Institute for Clinical and Economic Review Final Report Highlights Limitations in Evidence on Long-term Safety and Effectiveness of Elagolix for Endometriosis, Discusses Options for Insurance Coverage Criteria

— Because elagolix’s price aligns with clinical benefit only when compared to no other treatment, insurers may first encourage patients to try oral contraceptives and leuprorelin acetate before progressing to a new treatment that has an unknown long-term safety profile —  

Boston – August 3, 2018 – The Institute for Clinical and Economic Review today released a Final Evidence Report and Report-at-a-Glance on elagolix (OrilissaTM, AbbVie), for the management of moderate to severe pain associated with endometriosis.

ICER’s report was reviewed at a July 2018 public meeting of the New England Comparative Effectiveness Public Advisory Council (New England CEPAC), one of ICER’s three independent evidence appraisal committees. During the meeting, which occurred prior to FDA approval and the resolution of questions regarding potential liver toxicity and the time duration of the approved indication, the Council voted that the evidence was not adequate to determine whether elagolix offers a net health benefit compared to no treatment. The Council also voted unanimously that the evidence was not adequate to distinguish the net health benefit of elagolix from that of treatment with either a GnRH agonist (leuprorelin acetate) or a hormonal contraceptive (depot medroxyprogesterone), due to limited and mixed evidence on clinical effectiveness and potential risks.

During the deliberation, Council members discussed the broader considerations around the significant impact endometriosis can have on women’s quality of life, the advantages of elagolix’s availability as an oral agent, as well as other unique characteristics of the drug. They also noted the uncertainty around the therapy’s long-term side effects and clinical benefit, as elagolix use was limited to 6-12 months in clinical trials yet is intended to treat a chronic and disabling condition.

“While elagolix appears to offer short-term clinical benefits in terms of reduced pain and improved quality of life for women living with endometriosis, evidence is not yet sufficient to rule out the possibility that this therapy may pose long-term safety risks for patients,” noted Dan Ollendorf, PhD, ICER’s Chief Scientific Officer. “Until more is understood about the long-term effects, it would be reasonable for payers to consider prior authorization criteria to ensure prudent use of the therapy, such as requirements for previous treatment with other options, specialist prescribing, and clinician attestation of clinical improvements while using the therapy.”

ICER’s report concludes that, assuming typical discounts and rebates off its approximate $10,000 annual list price, elagolix meets traditional thresholds for cost-effectiveness only when compared to no alternative treatment.

Affordability and Access Alert

As part of its final report, ICER is issuing an Affordability and Access Alert for elagolix. This alert is intended to signal when the added costs associated with a new treatment may be difficult for the health care system to absorb over the short term without displacing other needed services or contributing to unsustainable growth in health care insurance costs. At current prices, only about one-quarter of eligible patients for whom clinical experts believe elagolix would be considered could be treated before the budget impact would cross ICER’s threshold of a net addition of over $915 million per year over five years.

Key Policy Recommendations

Following the voting session, a policy roundtable of experts – including patient advocates, physicians, and payers – convened to discuss the implications of the evidence for policy and practice. Key recommendations stemming from the roundtable discussion include:

  • Elagolix has known short-term side effects and no long-term comparative safety and efficacy data in relation to several other well-established treatment options for endometriosis. It is therefore reasonable for insurers to develop prior authorization criteria for elagolix to ensure prudent use.
  • Patient organizations should band together to seek commitments from government research funding agencies and manufacturers to increase research, both basic and clinical, for common conditions affecting women’s health such as endometriosis.
  • Manufacturers should engage with key stakeholders in a transparent process to evaluate fair pricing of new therapeutics based upon the added clinical benefit to patients.
  • Manufacturer-sponsored research should enroll patients who reflect the population of patients commonly encountered in clinical practice and who are most likely to benefit from treatment.
  • Manufacturers and researchers in endometriosis owe patients, clinicians, and insurers better information on the long-term comparative clinical effectiveness and value of innovative new therapies. They should take action to ensure that future studies directly compare elagolix with other treatment options using standardized research protocols that focus on outcomes that reflect what matters most to patients.

ICER’s full set of policy implications, along with more detailed explanations of each, are available in the full report.

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.