— Current evidence suggests that tafamidis and acoramidis provide a net health benefit when compared to no disease-specific therapy; these treatments would achieve common thresholds for cost-effectiveness if priced between $13,600 to $39,000 per year —
BOSTON, September 5, 2024 – The Institute for Clinical and Economic Review (ICER) today posted its revised 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).
“When transthyretin amyloid affects the heart, patients can develop progressive heart failure with symptoms that include fatigue, shortness of breath, and cardiac arrhythmias,” said ICER’s Chief Medical Officer David Rind, MD. “The existence of a therapy for ATTR-CM, with the approval of tafamidis in 2019, has altered the management of the condition and patients are now routinely diagnosed and treated earlier in the course of the disease. Treatment with the transthyretin (TTR) stabilizers tafamidis or acoramidis or with the RNA silencing agent vutrisiran slows progression of ATTR-CM and improves outcomes, all with minimal side effects. The ICER evidence ratings for the TTR stabilizers reflect uncertainties around the magnitude of the clinical benefits in those patients who are currently being diagnosed with ATTR-CM; tafamidis showed large benefits in the original (more severe) population in which it was studied. Vutrisiran shows large benefits in a contemporary population, but comparisons between it and the TTR stabilizers have continued uncertainties.”
This Evidence Report will be reviewed at a virtual public meeting of the Midwest CEPAC on September 20, 2024. The Midwest CEPAC is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.
A draft version of this report was previously open for a four-week public comment period. The updated Evidence Report and voting questions reflect changes made based on comments received from patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.
Key Clinical Findings
- Tafamidis: Compared with no disease-specific therapy, tafamidis provides at least a small net health benefit, but only moderate certainty that it provides a substantial net health benefit. (“B+”)
- Acoramidis: Compared with no disease-specific therapy, acoramidis provides at least a small net health benefit, but only moderate certainty that it provides a substantial net health benefit. (“B+”)
- Vutrisiran: Compared with no disease-specific therapy or when added to tafamidis, vutrisiran provides a substantial net health benefit. (“A”)
- Finally, the evidence is currently insufficient (“I”) to compare the net health benefits of the three treatments (tafamidis, acoramidis, vutrisiran).
Key Cost-Effectiveness Findings
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 such as 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.
ICER’s HBPB is a price range suggesting the highest US price a manufacturer should charge for a treatment, net of all rebates and discounts, based on the amount of improvement in overall health patients receive from that treatment, when a higher price would cause disproportionately greater losses in health among other patients in the health system due to rising overall costs of health care and health insurance. In short, it is the top price range at which a health system can reward innovation and better health for patients without doing more harm than good.
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.