Evaluating Frameworks That Provide Value Measures for Health Care Interventions

Abstract

The recent acceleration of scientific discovery has led to greater choices in health care. These new technologies, diagnostic tests, and pharmaceuticals have widely varying impact on patients and populations in terms of benefits, toxicities, and costs, stimulating a resurgence of interest in the creation of frameworks intended to measure value in health. Many of these are offered by providers and/or advocate organizations with expertise and interest in specific diseases (e.g., cancer, heart disease). To help assess the utility of and potential biases embedded in these frameworks, we created an evaluation taxonomy with seven basic components: (1) define the purpose; (2) detail the conceptual approach, including perspectives, methods for obtaining preferences of decision makers (e.g., patients), and ability to incorporate multiple dimensions of value; (3) discuss inclusions and exclusions of elements included in the framework, and whether the framework assumes clinical intervention or offers alternatives such as palliative care or watchful waiting; (4) evaluate data sources and their scientific validity; (5) assess the intervention’s effect on total costs of treating a defined population; (6) analyze how uncertainty is incorporated; and (7) illuminates possible conflicts of interest among those creating the framework. We apply the taxonomy to four representative value frameworks recently published by professional organizations focused on treatment of cancer and heart disease and vaccine use. We conclude that each of these efforts has strengths and weaknesses when evaluated using our taxonomy, and suggest pathways to enhance the utility of value-assessing frameworks for policy and clinical decision-making.

Do current approaches to assessing therapy related adverse events align with the needs of long-term cancer patients and survivors?

“…This review captures recommendations and perspectives from a total of 17 peer-reviewed publications [95,[102][103][104][106][107][108][109][110][111][112][113][114][115][116][117][118]. The reviewed studies were almost evenly split between those including a narrative/qualitative comparison of different frameworks and those incorporating case study/quantitative comparisons across different value frameworks.…”

“…Specifically, enhanced clarity and transparency as to the intended audience for the framework outputs [103,104,110,111,121] and improved guidance to enhance reproducibility were common recommendations [107,108,111,114,119,122].…”

“…Additionally, more comprehensive insights into methodological and data input assumptions across these studies could be gleaned by review of key underlying studies cited by the studies reviewed here. [103,112,119,138] Frameworks should incorporate data from sources other than clinical trials (e.g., observational studies) 3 of 17 (18%) [102] Frameworks should incorporate more robust and/or detailed safety and/or toxicity data 7 of 17 (41%) [103,104,108,112,115,116] Frameworks should use integrated quality of life measures in lieu of safety data 1 of 17 (6%) [117] Frameworks should incorporate more longitudinal data 2 of 17 (12%) [102,103] Frameworks should engage patients in the data evaluation and input process 3 of 17 (18%) [102,103,110] …”

“…Cost metrics used within the current value framework structures were equally variable but are largely restricted to cost of the drug and/or primary treatment visits. Many of the analyses reviewed here specifically recommended that future iterations of these models should incorporate the full cost of care including AEs [104,108,116,117,137] This recommendation, while sound on its face, begs the questions of what constitutes the burden of antineoplastic therapy-related AEs, who carries these burdens, and how broadly should costs be captured. It also speaks to the importance of transparent discussion regarding the stakeholders to whom the value assessment is intended to apply.…”

Measuring Value in Health Care: A Comparative Analysis of Value-based Frameworks

“…This definition played as an exclusion criterion, so that we included only applicable frameworks and not general "value" initiatives or evaluation methodologies, such as multiple-criteria decision-analysis methods, 8 evidencebased approaches (eg, Evidem 9 , www.evidem.org), or on-line tools for the calculation of incremental costeffectiveness ratio (eg, Innovation and Value Initiative, www.thevalueinitiative.org). Based on previously proposed taxonomies for the evaluation of VBFs, 5,7 we developed an extraction template aimed at collecting and contrasting information on selected common features: VBFs by developer cluster; country; target therapy; aim(s); target audience; definition of value; target use and interventions addressed; measures and variables considered; stakeholders involved in designing, commenting, and updating the framework; framework-update process; and implementation examples, when available.…”