July 15, 2021

For years, the rare disease community has looked forward to a time when patients and physicians will be able to choose among multiple treatment options to provide the best care. For some rare conditions, that day has arrived. In the U.S., four therapies are now available to patients with Duchenne muscular dystrophy (DMD) and three each for spinal muscular atrophy (SMA) and transthyretin amyloidosis (ATTR).

Syneos Health has surveyed payers who have direct experience with DMD, SMA and ATTR to learn how emergence of competition will affect their decisions about utilization management and other issues. Their responses suggest that manufacturers must be prepared to negotiate on price, rebates and other matters.

In this whitepaper—the first in a two-part series—our experts review the treatment landscape for DMD, SMA and ATTR and examine how payer perceptions and practices are evolving.

Download the white paper.

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