The Institute for Clinical and Economic Effectiveness’ draft cost-effectiveness report on the PCSK9 inhibitors has been updated in response to stakeholder comments and now suggests somewhat less of a price discount than originally thought necessary to allow the drugs to be considered cost-effective.

In its original draft report, released Sept. 8, ICER suggested discounts of at least 67% would be needed for Amgen Inc.’s Repatha and Sanofi and Regeneron Pharmaceuticals Inc.’s Praluent to be designated as cost-effective at the commonly used threshold of $150,000 per quality-adjusted life years (QALY) gained ([A#00150914013]).

A 67% discount would reduce annual wholesale acquisition cost (WAC) prices to about $4,811 from the current prices of $14,100 for Repatha (evolocumab) and $14,600 for Praluent (alirocumab).

Now, ICER indicates a 46% reduction in annual list pricing, to $7,735, could be cost-effective at the threshold of $150,000 per QALY gained. Achieving cost-effectiveness at a threshold of $100,000 per QALY would require price reductions of about 62% (to $5,404), the revised daft notes. The updated draft was released Oct. 8. A final version of the report will be developed after it is discussed at a public meeting of ICER’s Comparative Effectiveness Public Advisory Council on Oct. 27.

The change in projected discounts resulted from ICER’s decision to broaden its consideration of the benefit of PCSK9 treatment to span a patient’s lifetime and include outcomes in older patients.

Comments on the earlier version from Amgen, Sanofi, Regeneron and Pfizer Inc., which is developing the PCSK9 inhibitor bococizumab, had pointed out that “use of a 20-year time horizon rather than a lifetime horizon, as well as a focus only on patients age 35-74, ignores the benefits that would accrue to older patients, who experience a disproportionate number of [cardiovascular] events,” ICER explained.

“Despite our remaining concerns regarding the serious limitations of data on the effects of treatments in patients over age 75, we have revised the base-case economic model to extend the time horizon to lifetime, which captures the costs and benefits realized in patients as they age beyond 75.”

Stakeholders Commenting On PCSK9 Report

Amgen
Sanofi and Regeneron
Pfizer
Merck
Familial Hypercholesterolemia Foundation
AHIP
Blue Cross Blue Shield Association
Alliance for Patient Access
National Lipid Association
Advera Health Analytics

The revised cost-effectiveness calculations appear to be the most significant changes made to the report, although ICER received feedback in a number of other areas, including suggestions that the prevalence of familial hypercholesterolemia (FH) and its associated cardiovascular risks are greater than described in the report.

The PSCK9 drugs are indicated for patients with FH or clinical atherosclerotic cardiovascular disease who are taking maximally tolerated doses of statins but still require additional lowering of their LDL-cholesterol.

“The prevalence of FH in our modeling is consistent with the widely cited estimate of one in 500 for the US population, but we now acknowledge the variation in estimates in the executive summary” of the report, ICER said. The cardiovascular risk for FH patients used in the report “is approximately three-fold higher than the average population risk, which is consistent with that described in a study published after statin use became widespread,” the organization added.

Budget Impact Analysis Unchanged

Stakeholders also challenged the report’s novel analysis of the US “health system value” of the treatments. However, ICER has chosen not to revise that section of the report at this point.

The report concludes that a “value-based price benchmark” of $2,177 for each drug would help ensure that Repatha and Praluent do not cause overall US health care expenditures to rise faster than the economy, which is defined by ICER as exceeding the growth of the domestic gross product by 1%.

The $2,177 cost benchmark is based on estimates that 2.6 million patients would have access to the drugs after five years. ICER notes that comments criticized its assumptions about treatment uptake as “unreasonably optimistic, given lower rates of uptake observed with statins and other cardiovascular agents.”

ICER explained “these uptake levels reflect our estimate of utilization that is ‘unmanaged’ – without consideration of potential policies to limit utilization that might be instituted by provider groups or payers.”

The budget impact analysis is premised on the assumption that a drug costing more than $904m a year should prompt a consideration of different approaches to reducing costs. The $904m threshold was “criticized as an arbitrary ‘cap’ that is exclusively focused on drug cost and not on the net clinical benefit to patients,” ICER noted.

However, “this threshold is intended to serve as a policy trigger to stimulate efforts to address utilization, pricing and payment mechanisms to improve overall health system value,” the group explained.

Also criticized was the five-year timeframe used by ICER for the budget impact analysis. Some commenters felt five years was “too short to accurately reflect the long-term benefits of preventive therapies,” the organization said.

Nevertheless, ICER pointed out, “the five-year horizon we have adopted for short-term budget impact stretches as far as feasible to capture potential cost-offsets for preventive treatment while providing important perspective on the impact of net cost growth on health system affordability.”

Payers Applaud ICER Initiative

Payer organizations commenting on the draft report were generally supportive of ICER’s efforts. America’s Health Insurance Plans said the report “offers a common framework for payers, patients, providers and manufacturers.”

“Unsustainable costs and the introduction of new treatments and therapies without the support of strong evidence make value frameworks like that of ICER’s critically important to promoting affordability and value,” AHIP added. “ICER’s report on PCSK9 inhibitors sheds much needed light on the relationship, or lack thereof, between value and price.”

The Blue Cross Blue Shield Association agreed the report provides “valuable information to patients, payers, and providers regarding the effectiveness of the products relative to each other and existing medications. … This report, and the reports from ICER to follow, add an important contribution to the discussion of the sustainability of drug spending in the United States.”

PCSK9 Revised Analysis Indicates Less Price Discounting May Be Needed

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