Institute for Clinical and Economic Review Report Questions the Adequacy of Evidence on Overall Health Benefit of Elagolix Given Limitations of Information on Safety and Limited Evidence Comparing to Other Treatment Options

— The emerging drug may be cost-effective compared to no treatment if priced in line with analyst expectations, but large number of potentially eligible patients may pose short-term affordability challenges for health systems —

 

BOSTON, June 15, 2018 – The Institute for Clinical and Economic Review (ICER) today released an Evidence Report assessing the comparative clinical effectiveness and value of elagolix (AbbVie/Neurocrine Biosciences) for the management of endometriosis and associated pain. Elagolix is currently under review by the FDA, with an approval decision expected in mid-2018.

“Elagolix shows promise in reducing the pain women with endometriosis experience, but more research is needed to determine the drug’s long-term effectiveness and safety profile, particularly in comparison to alternative agents,” noted Dan Ollendorf, PhD, ICER’s Chief Scientific Officer. “While elagolix may be cost-effective in the long-term if priced in line with analyst expectations, the treatment will potentially challenge short-term budgets given the large number of women affected by endometriosis. During our meeting on July 12th, stakeholders will convene to discuss strategies to ensure appropriate patient access while managing system-wide affordability.”

This Evidence Report will be the subject of an upcoming public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC) in Burlington, VT on July 12, 2018. The New England 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. The Council will also review ICER’s recently released condition update on treatments for plaque psoriasis during the meeting.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 receivedfrom patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.

Key Findings

Evidence on the clinical effectiveness of elagolix compared to no treatment was determined to be promising but inconclusive. While the therapy appeared to decrease pain for women with endometriosis, particularly in those with dysmenorrhea, or menstrual-related pain, there is insufficient evidence to rule out the possibility of treatment-related harms. Compared to other treatments currently used to treat endometriosis, including GnRH agonists, hormonal treatments, and aromatase inhibitors, evidence was considered insufficient to judge elagolix’s net health benefit.

Using a $7,000 per year placeholder price based on analyst expectations, economic analyses found elagolix to fall within commonly accepted thresholds for cost-effectiveness of $50,000 – $150,000 compared to no treatment. These findings held true when assessed from both a shorter-term perspective of six months and a longer-term perspective that took into consideration factors such as pain recurrence, surgeries, and treatment-related cardiovascular and fracture risks. However, the actual price of elagolix remains unknown, as does its potential cost-effectiveness relative to oral contraceptives, GnRH agonists, or aromatase inhibitors.

For the price to align with the added benefit elagolix offers to patients, the therapy would need to be priced between $8,800 and $12,800 per year.

Despite the favorable preliminary cost-effectiveness findings, however, only about 26% of women with endometriosis-related pain could be treated before spending crossed ICER’s potential budget impact threshold of $915 million per year.

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.