Proposed framework for patient-centered outcomes-based measures in alternative payment models.

OBJECTIVES: Alternative payment models (APMs) are part of a growing shift from volume-based, traditional fee-for-service payment models toward payment for value. To date, however, patients have been largely omitted from efforts to design new payment models. We sought to identify key characteristics of outcomes-based quality measures to inform future APMs that are more patient-centered. STUDY DESIGN: Using oncology as a learning case, we explored gaps in current APM quality measures, then engaged multiple stakeholders to identify and prioritize key characteristics of outcomes-based quality measures to guide future APM development. METHODS: We used a mixed-methods approach that consisted of (1) literature review, (2) key informant interviews, (3) stakeholder work group (involving group discussions and completion of an online prioritization survey), and (4) synthesis. RESULTS: Based on the lessons generated at each step of this exploratory project, we suggest a framework to guide deliberations among payers, providers, patients, and other APM stakeholders when selecting outcomes-based measures for future APMs or other value-based payment models. CONCLUSIONS: The proposed framework offers a stepping stone on the path to clinically meaningful, patient-centered, high-value care. Next steps may include a broader review of gaps in APM quality measures across multiple therapeutic areas, additional vetting from a more diverse group of stakeholders, or a formal consensus.

Increasing the Patient-Centeredness of Health Economics and Outcomes Research Through Patient Engagement in Core Outcome Set Development.

Core outcome sets (COS) are becoming increasingly popular in clinical research and can provide important inputs for further health economics and outcomes research (HEOR) studies. Use of standard, consistently reported outcomes can demonstrate and allow differentiation of the effectiveness and value of different treatments. Incorporating patient values during COS development increases the patient centeredness of evidence available across decision-making contexts. However, the approach to meaningful patient engagement in the COS process is evolving and poses both unique challenges and opportunities. We describe an approach to patient-centered COS development and discuss challenges and adaptations to improve engagement across COS projects. We provide examples from our experience in patient engagement for COS development using three completed COS projects. This approach includes patient engagement in terms of partnering with patient organizations, orientation and training, and the consensus process. Including COS in clinical development programs and HEOR will ensure that relevant, consistent outcomes are available for healthcare decision making and should result in faster access to high-value and novel therapies for patients. Patient-centered COS development increases the likelihood that further HEOR studies and decisions made using the COS are relevant to patients.

The future of comparative effectiveness and relative efficacy of drugs: an international perspective.

Drug development takes place in a global marketplace, albeit with the USA and EU markets currently dominating. In the USA, demands for comparative effectiveness research have gained traction against a backdrop of health delivery reform, while European stakeholders deliberate the role of relative effectiveness in health technology assessment, trying to reduce the duplication of effort by regulators and health technology assessment bodies. In both arenas, drug-makers are faced with mounting drug development costs, and uncertainty over the types of evidence acceptable for a growing list of stakeholders. This article reports and compares future scenarios for evidence expectations for drugs for the USA and EU in 2020. The similarities, differences, and joint implications of the scenarios are considered to create an view of future evidence generation for drugs developed for these markets.

Futurescapes: expectations in Europe for relative effectiveness evidence for drugs in 2020.

AIM: Explore key factors influencing future expectations for the production of evidence of relative effectiveness (RE) for drugs in Europe in 2020; construct three plausible future scenarios for RE evidence generation. MATERIALS & METHODS: Semi-structured key informant interviews and three rounds of modified Delphi to gather expert perspectives and develop future scenarios. RESULTS & CONCLUSION: Most influential factors were degree of regulator use of postmarketing authorization (postlaunch) efficacy studies and adaptive licensing; degree of pan-European health technology assessment body coordination in reviewing prelaunch evidence and demanding postlaunch studies; the nature of regulator - health technology assessment body interaction. The most likely scenario entailed some change with postlaunch regulatory studies driving the likely nature of RE evidence generated.

A translation table for patient-centered comparative effectiveness research: guidance to improve the value of research for clinical and health policy decision-making.

This article provides background and context for a series of papers stemming from a collaborative effort by Outcome Sciences, Inc., the National Pharmaceutical Council and the Center for Medical Technology Policy to use a stakeholder-driven process to develop a decision tool to select appropriate methods for comparative effectiveness research. The perceived need and origins of the 'translation table' concept for method selection are described and the legislative history and role of the Patient-Centered Outcomes Research Institute are reviewed. The article concludes by stressing the significance of this effort for future health services and clinical research, and the importance of consulting end-users--patients, providers, payers and policy-makers--in the process of defining research questions and approaches to them.