Institute for Clinical and Economic Review Issues Final Report on Long-Term Prophylaxis for Hereditary Angioedema, Provides Policy Recommendations to Improve Cost-Effectiveness

— Cost-effectiveness and coverage considerations hinge on the frequency and intensity of attacks —

BOSTON, November 15, 2018 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report and Report-at-a-Glance assessing the comparative clinical effectiveness and value of therapies for long-term prophylaxis against hereditary angioedema (HAE) attacks. HAE is an ultra-rare condition characterized by attacks of deep tissue swelling within the skin and/or mucosa. In its report, ICER assessed three therapies for the prevention of HAE attacks: lanadelumab (Takhzyro™, Shire Plc), and two C1 inhibitors (Haegarda®, CSL Behring GmbH; and Cinryze®, Shire Plc).

ICER’s report was reviewed at an October 2018 public meeting of the California Technology Assessment Forum (CTAF), one of ICER’s three independent evidence appraisal committees. During the meeting, CTAF found that the evidence demonstrated a net health benefit for using the C1 inhibitors as long-term prophylaxis, but that the evidence was insufficient to distinguish between Cinryze and Haegarda. Because of concerns about risks with a new therapy, the committee also found that current evidence was not adequate to determine whether long-term prophylaxis with lanadelumab is superior to on-demand therapy alone.

Decision makers often give special considerations to therapies for ultra-rare diseases such as HAE, which may lead to coverage and funding decisions at higher thresholds for cost-effectiveness. During their deliberation, CTAF members underscored how HAE represents a particularly high lifetime burden of illness, and that all three prophylactic treatments may significantly improve both patients’ and caregivers’ ability to return to work or school. The panel recognized that Haegarda and lanadelumab offer simpler administration – subcutaneous injection – which may achieve better patient outcomes over a treatment like Cinryze, which is administered intravenously.

CTAF voted that both Cinryze and lanadelumab represent a low long-term value for money when compared to on-demand therapy. When evaluating the long-term value for money of Haegarda, the panel’s majority vote was split evenly between low and intermediate value. All three prophylactic treatments, at current pricing, exceed commonly cited thresholds for cost-effectiveness.

“Treatment for HAE attacks is very expensive, as are the prophylactic therapies used to prevent such attacks, and so the overall cost of prophylaxis includes the savings incurred from less on-demand treatment,” said David Rind, MD, ICER’s Chief Medical Officer. “The economic models produced for this report were very sensitive to small changes in assumptions about the frequency of attacks, the amount of on-demand treatment required, and the exact dosing regimens of prophylactic therapy. Similarly, relatively small changes in the price of prophylactic therapy greatly improved its cost-effectiveness. Insurers and manufacturers should work together to achieve appropriate access to these important therapies at a price that patients and society can afford.”

Key Policy Recommendations

Following the voting session, a policy roundtable of experts – including a patient advocate, physician, drugmaker, and payer representative – convened to discuss the implications of the evidence for policy and practice. Key recommendations stemming from the roundtable discussion include:

  • Payers seeking to negotiate better prices may consider giving all market share to the two injectable treatments, Haegarda and lanadelumab, due to these therapies’ simpler administration compared to intravenous drugs.
  • Prior authorization criteria should be based on clinical evidence with input from clinical experts and patient groups. Insurers crafting coverage policy may seek to confirm HAE through lab tests or physician attestation, determine the appropriateness of long-term prophylaxis based on the frequency and severity of attacks, and use a patient’s weight to more precisely manage dosing of weight-based treatments. Specific options are described in greater detail within the full report.
  • There are currently no consensus criteria on when to consider starting long-term prophylaxis for patients with HAE. Specialists involved in the care of patients with HAE should convene and work with patients to develop a consensus statement to guide policymakers and payers on the appropriate use of long-term prophylaxis for patients with Type 1 or 2 HAE.

ICER’s detailed set of policy recommendations is available in the Final Evidence 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.