Hemophilia A 2018

Patients Rising Now analyzes and advocates on behalf of patients for all patients with life-threatening and chronic diseases to have access to vital therapies and services. We believe that
if a patient needs treatments to survive and live a better and more productive life – access to
those treatments are warranted and essential – and access spans affordability, insurance coverage, and physical access.

In our 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 so that those impacted with critical medical needs can amplify their
collective voice and create a lasting impact on the future of health care in the United States. That
is, our goal is advancing a balanced dialogue and national conversation that illuminates the truth
about health care.

Our sister organization, Patients Rising, has previously written to you about our concerns with
ICER’s work and processes, and specifically, the lack of meaningful patient engagement,
inadequate attention to aspects of therapeutic choices that go beyond strict “clinical value”,
problems with evaluating Quality Adjusted Life Years (QALYs), and new information not being
included in updates to ICER’s analyses in a timely and meaningful fashion.
While all those areas are critically important to patients – and should be part of how patient
perspectives are considered by researchers, regulators, and payers – we are focusing our
comments about the draft evidence report “Emicizumab for Hemophilia A: Effectiveness and
Value” on the section titled “Potential Budget Impact.”

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