As stated before, Patients Rising is a non-profit with a very specific mission: We fight for access to vital therapies and services for all patients with life-threatening and chronic diseases. We believe that if a patient needs medicines to survive and live a better quality and more productive life – access to those treatments is warranted and essential. This is part of our desire for a balanced dialogue and national conversation that tells the truth about healthcare.

In this work, we are committed to engaging patients, caregivers, physicians, the media, health policy experts, payers, providers and other allied health professionals to develop realistic, solution-oriented discussions around these issues so that those impacted with critical medical issues can amplify our collective voice and create lasting impact on the future of health care in the United States.

It is for this reason, and others, that we write today and offer our review and observations of ICER’s value framework, what it represents and the manifest flaws we believe must be immediately addressed.

I. Use of the QALY

ICER’s statement that “The QALY was developed by health economists and doctors in the United States” is factually inaccurate.

The QALY was first developed not in the United States, but in Great Britain, specifically at the University of York’s Centre for Health Economics. Specifically, the QALY was developed and advocated for by an economist named Alan Williams of the University of York in the 1970s – to enable the rationing of health care in the UK’s National Health Service.

Last month, ICER defended its use of the QALY in describing value, defining it as “the gold standard” and intimating that it is used around the world. Yet, at an advocacy briefing held in Washington, D.C. on September 9th, ICER’s COO Sarah Emond, remarked that ICER is open to a reworking of the QALY (or the adoption of a different methodology) in 2017. Whatever methodology is used to describe or define “value” should be made completely transparent and published in a peer-reviewed journal meeting high scientific standards.

Patients Rising supports a systematic evaluation of the QALY and its appropriateness as a measure in determining value for patients. Furthermore, we recommend that ICER not use misleading terminology such as “gold standard” when referring to the QALY, given its documented flaws and biases as well as the ongoing debate within health economics and policy circles regarding its appropriate use. Patients Rising recommends an evidence-based, patient-centered approach to defining value that is based in science and eschews the possibility of ideological bias.

II. Shortfall of relevant, “in-field” medical experts as part of your voting panel

While there are always a finite number of members for any official voting panel, we do not believe past ICER practice has demonstrated best practices among organizations that make clinical recommendations, nor that ICER’s practices are in the best interest of the patient.

In looking ahead to the October 20 CEPAC meeting on Non-Small Cell Lung Cancer, the voting panel selected by ICER does not include a single expert clinician or clinical researcher treating Non-Small Cell Lung Cancer patients, despite the fact that many of the world’s leading oncologists in this subspecialty are working in the United States, including in St. Louis, Chicago, and other major Midwestern metropolitan areas. The lack of inclusion of such experts is a major flaw in the implementation of your methodology. Patient groups are alarmed at the possibility that individuals who are not board certified in oncology could vote on an issue that would impact the availability of cancer drugs to NSCLC patients.

ICER must redouble its commitment to make certain its organization is asking the most appropriate questions, extracting and examining the best and most up-to-date data and, ultimately, delivering an accurate assessment both clinically and economically that takes into account the severity and gravity of the diseases it evaluates. Unfortunately, ICER’s current practices do not seem fit to credibly serve this purpose.

III. Lack of meaningful patient involvement

At the New England CEPAC meeting in July, many patient advocates were curious as to ICER’s selection of a patient representative who would not actually be affected by the vote taken. Patients Rising believes that in policy discussions, the experts and stakeholders selected to participate should have specific relevance to the matter at hand. We recommend that ICER include on future panels patients who are taking or have taken the therapies being evaluated, or are current candidates for these therapies. We would be happy to provide individuals whose individual situations, whether taking a specialty medication or who have a mutation that necessitates a personalized medicine approach, would be appropriate members of the panels.

Patients may not understand the complexities and specifics of cost-per-QALY models or ICER’s budget impact calculations, but they understand when a group of people go into a room and vote on their ability to access their medicines. You should make the input of patients a top priority, not an afterthought.

IV. Exclusion of estimation of rebates

According to Express Scripts, Dr. Pearson is one of the key thought leaders contributing to their Oncology Care Value Program. We believe that ICER should convene a fair and balanced forum with your pharmacy stakeholders –specifically the two largest in the United States (Express Scripts and CVS Caremark) – to develop a formula for the estimation of rebates as part of ICER’s economic methodology.  ICER’s methodology contains dozens of estimates and assumptions to facilitate analysis and synthesis of complex information.

If ICER is unwilling to take a serious look at the flawed and inflated payment structure or the absurd length of time to develop new treatments, how can you expect to represent an honest cost dialogue?

V. Conclusion

The only value framework that will ever be the right value framework is one where patients and their doctors can input information based on the whole picture of the treatment journey. This is because treating the whole patient is the most cost-effective means of treating the larger patient population. While there is much to be addressed in streamlining our finite health care resources, sacrificing patient care to meet some arbitrary metric fails every professional and moral standard.

For ICER, a new methodology is absolutely necessary.


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