Value Viewpoint: August 9, 2024

In a new “Viewpoint” article published in the Journal of Managed Care & Specialty Pharmacy (JMCP), the authors of ICER’s 2022 evidence report on gene therapy treatments for hemophilia A and B advocate for using ICER’s highly controversial shared savings scenarios to guide formulary decisions for etranacogene dezaparvovec in treating hemophilia B.

It is questionable at best and irresponsible at worst to see ICER promoting the use of its shared savings scenarios despite well-documented methodological limitations and strong opposition from healthcare stakeholders. Last year, in response to public comments on its proposed revisions to its 2023 Value Assessment Framework, ICER acknowledged that most commentators  “opposed the ongoing use of shared savings scenarios within analyses of high impact single or short-term therapies such as gene therapies.”

Notably, the article is not part of the ICER/JMCP “Perspectives of Value” series, in which ICER summarizes the key findings from its assessments. It is an opinion piece asserting that allocating full cost offsets to the manufacturer of a new one-time treatment perpetuates inefficient healthcare spending and “introduces the risk of incentivizing development of gene therapies for conditions that already have treatments, particularly expensive treatments, rather than conditions that currently lack effective treatments.”

Real-world cost offsets for hemophilia B far exceed ICER's arbitrary cost offset cap of $150,000 annually. Thus, applying ICER's cost offset cap in this scenario results in an artificially low “value-based” price determination, which is not based on value and could paradoxically result in the perpetuation of an inefficient and expensive standard of care. 

There were four new pre-proof articles from Value in Health worth checking out:

In The health benefits, costs, and cost-effectiveness of ultra-orphan drugs (paywalled), study authors compared QALYs gained, costs, and incremental cost-effectiveness ratios (ICERs) of ultra-orphan, orphan, and non-orphan drugs approved by the FDA between 1999-2019. They found that compared to non-orphan and other orphan drugs, ultra-orphans had larger QALY gains, higher costs, and larger ICERs. The study concluded,

“Novel ultra-orphan drugs typically offer larger incremental health gains than drugs for more prevalent diseases, but due to their substantial added costs, are typically less cost-effective..”

In Discriminatory properties of QALY-based CEA for patients with disabilities: A Duchenne muscular dystrophy case study, study authors utilized ICER’s 2019 DMD model to compare cost-effective treatment prices at different willingness-to-pay (WTP) thresholds for two patient populations: early ambulatory (EA) and early non-ambulatory (ENA) patients. The study found that ENA patients (who had a health state with lower utility) compared to EA patients (who had a health state with higher utility) had a 98% decline in cost-effective treatment price at a WTP of $150,000/QALY or higher and had no cost-effective treatment price at a WTP of $100,000/QALY or lower. There’s an interesting scenario analysis with the QALY alternative equal-value life-year gained (evLYG); the trend is the same, but the magnitude of the difference is considerably softened.

These findings present additional evidence of the potential discriminatory effects of the QALY. Both groups of patients could receive equal treatment benefit in having the progression of their condition delayed, but only one group’s treatment is considered cost-effective. The study concludes,

“For certain severe, disabling conditions, traditional approaches are likely to conclude that treatments are not cost-effective at any price once a patient progresses to a disabled health state with low utility value. These findings elucidate theoretical/ethical concerns regarding potential discriminatory properties of traditional QALY assessments for people with disabilities, particularly those who have lost ambulation or have other physical limitations.”

Informing the US Medicare drug price negotiation for apixaban and rivaroxaban: Methodological considerations for value assessments many years after launch (paywalled), is a rehash of ICER’s October 2023 white paper, “Special Assessment to Inform CMS Drug Price Negotiation: Eliquis and Xarelto,” designed to illustrate how a traditional HTA could potentially be modified to inform Medicare price negotiations, which occur well into a product’s life cycle, rather than at launch. According to ICER, these modifications include:

• “…selecting appropriate populations and comparators for drugs that have multiple indications,
• stricter inclusion criteria (e.g., limits on trial duration and sample size),
• use of the evLY as a composite health outcome measure in our cost-effectiveness analysis,
• inclusion of observational data with a specific role for clinical and cost-effectiveness approaches, and
• calculation of threshold price premiums to account for unknown and unstable comparator drug prices.”

In Development of a prioritisation framework to aid healthcare funding decision-making in health technology assessment (HTA) in Australia: application of multi-criteria decision analysis, researchers in Australia found that by utilizing an MCDA tool, five criteria were ranked highest by healthcare decision makers (clinical efficacy/effectiveness; safety and tolerability; severity of the condition;  quality/uncertainty; and direct impact on healthcare costs) and they were able to better reflect public preferences and values in HTA. The authors concluded,

“This study also demonstrates the MCDA framework's practical utility as a transparent and cost-effective decision-aid tool, offering valuable implications for informed healthcare funding decisions and resource allocation.”

Last week, ICER announced via its ICER Analytics newsletter the addition of an updated model for obesity management medications to its ICER Analytics platform. The model builds on the 2022 ICER assessment model for obesity management medications, including new clinical trial data and new patient populations not included in the original assessment.

As I have written about in the past (see the October 7, 2023, Value Viewpoint), there have been a number of concerns related to new models being added to ICER Analytics that do not go through the standard assessment process. Issues of transparency and missed stakeholder engagement are created when paywalled models cannot be publicly accessed or commented on.

Obesity management medications are a vitally important policy topic, and new data, models, and assessments should be publicly available, especially given the significant potential health implications of these medicines. 

Last week, ICER released its plans to assess the comparative clinical effectiveness and value of suzetrigine for the treatment of Acute Pain. Public comments are now being accepted on the Draft Scoping Document through August 16, 2024. The assessment will be discussed at the Midwest CEPAC in February 2025.