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