Regardless of how innovative a new technology may be, all therapeutic approaches face increasing pressure on target product profiles. Determining what benchmarks novel therapies will need to demonstrate in order to prove differentiation and therapeutic value and compete in the changing rare disease landscape should be an early priority for developers. The transformation of the hemophilia market provides an important paradigm for the development of other rare disease treatment landscapes.
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Contributors: Aruni S. Arachchige Don, PhD, Senior Consultant; Katie Gersh, Associate Scientific Director; Akash Katakam, Research Analyst; Brent W. Osborne, PhD, Consultant; Michael C. Rice, MS, MBA, Principal
The transformation of the hemophilia market may prove an important archetype for the development of other rare disease treatment landscapes. Progress in the hemophilia landscape has certainly outpaced that of other rare diseases and is increasingly competitive, with on-demand and prophylactic treatments plus the curative potential of gene therapies. What has enabled this to happen? What needs and decisions have driven clinical trial design to enable new entrants to prove value? How has the payer and access market responded? How are physicians and patients making sense of new treatment options?