Incomplete evaluations undermine patient access to transformational medicine

The Institute for Clinical and Economic Review (ICER) bills itself as the arbiter of “clinical and economic value” in health care. The problem: Its assessments continue to lack transparency and ignore critical factors that contribute to the value of a medicine. The result is misleading evaluations that threaten access to, and the development of, innovative therapies.

Thanks to the many cell and gene therapies currently available — with many more still in development — patients can experience long-lasting or even curative effects from just one administration. These therapies can bring tremendous value to patients, their families and society. Unfortunately, patients also could face restricted access to these treatments if payers rely on value assessment methodologies that undervalue these life-changing therapies.

In 2019, ICER updated its framework for single- and short-term therapies (SST), defined as treatments administered once or over a short period of time. Now that ICER has applied that framework to its review of several SSTs, Cencora (formerly AmerisourceBergen/Xcenda) evaluated the strengths and weaknesses of the organization’s approach. They found:

ICER is falling short: After examining ICER’s SST framework to determine the degree to which it addresses shortcomings in traditional value assessments, the report finds limitations in both the methodology and application: 

• Methodology: ICER’s SST framework lacks scientific rigor and does not reflect the societal perspective or important considerations raised by diverse stakeholders.
• Application: Inconsistency in ICER’s application of its SST framework and lack of meaningful transparency undermines its credibility.

Cencora also found that ICER:

• Is not adhering to commitments (such as continuous framework updates, inclusion of the societal perspective, and inclusion of three additional value domains) outlined in its SST framework;
• Continually employs untested methodologies that lack empirical evidence or scientific justification; and
• Uses processes that lack the transparency and stakeholder engagement that ICER misleadingly heralds as a bedrock of its practice.

As some payers and policymakers use ICER value assessments to make important coverage decisions, the absence of much-needed caveats and context may undermine meaningful access for patients.

Here is why it matters: Developments in medicine are only valuable when the patients they are developed for can get them. Durable, single-administration medicines, like cell and gene therapies, hold the promise to revolutionize health care, providing significant relief for patients with diseases that were previously thought to be incurable. However, the potentially curative nature of some of these single-administration medicines requires different models in order to account for the full value these medicines can bring to patients and society. Failure to recognize and reward the value of these potential cures could endanger meaningful access to these innovative therapies and undermine incentives for the development of future transformational medicines.

Patients who need these treatments must be able to access them, which is why our payor reimbursement approaches should be able to keep pace with innovation — which includes the incorporation of innovative contracts that can help spread costs or tie payment to outcomes. These approaches can help payers manage budget impacts while increasing focus on patient outcomes and access.

Studies that rely on cost-effectiveness analysis — like those ICER uses — should only be one of many tools used to inform decision-making. The inappropriate use of these tools, including by the government for price setting, could have devastating effects on patients by using flawed one-size-fits-all approaches to override individualized decision-making about which treatment is best for each patient. 

Read Cencora’s full report here.