A recent issue of Value in Health includes seven papers that comprise the ISPOR Special Task Force Report on US Value Assessment Frameworks.
The papers provide an introduction to value frameworks
; define elements of value
; discuss opportunity costs
; present a review of existing frameworks, and; outline a set of best practice recommendations
. OHE’s Adrian Towse
was a member of the Special Task Force and is a co-author of three of the papers. OHE Senior Visiting Fellow Lou Garrison was a co-chair of the Special Task Force and a co-author of five of the papers.
The emergence of competing value frameworks has led to the need to scrutinise the emerging frameworks to ensure scientific rigour. Hence the establishment of the ISPOR Task Force.
provides an overview of value and perspective and explores the relationship between these and the context of decision making. Defining value is crucial, as different interpretations exist – consider that value could represent what someone is willing to pay
for a technology (“gross value
”), or it could be the willingness to pay minus the opportunity cost incurred to obtain the technology (“net value
”). The latter definition is typically used when considering economic efficiency
, i.e. obtaining maximum value for the money spent. Value frameworks vary in decision context, e.g., whether they are used to decide whether to include a medicine in a plan health benefit package or whether they are tools to support clinical decision making.
The perspective (or ‘standpoint’ of the decision maker) taken will dictate elements of value that are considered to be relevant (elements of value are discussed in detail in paper 3). Elements of value typically include health gains and net costs, but can also include adjustments for severity of disease, the reduction in uncertainty to risk-averse patients, or scientific spillovers (for OHE research in this area see OHE and EPEMED, 2016 and Marsden et al., 2017). This part of the report explores each of these elements, mapping them to the relevant perspectives and classifying them according to whether they are core or novel. The authors conclude that, despite limitations, the quality-adjusted life year (QALY) remains the most accepted measure for capturing health benefit, but that QALYs should be combined with other - perhaps more novel - elements of value (e.g., insurance value or the value of hope) for a more comprehensive economic evaluation, which this report labels as “augmented cost-effectiveness analysis (ACEA).”
of the Special Task Force report looks at value-based reimbursement decisions
. The authors suggest that all decision-makers should adopt a decision rule (a cost-per-QALY threshold is suggested) that allows them to determine what is good value for money given their budget. Different budgets (and different definitions of value) between organisations will mean that decision rules may not necessarily be the same across institutions. Modifications to the threshold may be necessary over time, or when affordability concerns
arise. How to incorporate affordability – when the budget impact is non-marginal – are discussed.
Different ways to aggregate the various elements of value that matter to enrollees, citizens and payers acting on their behalf, are discussed in paper 5.
In addition to ACEA, extended cost-effectiveness analysis
(ECEA) and multi-criteria decision analysis
(MCDA) are also reviewed, as well as more deliberative processes for discussing evidence and reaching a decision via consensus or voting. One or other of these mechanisms is necessary to aggregate the various value elements, as including additional elements of value (i.e. those beyond health gains and net costs) is “conceptually appealing but currently impractical to implement
”. The authors conclude that all of the approaches that are reviewed are imperfect for aggregation, and that deliberative approaches can help, but often lack transparency.
MCDA techniques in support of a deliberative process represent an attractive option, but further research is required for proposed frameworks to reach their full potential. Pilot projects could be invaluable.
Finally, in paper 7
, six recommendations are provided:
- Be explicit about decision context and perspective for the value assessment framework
- Base health plan coverage and reimbursement decisions on the incremental costs and benefits of health care technologies, as provided for in a CEA
- Develop value thresholds (i.e. willingness to pay for, or opportunity cost of paying for, value appropriately defined) as one important input to guide coverage and reimbursement decisions
- Manage budget constraints and affordability on the basis of cost-effectiveness principles
- Test and consider using structured deliberative processes for health plan coverage and reimbursement decisions
- Explore and test novel elements of benefit to improve value measures, such that they reflect the perspectives of both plan members and patients.
Responses to the report are most concerned with elements of value and the use of cost-effectiveness analysis principles:
- Solow and Pezalla suggest that the additional value elements considered are too broad. Other than disease severity, they are unlikely to get picked up in the US, where the majority of payers are not motivated by a societal perspective.
- On the other hand, Burkholder et al. and Perfetto suggest that the focus on cost-effectiveness analysis is too narrow as this restricts the inclusion of non-QALY benefits and does not sufficiently align with a patient-centric approach.
- Sculpher questions whether the underlying normative framework has been fully explored - perhaps economics should be used to identify potential value elements, and not to decide what is included in assessments (which is ultimately a value judgement). This view is endorsed by Norman et al. in their Editorial.
- Overall, Sculpher concludes, however, that the recommendations (in paper 7) “offer a sensible basis for informing decisions” and they align well with good practice for health technology assessment (the older, more established sibling of value assessment frameworks).