Institute for Clinical and Economic Review Publishes Final Evidence Report on Treatments for Transthyretin Amyloid Cardiomyopathy

— Independent appraisal committee voted that current evidence is adequate to demonstrate superior net health benefits for tafamidis, acoramidis, and vutrisiran when compared to no disease-specific therapy; tafamidis and acoramidis would achieve common thresholds for cost-effectiveness if priced between $13,600 to $39,000 per year —

BOSTON, October 21, 2024 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness of tafamidis (Vyndamax®/Vyndaqel®, Pfizer Inc.), acoramidis (BridgeBio Pharma, Inc.), and vutrisiran (Amvuttra®, Alnylam Pharmaceuticals, Inc.) for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).

ICER’s report on these therapies was the subject of the September 2024 public meeting of the Midwest CEPAC, one of ICER’s three independent evidence appraisal committees. 

Downloads: Final Evidence Report | Report-at-a-Glance | Policy Recommendations 

“The independent panel confirmed the substantial clinical benefits of treating ATTR-CM with either a stabilizer or an RNA silencer, but also that the current price for tafamidis is much too high,” said ICER’s Chief Medical Officer David Rind, MD. “The panel disagreed with ICER’s conclusion that there is clear evidence for adding the RNA silencer vutrisiran to tafamidis in treating ATTR-CM, though the ICER research team stands by this conclusion. This adds even greater concerns around pricing, since combination therapy with drugs priced out of alignment with their clinical benefits will be extremely expensive and create strains in the health system.”

ICER’s Virtual Public Meeting: Voting Results on Clinical Effectiveness and Contextual Considerations 

ICER assessed, and the independent appraisal committee voted on the evidence for adults with ATTR-CM: 

A majority of panelists (14-0) found that current evidence is adequate to demonstrate a net health benefit for tafamidis when compared to no disease-specific treatment. 

A majority of panelists (15-0) found that current evidence is adequate to demonstrate a net health benefit for acoramidis when compared to no disease-specific treatment. 

A majority of panelists (14-0) found that current evidence is adequate to demonstrate a net health benefit for vutrisiran when compared to no disease-specific treatment. 

A majority of panelists (15-0) found that current evidence is not adequate to demonstrate a net health benefit for vutrisiran added to tafamidis when compared to tafamidis alone. 

A majority of panelists (15-0) found that current evidence is not adequate to distinguish a net health benefit among the interventions when used as monotherapy (tafamidis, acoramidis, vutrisiran). 

Panel members also weighed potential benefits and disadvantages beyond the direct health effects, and weighed special ethical priorities. Voting highlighted the following as particularly important for payers and other policymakers to note: 

There is substantial unmet need despite currently available treatments.

This condition is of substantial relevance for people from a racial/ethnic group that have not been equitably served by the healthcare system.

The TTR stabilizers are likely to produce substantial improvement in caregivers’ quality of life and/or ability to pursue their own education, work, and family life.

ICER’s Virtual Public Meeting: Voting Results on Long-Term Value for Money 

Tafamidis has been approved by the FDA for ATTR-CM and has a list price of approximately $268,000 per year. Acoramidis and vutrisiran have not yet been approved by the FDA for ATTR-CM, and the manufacturers have not announced US prices for each therapy if approved.

ICER has calculated a health benefit price benchmark (HBPB) for TTR stabilizers tafamidis and acoramidis to be between $13,600 to $39,000 per year. Given the timing and availability of information about the effectiveness of the drug, the value of vutrisiran was not assessed.

For adults with ATTR-CM:

A majority of panelists (13) found at the current pricing, tafamidis compared to no disease-specific treatment represents a “low” long-term value for money. Two panelists voted that the therapy represented an “intermediate” long-term value for money.

Key Policy Recommendations 

ICER’s independent assessment of value informs the critical decisions that stakeholders across the US health system need to make around pricing and coverage. Following the voting session, a policy roundtable of experts — including clinical experts, patient advocates, and representatives from US payers — convened to discuss the pricing implications and recommendations to ensure fair access. Key recommendations stemming from the roundtable discussion include: 

Federal and state policymakers should remove barriers to the use of telemedicine, including across state lines, so that individuals with ATTR-CM can access the most knowledgeable centers of excellence, regardless of geographic location. 

Clinical specialty societies should establish diagnostic cutoffs normalized for gender and/or body size both for screening for ATTR-CM in clinical practice and enrollment in clinical trials, to reduce failure to accurately diagnosis the condition in women and smaller patients. 

Manufacturers should set prices that will foster affordability and access for all patients by aligning prices with the patient-centered therapeutic value of their treatments. 

Researchers and funding agencies should focus future research on efforts to establish the comparative effectiveness of tafamidis, acoramidis, and vutrisiran in similar populations. 

ICER’s detailed set of policy recommendations is available in the Final Evidence Report and in the standalone Policy Recommendations document. 

About ICER 

The Institute for Clinical and Economic Review (ICER) is an independent, non-profit research institute that conducts evidence-based reviews of health care interventions, including prescription drugs, other treatments, and diagnostic tests. In collaboration with patients, clinical experts, and other key stakeholders, ICER analyzes the available evidence on the benefits and risks of these interventions to measure their value and suggest fair prices. ICER also regularly reports on the barriers to care for patients and recommends solutions to ensure fair access to prescription drugs. For more information about ICER, please visit www.icer.org.