Institute for Clinical and Economic Review Publishes Final Evidence Report on Treatment for Chronic Obstructive Pulmonary Disease

— Independent appraisal committee voted that current evidence is adequate to demonstrate superior net health benefits for ensifentrine added to maintenance therapy compared to maintenance therapy alone; ICER calculated a health-benefit price benchmark (HBPB) for ensifentrine between $7,500 to $12,700 per year, and the therapy is priced at $35,400 per year —

BOSTON, July 16, 2024 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness and value of ensifentrine (Ohtuvayre, Verona Pharma) for the treatment of chronic obstructive pulmonary disease (COPD).

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

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

“COPD is a common cause of severe respiratory problems,” said ICER’s Chief Medical Officer, David Rind, MD. “Current evidence shows that ensifentrine decreases COPD exacerbations when used in combination with some current inhaled therapies, but there are uncertainties about how much benefit it may add to unstudied combinations of inhaled treatments. Unfortunately, the manufacturer chose a price for ensifentrine far above the value-based price. This may lead payers to implement formulary hurdles that could limit provider and patient access to this promising new treatment.”

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 ensifentrine for adults with moderate-to-severe COPD: 

A majority of panelists (11-4) found that current evidence is adequate to demonstrate a net health benefit for ensifentrine added to maintenance therapy when compared to maintenance therapy alone. 

Panel members also weighed potential benefits and disadvantages beyond the direct health effects and 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.

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

Consistent with ICER’s process, because there was no firm estimate of a potential launch price during the June 14, 2024 ICER public meeting, the panel did not take a vote on the treatment’s long-term value for money. 

On June 26, 2024, ensifentrine was approved by the FDA. The manufacturer announced a US price of $35,400 per year. ICER has calculated a health-benefit price benchmark (HBPB) for ensifentrine to be between $7,500 to $12,700 per year.

Access and Affordability Alert

At the price of $35,400 per year for ensifentrine, approximately 0.5% of US patients eligible could be treated within five years without crossing the ICER potential budget impact threshold of $735 million per year.

ICER is issuing an access and affordability alert for ensifentrine for the management of COPD.  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.

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: 

Payers should include coverage of effective smoking cessation therapies, including nicotine replacement products, pharmacologic therapies, cognitive behavioral therapy (CBT) and combinations thereof, as smoking cessation is a critical part of the treatment of COPD.

Manufacturers should set prices that will foster affordability and good access for all patients by aligning prices with the patient-centered therapeutic value of their treatments. For ensifentrine, the manufacturer has priced far above this level and therefore missed an opportunity to provide broad access and increased uptake of the drug.

Researchers should conduct studies that directly compare real-world treatment options and sequential treatment effectiveness.

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.