ICER Draft Report on Effectiveness, Value, and Pricing Benchmarks for PCSK9 Inhibitors for High Cholesterol Posted for Public Comment

Boston, Mass., September 8, 2015 – The Institute for Clinical and Economic Review (ICER) has released a new draft report, titled PCSK9 Inhibitor Therapies for High Cholesterol: Effectiveness, Value, and Value-Based Price Benchmarks. The report includes a comprehensive review of currently available evidence on the newly approved PCSK9 inhibitors alirocumab (Praluent®, Regeneron/Sanofi) and evolocumab (Repatha™, Amgen), and also provides analyses of cost-effectiveness and potential budget impact under different utilization assumptions. Based on these findings the report presents the draft ICER value-based price benchmark for these drugs; prices that reflect the magnitude of estimated improvements in long-term patient outcomes and a threshold for new drug costs that does not exceed growth in the overall national economy.

It has been estimated that as many as 3.5-15 million Americans with elevated cholesterol may be eligible and considered for treatment with PCSK9 inhibitors. But there is remaining uncertainty about interpretation of the existing evidence on the clinical effectiveness of these drugs for different types of patients. In addition, with a list price over $14,000 per year there are serious questions regarding the price at which these drugs would represent a sensible value to patients and to the health care system.

“Patients, clinicians, insurers – everyone needs good information in order to make the best decisions about the use and pricing of new drugs,” Steven D. Pearson, MD, MSc, the Founder and President of ICER noted. “Our goal is to meet that need and in doing so not shy away from analyzing cost-effectiveness and potential budget impact.”

ICER’s draft report summarizes evidence demonstrating that PCSK9 inhibitors reduce LDL-cholesterol by approximately 55-60% among patients who are already on statins or who cannot take them. ICER concludes that this evidence provides moderate certainty that PCSK9 inhibitor therapy improves patient outcomes given remaining uncertainty about whether markedly lower LDL-cholesterol levels will translate into lower rates of heart attack and stroke. ICER’s report also concludes that the two PCSK9 drugs appear to have equivalent overall effectiveness for most patient groups.

The results of ICER’s analyses indicate that the price that best represents the overall benefits these drugs may bring to patients would be between $3,615 and $4,811, representing a 67% discount off the list price. But, as Pearson noted, this price range excludes a critical consideration: potential budget impact.

“Even if these drugs were used in just over 25% of eligible patients, then employers, insurers, and patients would need to spend on average more than $20 billion a year for these drugs, a cost that would continue on into the future.”

The report concludes that it would take a further price reduction to an annual drug cost of $2,177 for the total costs of these new drugs to come down to a level at which doctors and insurers would not have to try to limit patient use in some way to keep overall health care cost growth within bounds. Pearson concluded, “Our draft report therefore suggests that $2,177 is the price that should serve as an alarm bell –if the cost is more than $2,177 a year, drug companies, doctors, insurers, and other parties may need to work together to determine ways to limit the use of these drugs, find savings in other parts of the health care system, or adopt other measures to help make these drugs more affordable.”

The report, as well as accompanying draft voting questions, will be open to public comment until September 22nd at 5:00 PM EDT. Comments should be emailed to ICER will review all comments received and incorporate changes to the report as necessary. A revised draft report and voting questions will be posted on October 6th. Guidelines for submitting public comments, including formatting specifications, are posted on the CEPAC website.

The revised draft report will be the subject of deliberation and vote at the next meeting of the New England Comparative Effectiveness Public Advisory Council (CEPAC) on October 27, 2015 in Boston, MA. During the meeting on October 27th, CEPAC members will publicly deliberate on the evidence on comparative effectiveness and value as presented in the report and discuss the findings with a policy roundtable of experts to determine strategies to implement evidence-based recommendations that support effective coverage and use of PCSK9 inhibitors. The meeting will be free and open to the public. There will be a limited number of slots available for anyone wishing to make an oral comment on the report during the in-person meeting. Requests to make an oral comment should be submitted to by Tuesday, October 20th.

Meeting Details
Date: Tuesday, October 27, 2015
Time: 10:00 AM – 4:00 PM ET
Location: The Joseph B. Martin Conference Center, Harvard Medical School, 77 Avenue Louis Pasteur, Boston, MA, 02115
Click here to register to attend the meeting.

A webcast of the meeting will also be available. Click here to register for the webcast.

Additional explanatory material is available on the ICER website:
New Drug Assessment Project
Value Assessment Project
ICER Rating System Overview [including Evidence-Based Medicine (EBM) matrix]
Drug Assessment Report and Meeting Process Overview
Frequently Asked Questions regarding the drug assessment program


Mitchell Stein: 617-528-4013 ext. 7010