Spinal Muscular Atrophy 2019

Patients Rising Now advocates on behalf of patients with life-threatening conditions and chronic diseases for them to have access to vital therapies and services. Access is a matter of survival for those patients, and it spans affordability, insurance coverage, and physical access. To support improved access, we are committed to engaging patients, caregivers, physicians, media, health policy experts, payers, providers, and others to foster realistic, patient-centered, solution-oriented discussions for particular conditions and the entire U.S. health care system. That is, our goal is a balanced dialogue that illuminates the truth about health care in a just and equitable way.

We appreciate the opportunity to provide our comments on ICER’s December 20th Draft Report, “Spinraza® and Zolgensma® for Spinal Muscular Atrophy: Effectiveness and Value.”

Before commenting on specific aspects of the Spinal Muscular Atrophy (SMA) Draft Report, we want to revisit some past ICER reports and comments – and particularly responses to some of our previous comments, since they provide critical insights into ICER’s approach to health care challenges, and ICER’s perceived role in attempting to inform decisions about innovations.

Because imprecise language can lead to misleading conclusions, the specific issue we want to address is ICER’s decisions about choices regarding word usage and phrasing to describe its work. The danger of such rhetorical imprecision is well summarized in this quote: “[Language] becomes ugly and inaccurate because our thoughts are foolish, but the slovenliness of our language makes it easier for us to have foolish thoughts.”[i]

Specifically, in responding to our comments on the Opioid Use Disorder Draft Report, ICER noted that its use of the term “healthcare” rather than “health care” “does not affect the conclusions of our report.”[ii] While in that specific instance the meaning is likely the same, that is not always true. For example, the two phrases “mental healthcare” and “mental health care” have two distinct and different meanings. And we are very concerned that ICER apparently fails to recognize that such differences can lead to misinterpretation of data or results.

This concern is even more problematic in the Final Report about OUD treatments where ICER equivocates on the definition of MAT, declares that its assessment MAT can have two different meanings, and that ICER will use them interchangeably.[iii]  In that report ICER also misconstrues and misrepresents the meaning of the statement from the FDA: “Because OUD is a chronic illness, we should consider treating it much like we would any other chronic condition. We do not think of the medications used to treat diabetes or hypertension as ‘medication assisted treatment.’ We simply call it ‘treatment.’ OUD should be viewed similarly.” [iv] First ICER fails to understand that the FDA is not questioning the meaning of the definition of MAT – the heading for the article in fact is “Medication Assisted Treatment.”

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