Hemophilia B – Unmet Need – Unmet Need – Severe Noninhibitor Hemophilia B (US/EU)
Hemophilia B is a genetic disorder caused by a deficiency in clotting factor IX (FIX). Individuals with this condition experience prolonged bleeding. Prescribing options include factor balancing therapies, including plasma-derived therapies, recombinant FIX (rFIX) therapies, and a gene therapy (Hemgenix by CSL Behring) for patients without inhibitors. The main goal of treatment is to improve patients’ quality of life by reducing the frequency and severity of bleeding episodes. While existing treatments can manage bleeding episodes, approved treatments that are curative or can fully restore the body’s clotting mechanisms are lacking. In this report, U.S. and European hematologists’ reveal which attributes drive their prescribing and the efficacy, safety, and delivery they expect from future treatments for severe noninhibitor hemophilia B. Using the Target Product Profile (TPP) simulator, we can establish physicians’ preference for emerging drugs and asses the remaining commercial opportunities in this area.
Questions answered
- How well do current therapies perform on key treatment drivers and goals for the severe hemophilia B noninhibitor patient population?
- What drug attributes are key influencers, which have limited impact, and which are hidden opportunities in treating severe noninhibitor hemophilia B patients?
- What trade-offs across different clinical attributes and price are acceptable to U.S. and European physicians for new hemophilia B drugs to be preferred over current treatment options?
- Where do surveyed hematologists perceive the largest gaps in the treatment of severe noninhibitor hemophilia B patients?
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 Clarivate experts, and additional customized market scenarios can be evaluated with the corresponding TPP Simulator.
Key feature
The Target Product Profile (TPP) Simulator tool allows for customizable market simulations based on conjoint analysis that depicts how physicians make decisions based on actual behavior rather than opinion. Compare up to seven TPPs across multiple disease-specific attributes and price points to gauge which variables influence prescribing behavior.
Markets covered: United States, France, Germany, United Kingdom
Primary research: 60 U.S. and 34 European hematologists surveyed in June 2024
Key companies: Takeda, Sanofi, CSL Behring, Pfizer, Novo Nordisk
Key drugs: Idelvion, Alprolix, Rixubis, Rebinyn / Refixia, Benefix, Hemgenix, plasma-derived FIX
