Value Viewpoint: August 28, 2025
Kimberly Westrich
NPC Chief Strategy Officer | Kimpossibility Coaching, Retreats, & Yoga | Biopharmaceutical & Health Policy Researcher | Public Speaker
New research published in Value in Health (paywalled) utilizes the Generalized Risk Adjusted Cost Effectiveness (GRACE) model to quantify how the value of a hypothetical neurological condition treatment changes when considering patient risk preferences and baseline disease severity, neither of which is incorporated in traditional cost-effectiveness analysis (TCEA). Study authors found that incorporating these factors increased the treatment’s value:
“GRACE increased the net monetary benefit by 11.6%, driven by a 9.7% increase in the cost-effectiveness threshold due to disease severity and a 21.4% increase in patient utility due to reductions in the variance of health outcomes.”
A new primer published in the Journal of Comparative Effectiveness Research discusses the portability of real-world evidence (RWE) across national borders, including methods to address issues when importing RWE from other countries for use in HTA. Authors summarize the key issues when transporting RWE in the table below:
The authors conclude,
“While methods for evaluating transportability are advancing, clear guidance and practical examples can help HTA agencies, regulators and industry stakeholders use nonlocal data more confidently. Ethical considerations and stakeholder involvement remain critical to ensure that the application of transportability methods aligns with patient and system priorities. Indeed, the thoughtful use of RWE, whether local or nonlocal, can ultimately accelerate patient access to novel therapies.”
A guest column from Jason Shafrin in The Evidence Base entitled "Perspectives from the Healthcare Economist: CMS doesn’t consider QALYs – What’s a health economist to do?" reviews alternatives to the QALY. The alternative measures discussed in the article are summarized in the handy table below:
Speaking of the Evidence Base, it was a delight to see the August 25th issue of The Daily Dose spotlight a range of the National Pharmaceutical Council’s policy-relevant work on topics ranging from the unintended consequences of the IRA to patient access to gene therapies to value frameworks in the US.
The Center for Innovation & Value Research is hosting its Fall Policy Summit, “Centering Patient Values in Drug Policy: Transparency, Value, and Access,” on October 7, 2025, in Washington, DC. Registration options include in-person and virtual.
In partnership with the Rutgers Pharmaceutical Industry Fellowship Program, NPC wll soon be recruiting for our 2026-2028 Health Policy & Communications Fellow. This two-year fellowship is an accelerated path to industry experience for current or soon-to-be graduated PharmDs. Learn how to conduct policy-relevant research and communicate it with impact with NPC! Applications open on October 8, 2025. More information can be found here.
👁️ Eye on ICER
A calendar of ICER’s upcoming reports & meetings:
Policy White Papers/Special Assessments:
- 9/24/25: HEMA Draft Report: Defining Appropriate Benefits for Economic Evaluation of Health Care Technologies (public comment period 9/24/25-10/15/25)
- 1/22/26: HEMA Final Report: Defining Appropriate Benefits for Economic Evaluation of Health Care Technologies
Value Assessment Reports:
- 10/20/25: Smoking Cessation — Draft Evidence Report
- 10/29/25: Obesity Management — Revised Evidence Report
- 12/10/25: IgA Nephropathy — Draft Evidence Report
- 12/11/25: Smoking Cessation — Revised Evidence Report
- 12/16/25: Obesity Management — Final Evidence Report
- 2/12/26: Smoking Cessation — Final Evidence Report
- 3/31/26: IgA Nephropathy — Revised Evidence Report
Meetings:
- 9/25/25: Non-Cystic Fibrosis Bronchiectasis — Public Meeting (California Technology Assessment Forum CTAF)
Contributing author: Brian Sils
Health Policy Regulatory and Legislative Expertise; Market Innovator
3wThank you I loved both these charts, and what a service for selecting them. -On the RWE, it says simply defining the treatment protocol and dose precisely; an issue highly relevant to today. —-Take for instance studies in radiation therapy for breast cancer. Surprisingly few and the ones most relevant I was looking at were from China. An expert in research said no you can’t use those because of country differences in therapy dose. —-Or take for example any product where say a bioengineered product is compared to a simple chemical mixture. No precision in the science of the comparison. (This does make me wonder if the adverse event GLP1 data is polluted by compounded products.) Perhaps a little out of across areas focus but I am obsessing about it.