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 to advance a balanced dialogue that illuminates the truth about health care in a just and equitable manner.

We appreciate the opportunity to provide our comments on ICER’s July 20th Draft Evidence Report, “Inotersen and Patisiran for Hereditary Transthyretin Amyloidosis: Effectiveness and Value.” As the Draft Report articulates, amyloidosis is a complicated disease, and focusing on a hereditary sub-type of a rare condition both further specifies the condition and pathology, and reduces the patient population. Such rare diseases present patients and clinicians with clinical challenges. As the Draft Evidence Report describes, there are now at least two new compounds expected to be approved by the FDA for this patient population. Our specific patient-focused comments about this Draft Report encompass both the complexity of treatment as well as ICER’s approach and analytical methodologies. Our concerns are expressed in the following major sections: Patient Perspectives; Data Uncertainties and Assumptions; and Humanistic Perspectives on ICER’s Approach.

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