John B. Watkins

Challenges in the Development and Reimbursement of Personalized Medicine—Payer and Manufacturer Perspectives and Implications for Health Economics and Outcomes Research: A Report of the ISPOR Personalized Medicine Special Interest Group

BACKGROUND Personalized medicine technologies can improve individual health by delivering the right dose of the right drug to the right patient at the right time but create challenges in deciding which technologies offer sufficient value to justify widespread diffusion. Personalized medicine technologies, however, do not neatly fit into existing health technology assessment and reimbursement processes. OBJECTIVES In this article, the Personalized Medicine Special Interest Group of the International Society for Pharmacoeconomics and Outcomes Research evaluated key development and reimbursement considerations from the payer and manufacturer perspectives. METHODS Five key areas in which health economics and outcomes research best practices could be developed to improve value assessment, reimbursement, and patient access decisions for personalized medicine have been identified. RESULTS These areas are as follows: 1 research prioritization and early value assessment, 2 best practices for clinical evidence development, 3 best practices for health economic assessment, 4 addressing health technology assessment challenges, and 5 new incentive and reimbursement approaches for personalized medicine. CONCLUSIONS Key gaps in health economics and outcomes research best practices, decision standards, and value assessment processes are also discussed, along with next steps for evolving health economics and outcomes research practices in personalized medicine.

Multiple Criteria Decision Analysis for Health Care Decision Making—Emerging Good Practices: Report 2 of the ISPOR MCDA Emerging Good Practices Task Force

Health care decisions are complex and involve confronting trade-offs between multiple, often conflicting objectives. Using structured, explicit approaches to decisions involving multiple criteria can improve the quality of decision making. A set of techniques, known under the collective heading, multiple criteria decision analysis (MCDA), are useful for this purpose. In 2014, ISPOR established an Emerging Good Practices Task Force. The task forces first report defined MCDA, provided examples of its use in health care, described the key steps, and provided an overview of the principal methods of MCDA. This second task force report provides emerging good-practice guidance on the implementation of MCDA to support health care decisions. The report includes: a checklist to support the design, implementation and review of an MCDA; guidance to support the implementation of the checklist; the order in which the steps should be implemented; illustrates how to incorporate budget constraints into an MCDA; provides an overview of the skills and resources, including available software, required to implement MCDA; and future research directions.

Health Technology Assessment in Health-Care Decisions in the United States

Health technology assessment (HTA) is a form of policy research that examines shortand long-term consequences of the application of a health-care technology. Properties assessed include evidence of safety, efficacy, patient-reported outcomes, real-world effectiveness, cost, and cost-effectiveness as well as social, legal, ethical, and political impacts [1]. At least five distinct activities define a formal health technology assessment process: 1) horizon scanning; 2) topic determination and queuing; 3) collection and assessment of evidence; 4) appraisal; and 5) funding and policy implementation. Horizon scanning involves the early examination and active monitoring of emerging technology to determine, in part, potential evidence requirements, and budgetary implications. Topic determination and queuing activities focus on setting priorities and sequencing of emerging and previously considered technologies for assessment or reassessment. The assessment function entails the process of collecting, evaluating, and systematically reviewing all available evidence for the technology under consideration. Appraisal is the decision-making function and is often distinguished by an external body (e.g., a pharmacy and therapeutics committee in the United States) that considers and weighs the summarized evidence in order to render a recommendation to the payer. Funding and policy implementation are the final steps in the HTA process. The explicit objective of organizations that operate formal HTA programs is to carefully consider a full range of clinical and economic evidence in order to render decisions as to the acceptance, modification, or rejection of technologies on a rational basis [2]. The UK National Health Service National Coordinating Center for HTA suggests that HTA programs directly consider the following attributes of health technology as they undertake their mission: When compared with existing alternatives, does the technology work, in whom does the technology work, and what is the cost impact? [3]. The funding for and use of health technology assessment programs in the United States has a long and storied history, is fragmented and uncoordinated, and includes both public and private sector initiatives. Some readers will be surprised to learn that a number of US HTA programs predate the development of well-known international efforts in Australia (Pharmaceutical Benefits Advisory Committee), Canada (Canadian Agency for Drugs and Technologies in Health), Sweden (Swedish Council on Technology Assessment in Health Care) and the United Kingdom (National Institute for Health and Clinical Excellence [NICE]). Regrettably, a number of these early US initiatives have been discontinued or have been substantially altered in large part because of political, financial and commercial pressures. In this article, we consider the changing landscape for systematic health technology assessment in the United States and what it means for health-care payers. In Section I, we start with a description of the many public and private agencies supplying and using health technology assessment reports to make coverage and reimbursement decisions. In Section II, against a backdrop of escalating costs and few restrictions on the pricing and use of health-care technology, we discuss the factors that are shaping and challenging private and public sector HTA programs. Finally, in Section III, we offer commentary on the potential role that a more formal approach to HTA can play in health care in the United States.

Impact of a Value-based Formulary on Medication Utilization, Health Services Utilization, and Expenditures

Background: Value-based benefit design has been suggested as an effective approach to managing the high cost of pharmaceuticals in health insurance markets. Premera Blue Cross, a large regional health plan, implemented a value-based formulary (VBF) for pharmaceuticals in 2010 that explicitly used cost-effectiveness analysis (CEA) to inform medication copayments. Objective of the Study: The objective of the study was to determine the impact of the VBF. Design: Interrupted time series of employer-sponsored plans from 2006 to 2013. Subjects: Intervention group: 5235 beneficiaries exposed to the VBF. Control group: 11,171 beneficiaries in plans without any changes in pharmacy benefits. Intervention: The VBF-assigned medications with lower value (estimated by CEA) to higher copayment tiers and assigned medications with higher value to lower copayment tiers. Measures: Primary outcome was medication expenditures from member, health plan, and member plus health plan perspectives. Secondary outcomes were medication utilization, emergency department visits, hospitalizations, office visits, and nonmedication expenditures. Results: In the intervention group after VBF implementation, member medication expenditures increased by

Improving the Usefulness of Budget Impact Analyses: A U.S. Payer Perspective

2 per member per month (PMPM) [95% confidence interval (CI),

Creating Models That Meet Decision Makers' Needs: A US Payer Perspective

1–

The AMCP Format for Formulary Submissions: Welcome to version 4.0

3] or 9%, whereas health plan medication expenditures decreased by

Php27 Quality of Economic Models in Dossiers Submitted Undertheamcp Format

10 PMPM (CI,

Principles of Good Practice for Budget Impact Analysis: Report of the ISPOR Task Force on Good Research Practices—Budget Impact Analysis

18–

Multiple Criteria Decision Analysis for Health Care Decision Making—An Introduction: Report 1 of the ISPOR MCDA Emerging Good Practices Task Force

2) or 16%, resulting in a net decrease of