DRG expects the next ten years to be transitional for patients with hemophilia A, with and without inhibitors, as both the number of therapy options and the hemophilia market size will increase. With the emergence of Roche / Chugai’s bispecific antibody, Hemlibra, and a diverse pipeline, both in terms of technology type and administration route, we forecast a significant change in approach to disease management for many patients. The R&D emphasis has shifted to nonfactor treatment approaches to restore coagulation. Examples include Sanofi’s anti-ATIII siRNA, fitusiran, Novo Nordisk’s anti-TFPI MAb, concizumab, and BioMarin’s gene therapy, BMN 270. Despite novel nonfactor hemophilia agents forecast to launch, we expect the demand for factor-based concentrates to persist.
- How will physicians treating hemophilia A differentiate between the various emerging therapies? Which therapies will gain the greatest market share in an increasingly stringent regulatory environment?
- What clinical roles will Roche / Chugai’s Hemlibra, Sanofi’s fitusiran, Novo Nordisk’s concizumab, and gene therapy play in the evolving hemophilia A treatment landscape?
- How will the management of patients with inhibitors change when new treatment options become available? How will Hemlibra’s head start on its novel competitors play out over the forecast period?
- How will the factor VIII concentrate market evolve? How will nonfactor treatment options impact the multitude of brands vying for patient share in this space?
- What do interviewed payers advise developers to do to drive market access of new hemophilia drugs? To what extent will the cost of FVIII gene therapy drive its rationing in terms of a patient’s capacity to derive benefit?
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- Hemophilia - Landscape & Forecast - Disease Landscape & Forecast
Author(s): Kerri Brown, M Pharm; Abey John, MPH
Kerri Brown, M.Pharm., is an analyst with Decision Resources Group, focused on finding stories in data and developing products to inform strategic decisions in the hemophilia and diabetes spaces. Kerri holds a master of pharmacy degree from King’s College London with 1st class honors. Prior to joining DRG, she was a national clinical educator at a UK diabetes start-up, and has extensive experience of diabetes medical devices. Kerri has worked at AstraZeneca, and as a pharmacist in a national psychiatric clinical trial center. In addition to her work at DRG, Kerri is a practicing pharmacist with extensive clinical experience of UK physician prescribing in diabetes.
Abey John is a medical graduate with a Master’s in Public Health and has been associated with DRG since September 2015. He works with a global team of epidemiologists in performing systematic reviews of assigned diseases and prepare forecast models for clients. He also is involved in producing analyses for pharmaceutical drug developers on the descriptive epidemiology of major drug indications in mature and developing markets and have an overall experience of three years working in different healthcare sectors across the country.
Prior to joining DRG, Abey had been working with Jhpiego (an affiliate of Johns Hopkins Medical University) in implementing Family Planning Health Programs in India with collaborating with the Government of India. He also has worked with a grassroots level NGO as a health team manager which worked for the benefit of the rural population living in the foothills of the Himalayas.