Hemophilia | Unmet Need | Hemophilia A (Severe Without Inhibitors) | US/EU | 2021
The primary goal of hemophilia A treatment is to prevent bleeding. Hemophilia A patients are a heterogeneous cohort; the tendency to bleed is highly individual. Currently available treatments including FVIII replacement therapies (e.g., Takeda’s Advate, Sanofi’s Eloctate) and Roche’s Hemlibra have helped significantly to reduce bleeding rates; nevertheless, breakthrough bleeds remain almost inevitable for severe hemophiliacs. Although full correction of coagulation is not necessary to stop bleeding, having a greater correction than is achievable using the current prophylactic treatments remains an unmet need, alongside an improved QOL and reduced dosing burden.
QUESTIONS ANSWERED
- What are the key treatment drivers and goals within severe hemophilia A? How do the current therapies perform on these goals?
- Which unmet needs are viewed by physicians as most important? Is further reduction in ABR the ultimate goal?
- Are the impact on QOL and dosing burden viewed as important attributes of emerging therapies?·
- Based on a conjoint analysis and TPP simulation, what trade-offs across different clinical attributes and prices are acceptable to U.S. and European hematologists for a hypothetical hemophilia drug?
PRODUCT DESCRIPTION
Unmet Need supports clinical development decisions by identifying key attributes and assessing areas of unmet need for a specific disease or subpopulation. Based on surveys with U.S. and European physicians, this report provides insight into key treatment drivers and goals, the performance of current therapies, and the remaining commercial opportunities. One market scenario is profiled in detail by DRG experts, and additional customized market scenarios can be evaluated with the corresponding TPP simulator.
Markets covered: United States, United Kingdom, France, Germany
Primary research: Survey of 60 U.S. and 30 European hematologists fielded in March 2021
Key companies: Bayer, Novo Nordisk, Roche, Sanofi, Takeda
Key drugs: Advate, Eloctate, Esperoct, Hemlibra, plasma-derived FVIII
