Boston, Mass., November 24, 2015 – The Institute for Clinical and Economic Review (ICER) has posted the final report PCSK9 Inhibitors for Treatment of High Cholesterol: Effectiveness, Value, and Value-Based Price Benchmarks, and its accompanying Action Guides to the New England Comparative Effectiveness Public Advisory Council website. This final report is an update to ICER’s earlier draft report on the two PCSK9 inhibitors approved by the FDA earlier this year: Alirocumab (Praluent®, Regeneron/Sanofi) and evolocumab (Repatha™, Amgen).  The final report incorporates the results of the deliberation and voting on the strength of evidence by the New England Comparative Effectiveness Public Advisory Council (New England CEPAC).  In addition, the final report includes an updated value-based price benchmark for PCKS9 inhibitor drugs and policy recommendations for coverage policy and for clinical practice generated by discussion with clinical experts, payers, patients, and other stakeholders at the public meeting of the New England CEPAC.  To accompany the final report ICER has released an Action Guide for policymakers and clinicians, as well as a separate guide for patients. The Action Guides further distill key policy recommendations and provide resources to support their implementation.

During consideration of the ICER draft report, the New England CEPAC heard public comment and engaged in discussion with clinical experts.  The first vote of the New England CEPAC unanimously confirmed that the evidence was not adequate to distinguish between the effectiveness of the two PCSK9 inhibitors.  A majority of the New England CEPAC voted that the evidence was adequate to demonstrate that PCSK9 inhibitors provide a net health benefit compared to other treatment options for patients with Familial Hypercholesterolemia (FH).  However, for patients with documented cardiovascular disease (e.g. strokes or heart attacks), the majority found the evidence inadequate, whether for patients who have high cholesterol because they cannot tolerate statins, or who do take statins but cannot achieve a low enough cholesterol to reach their clinical goal.  The driving consideration in the voting was the remaining uncertainty over whether lower cholesterol levels from PCSK9 inhibitor treatment will translate into lower rates of future heart attacks or strokes.  For patients with FH, however, a majority of the New England CEPAC felt that the additional, exceptional risks of this smaller patient population create an unmet medical need that outweighs the uncertainty in the evidence base.

The list price of the PCSK9 inhibitors is over $14,000 per year, and the final ICER report includes updated value-based price benchmarks of $5,404-$7,735 linked to long-term value to patients, and as low as $2,177 when potential short-term budget impact is considered.  Given the cost of PCKS9 inhibitors, the predominant view of the New England CEPAC was that these drugs at list price represent a “low” long-term value for patients.  With an even greater majority view, the provisional health system value was judged as “low” given the need to consider the potentially substantial budget impact that would occur if these new agents are used for even a small percentage of eligible patients.

Among the key recommendations for coverage policy and clinical practice arising from the discussion, votes, and subsequent policy debate were the following:

  • Professional societies should take prompt action to update clinical guidance, including a likely need to return to treatment goals based on target LDL-C levels.
  • Payers should consider using prior authorization to enhance health system value by limiting treatment to patients for whom extended trials of high-dose statins combined with ezetimibe have been unsuccessful.
  • Prior authorization criteria may need to require most patients who believe they are statin intolerant to be re-tried on statins.
  • If the pricing for PCSK9 inhibitors were to fall 50%-85% to a level that aligns with the benefit to patients and with a reasonable short term affordability, some payers would likely consider lifting many elements of proposed prior authorization requirements.

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.