Value Viewpoint: January 17, 2025

New research published in BMC Health Services Research this week compares how different HTA bodies around the world (South Korea, England, Australia, and Canada) assessed 22 innovative, high-priced drugs with limited evidence at launch. Cost-effectiveness analysis (CEA) typically plays an important role in reimbursement and pricing decisions in these countries, although South Korea granted CEA waivers for these products.

The study authors found that the recommendations for the drugs included in the analysis were largely positive, ranging from 84.2% in Australia to 100% in Canada. They also found a high use of managed entry agreements (MEAs) to help facilitate access when high levels of uncertainty existed.

There were differences between countries in terms of comparators, with England having a higher proportion of indirect comparison and Australia and Canada utilizing a mixture of indirect comparison and head-to-head trial data.

Also in international HTA news this week, a new white paper entitled “Surrogate endpoints in cost-effectiveness analysis for use in health technology assessment” was published by a group of four HTA agencies:

• National Institute for Health and Care Excellence (NICE),
• Canada’s Drug Agency (CDA-AMC),
• the Institute for Clinical and Economic Review (ICER),
• the National Health Care Institute (ZIN) in the Netherlands.

The working group for the project also included representatives from:

• the Australian Department of Health and Aged Care,
• the Institute for Technology Assessment in Health (IETS) in Colombia, and
• Rubix Health in the US.

Surrogate endpoints are increasingly used to inform decision-making, but there has historically been limited guidance on economic modeling considerations and validation. This white paper aims to provide

“recommendations on best practice when using surrogate endpoints in health economic models to inform HTA decision making.”?

The white paper includes recommendations in the following areas:

• Designing studies using surrogates
• Choosing an appropriate model structure
• Selecting final outcomes for economic model
• Validating a surrogate endpoint
• Assessing uncertainty in the economic model
• Reporting?

A few “quick hits” of interest:

• The New York Times on 340B: How a Company Makes Millions Off a Hospital Program Meant to Help the Poor
• The Federal Trade Commission released its second interim report on PBMs: Pharmacy Benefit Managers: The Powerful Middlemen Inflating Drug Costs and Squeezing Main Street Pharmacies (press release)
• My predictions for Managed Healthcare Executive on ways value will play an increasingly important role this year: Smart Money Bets on Value | Predictions for 2025

??? Eye on ICER

A calendar of ICER’s upcoming reports & meetings:

Policy White Papers/Special Assessments:

- 3/4/25: Special Assessment to Inform CMS Drug Price Negotiation: Breo Ellipta and Trelegy Ellipta — Final Report

Value Assessment Reports:

- 2/5/25: Acute Pain — Revised Evidence Report

- 2/6/25: Retinitis Pigmentosa — Draft Evidence Report

- 3/26/25: Retinitis Pigmentosa — Revised Evidence Report

- 3/27/25: Acute Pain — Final Evidence Report

- 4/10/25: Multiple Sclerosis: SPMS — Draft Evidence Report

- 5/12/25: Retinitis Pigmentosa — Final Evidence Report

- 5/27/25: Spinal Muscular Atrophy — Draft Evidence Report

- 5/29/25: Multiple Sclerosis: SPMS — Revised Evidence Report

- 7/15/25: Multiple Sclerosis: SPMS — Final Evidence Report

- 7/17/25: Spinal Muscular Atrophy — Revised Evidence Report

- 9/2/25: Spinal Muscular Atrophy — Final Evidence Report

Meetings:

- 2/28/25: Acute Pain — Public Meeting (Midwest CEPAC)

- 4/11/25: Retinitis Pigmentosa — Public Meeting (New England CEPAC)

- 6/13/25: Multiple Sclerosis: SPMS (California Technology Assessment Forum CTAF)

- 8/1/25: Spinal Muscular Atrophy — Public Meeting (Midwest CEPAC)

Contributing author: Brian Sils