ICER Publishes Final Evidence Report on Treatment for Pulmonary Arterial Hypertension

— Independent appraisal committee voted that sotatercept demonstrated a net health benefit when compared to background therapy; sotatercept would achieve common thresholds for cost-effectiveness if priced between $17,900 to $35,400 per year —

BOSTON, January 8, 2024 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness and value of sotatercept (Winrevair, Merck & Co.) for pulmonary arterial hypertension (PAH).

ICER’s report on these therapies was the subject of the December 2023 public meeting of the of the Midwest CEPAC (Midwest CEPAC), one of ICER’s three independent evidence appraisal committees.

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

“Pulmonary arterial hypertension is a progressive disease that can lead to debilitating shortness of breath, fatigue, and lightheadedness as the heart struggles to pump blood through the lungs,” said ICER’s Chief Medical Officer, David Rind, MD. “Current drug treatments are primarily combinations of vasodilators, and these can be very burdensome and cause significant side effects. Sotatercept has a novel mechanism of action, and it is administered subcutaneously every few weeks, which reduces burdens. Current evidence suggests that sotatercept has fewer side effects and improves short-term outcomes, but we have some uncertainties about long-term efficacy and safety.”

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

ICER assessed, and the independent appraisal committee voted on, the evidence of sotatercept for adults with World Health Organization Functional Class (WHO-FC) II and III pulmonary arterial hypertension (PAH) who are on background therapy:

All panelists (13-0) found that current evidence is adequate to demonstrate a net health benefit for sotatercept when compared to background therapy alone.

Panel members also weighed potential benefits and disadvantages beyond the direct health effects and broader contextual considerations. Voting highlighted the following as particularly important for payers and other policymakers to note:

The magnitude of the lifetime impact on individual patients with PAH.

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

Sotatercept has not yet been approved by the FDA, and the manufacturer has not announced a US price if approved. ICER has calculated a health-benefit price benchmark (HBPB), meaning a price range that matches the clinical benefit patients are expected to receive, for sotatercept to be between $17,900 to $35,400 per year.

Consistent with ICER’s process, because there is no firm estimate yet of a potential launch price for the treatment, the panel did not take separate votes on the treatment’s long-term value for money.

Access and Affordability Alert

At sotatercept’s assumed placeholder price of $400,000 annually, 7% of the estimated 60,675 eligible patients in the US could be treated within five years without crossing the ICER potential budget impact threshold of $777 million per year.

In the absence of a known price for sotatercept, ICER is not issuing an access and affordability alert. Stakeholders should be aware, however, that at prices substantially lower than analyst estimates, payers will face a significant short-term budget impact if sotatercept is approved.  The purpose of an ICER access and affordability alert is to signal to stakeholders and policymakers that the amount of added health care costs associated with a new service may be difficult for the health system to absorb over the short term without displacing other needed services, creating pressure on payers to sharply restrict access, or causing rapid growth in health care insurance costs that would threaten sustainable access to high-value care for all patients.

Update: ICER added the “Access and Affordability Alert” shortly after FDA approval. The list price of sotatercept is approximately $240,000 per year.

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:

Given sotatercept’s efficacy and safety profile, efforts from all stakeholders are needed to ensure that sotatercept is accessible and affordable to all eligible patients. This includes setting sotatercept’s price in fair alignment with the long-term benefits, rapidly transitioning sotatercept to home-based administration, and ensuring affordability through the combination of benefit design and financial assistance programs. 

For optimal care, access to PAH specialists should be facilitated – particularly across state lines – by ensuring that clinicians are licensed and able to be reimbursed in the appropriate states, COVD-era telemedicine and inter-state consultations remain in effect, and including wraparound coverage for travel to specialists. 

Increasing access to liquid oxygen when medically necessary through reform of supplemental oxygen reimbursement policies, including setting differential reimbursement rates for more expensive forms of oxygen. 

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 produces reports analyzing the evidence on the effectiveness and value of drugs and other medical services. ICER’s reports include evidence-based calculations of prices for new drugs that accurately reflect the degree of improvement expected in long-term patient outcomes, while also highlighting price levels that might contribute to unaffordable short-term cost growth for the overall health care system.

ICER’s reports incorporate extensive input from all stakeholders and are the subject of public hearings through three core programs: the California Technology Assessment Forum (CTAF), the Midwest Comparative Effectiveness Public Advisory Council (Midwest CEPAC), and the New England Comparative Effectiveness Public Advisory Council (New England CEPAC). These independent panels review ICER’s reports at public meetings to deliberate on the evidence and develop recommendations for how patients, clinicians, insurers, and policymakers can improve the quality and value of health care. For more information about ICER, please visit ICER’s website.