protection in California's health care system.