Stakeholder perception of pharmaceutical value: A multicriteria decision analysis pilot case study for value assessment in the United States

Study shows the impact of a more holistic approach to assessing value and how it can help address gaps within conventional value assessment.

Authors

Mendola ND, Oehrlein E, Perfetto EM, Westrich K, McQueen RB. 

Publication

Journal of Managed Care & Specialty Pharmacy, 2022 Oct; 28(10): 1190-1196.

Current value assessment frameworks tend to focus on criteria that matter most to clinicians and payers, such as clinical effectiveness, QALY gains, and net costs. Other factors that might matter more to patients – like a treatment’s impact on caregiver burden, the simplicity of dosing, having a treatment option where others have failed, or the severity of the disease being treated – are typically not included in the assessment of value. 

In a study published in JMCP, researchers applied multicriteria decision analysis (MCDA) to assess how a broader range of factors might impact how a treatment is valued. The research team facilitated a roundtable to discuss the value of two drug among a group of stakeholders, including patients, health technology assessment professionals, researchers/academics, payers and industry professionals. 

Participants were asked to rate each drug, before and after thinking about additional factors not often included in conventional ratings. Almost half of the people changed their ratings after considering additional factors, suggesting that a fuller picture of how treatments impact patients should be included when deciding how much to pay for a drug.

The study confirms that MCDA can serve as a more holistic approach to assessing value, helping to address gaps within conventional value assessment. By taking patients’ concerns, experiences, and treatment preferences into consideration, MCDA can offer decision-makers a way to quantify what matters most to patients when they consider the value of a health care treatment and its impact on their quality of life.

Background
Recent attention to value frameworks has highlighted limitations of current conventional value and health technology assessment (V/HTA) methods (eg, cost-effectiveness). Multicriteria decision analysis (MCDA) has the potential as a supplemental tool to incorporate additional value criteria into conventional value assessment. The aim of this study was to conduct a pilot study to illustrate the impact of an MCDA approach on the value perceptions of hypothetical treatment profiles from a multistakeholder panel.

Methodology
Participants voted on value perceptions of 2 hypothetical treatments with similar cost-effectiveness evidence: Treatment A for aggressive B-cell non-Hodgkin lymphoma in adults and treatment B for episodic migraine in adults. Participants voted treatments A and B as low, intermediate, or high value before and after a weighting exercise on prespecified, additional value criteria. Weights from participants were used to calculate treatment-specific MCDA scores from 0 (least favorable) to 100 (most favorable) and were presented to participants for a second value-perception vote. Analyses compared changes in value perceptions within treatments A and B post–MCDA exercise.

Results

  • Of all stakeholders, 41% altered their perception of value for treatment A (9% negatively and 32% positively) and separately, 45% for treatment B (23% both negatively and positively) after considering MCDA scores.
  • Nearly half of all participants altered their perception of value after consideration of additional value criteria. These findings support the need for a more inclusive and flexible value assessment process. 
  • Before considering MCDA scores for treatment A, 0% of participants considered it to be low, 52% intermediate, and 48% high value. After considering MCDA scores for treatment A, 4% considered it low, 29% intermediate, and 67% high value. 
  • Both before and after considering MCDA scores for treatment B, 13%, considered it low, 57% intermediate, and 30% high value. Mean MCDA scores for treatments A and B were 67 and 63, respectively. 

A Growing Body of Research

This article is the latest NPC research highlighting the importance of considering the patient perspective in value assessments.