Are there any variations in the relevance of broader value elements to cost-effectiveness analysis across diseases?

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Current value frameworks in the US assessing values of pharmaceutical interventions use cost-effectiveness analysis which traditionally compares directs costs and benefits (e.g., quality adjusted life years, life years) between comparators. “Often, these value assessment frameworks use a health system perspective without fully accounting for societal and broader benefits and costs of an intervention.”

In my recent paper published in JMCP and conducted in collaboration with co-authors- Dr. Jason Shafrin, Mr. Syvart Dennen, Ms. Kelly Birch, Dr. Julie Kanter, Dr. Menaka Bhor and Dr. Peter Neumann- reviewed and analyzed evidence reports from the U.S.-based Institute for Clinical and Economic Review to identify the degree to which broader value elements (e.g., caregiver burden, productivity loss) may be relevant for evaluation of treatments for various diseases.

We found

  1. “evidence of significant heterogeneity in the number of broader value elements cited in ICER evidence reports across diseases. Inherited retinal disease and sickle cell disease had the greatest number of broader value elements discussed by ICER reports, whereas cardiovascular disease and diabetes had the fewest.”
  2. “If value assessment ignores a large number of value elements, treatments that have wide-ranging impacts may be undervalued and receive inappropriately high incremental cost-effectiveness ratios. We found an association between the number of value elements and the incremental cost-effectiveness ratios, providing some evidence that this may be the case.”
  3. “rare-disease reports cited more elements on average than nonrare-disease reports, which may put rare diseases at greater risk of undervaluation.”

“Inclusion of all relevant value elements in value assessments will more appropriately incentivize innovation and improve allocation of research funding. Future research should aim to better quantify these value elements and could consider other techniques, such as MCDA, to prioritize the relevance of these value elements across diseases.”

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