Amgen Response to ICER’s Updated Value Assessment Framework (as of May 5, 2017)

Recommendations for Value Assessment of PCSK9 Inhibitors

At Amgen, we are dedicated to patients and our mission is clear – to expand the boundaries of science and to transform medicine for patients who face serious illnesses. Because healthcare resources are not infinite, the U.S. needs rigorous and clinically relevant assessments of health technologies to help decision-makers get the greatest value for the dollars spent, by investing in interventions and innovations that add significant value over existing standards of care in the real-life setting of patient care.

Organizations that play a role in this process, like the Institute for Clinical and Economic Review (ICER), must offer information to inform decision-making by bringing practical, unbiased guidance to the healthcare system through a clinically relevant, pragmatic and methodologically rigorous approach to the evaluation of currently available evidence. Its latest report, titled “Updated Value Assessment Framework,” does not deliver against that standard. The revised framework is even more invested in a methodology that advocates for arbitrary and paternalistic willingness-to-pay thresholds while doing little to help the healthcare system understand the impact of new technology in a truly usable way.

For the upcoming update of ICER’s PCSK9 report, the relevance, integrity and usability of the results will be particularly important to stakeholders who are looking to apply the recently published results of the Repatha Cardiovascular Outcomes Trial (CVOT) to decision making. Specially, we urge ICER to apply methodologically sound and clinically useful techniques in the following three areas:

First, in cardiovascular disease, one of the main determinants of clinical value is the expected cardiovascular event rate in real people seeing real physicians. The ICER PCSK9 inhibitor report from 2015, by contrast, forecast largely trial-derived cardiovascular event rates that are substantially lower than event rates in actual clinical practice. Despite it being methodologically correct to calibrate health services models for differences between the trial and clinical practice environments, ICER based its value assessment on event rates that were generally 2-3 times lower compared to the rates from clinical practice. The recently concluded Repatha CVOT has confirmed Amgen’s assertion that real world event rates are, in fact, about 2-3 times more frequent than ICER’s initial underestimate. Rates of events derived from payer’s own data are even higher.

Second, and as noted in the recently-published results, it will be important that ICER appreciate that the Repatha CVOT results were consistent with the published, CTTC-modeled relationship between LDL and clinical outcomes benefits seen in nearly every other outcomes study of cholesterol-lowering agents powered to do so. This relationship has been used in 99% of the more than 100 economic analyses and models for cardiovascular disease, LDL lowering, and event reduction. The Repatha CVOT results also confirm a lag effect on outcomes whereby the outcomes reduction effect increases markedly after a year of treatment, consistent with the effects seen in past LDL outcomes studies. It will be critical for patients at high risk of CV events and eligible for Repatha that ICER appropriately project the long term benefits of LDL reduction using the CTTC-derived long term data for all outcomes, adjusted for the expected rate of events in the subject population.

Finally, after their initial bold claims that PCSK9 drugs would cost the health care system $20B a year and $100B after 5 years, it has become clear that these estimates were so far off as to be irresponsible, and have likely resulted in an environment that has denied access to many patients who may have otherwise been eligible for treatment. We urge ICER to apply clinically useful, realistic, and responsible estimation techniques to any uptake modeling they perform, and correct the previous “worst case” estimates.

We are hopeful that ICER will progress past the transient role of headline-centric price watchdog by maturing into a reliable and balanced scientific presence in our healthcare system that helps stakeholders understand the potential value of various treatment options in actual use. To that end, we have provided ICER with a number of recommendations based on worldwide pharmacoeconomic methodological guidelines and put forth in our response to ICER’s updated value assessment framework. We believe this will enable robust and productive dialogue across all stakeholders while building understanding of the complex science associated with longer-term health care valuation.