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Newly Competitive Gaucher’s Disease Marketplace: Showcases Strategies for Rare Diseases
Decision Resources, Inc., June 2010, Pages: 39
Having enjoyed a protracted period of market monopoly, Genzyme - which pioneered enzyme replacement therapy for Gaucher’s disease - now faces the first real competition to Cerezyme in the form of two novel ERTs. Genzyme’s strategy in the Gaucher’s disease marketplace has been comprehensive and successful; the company can be said to be a model for other companies attempting to carve a treatment paradigm in a rare and underserved indication. Genzyme is a formidable established force in the Gaucher’s disease market, but the new entrants appear to have found viable means to compete. The Gaucher’s disease marketplace overall is thus a model of opportunities, challenges, and strategies relevant to operating in the realm of rare diseases.
Questions answered in this report:
- Gaucher’s disease arises from specific genetic mutations that correspond to distinct phenotypes of vastly different severity. What are these patterns? How do the subtypes of Gaucher’s disease manifest? How is the disease diagnosed, and what trends in diagnosis do interviewed physicians reveal?
- Gaucher’s disease patients are tracked by various registries. What does our analysis of the reported numbers reveal about overall patient population size in the major pharmaceutical markets in 2009? In 2014? In 2019? Gaucher’s disease is particularly common in people of Ashkenazi Jewish descent; how common is the disease in Israel?
- Gaucher’s disease is one of the few lysosomal storage disorders (LSDs) that is treatable, and one of only two LSDs for which more than one treatment is available. What are the current treatments? What are the unmet needs? What do interviewed physicians reveal about the current state of the art in Gaucher’s disease therapy? How are emerging therapies positioned to compete with the gold-standard treatment, Cerezyme from Genzyme?
- Treatment for Gaucher’s disease can cost up to $350,000 per adult patient per year. How is treatment currently reimbursed? What does the advent of competition mean for payer policies? How long will it take for these payer policies to evolve?
- Cerezyme’s success has sparked enhanced industry interest in research into orphan diseases such as Gaucher’s disease. At its peak in 2008, Cerezyme generated sales of more than $1.2 billion worldwide. What does a look at the dynamics of the Gaucher’s disease marketplace reveal about success factors and strategies for companies operating in this and other niche disease marketplaces? How is new market entrant Pfizer positioning itself?
Scope of the report:
- Markets covered: Major markets - United States, France, Germany, Italy, Spain, United Kingdom, Japan - and Israel.
- Primary research: Approximately a dozen interviews with thought-leading physicians and representatives of payer organizations.
- Epidemiology: Patient populations in the major markets--overall prevalence and prevalence by Gaucher’s disease subtype.
- Current and emerging therapies: Cerezyme, Zavesca, VPRIV, Uplyso, eliglustat tartrate.
- Strategic considerations: Commercial implications of differing manufacturing, pricing, and marketing strategies.
Key terms for this report:
Cerezyme, Enzyme replacement therapy (ERT), Gaucher’s disease, Genzyme, Hepatosplenomegaly, Lysosomal storage disorders (LSDs), Manufacturing, Metabolic disorder, Metabolic disease, Patient populations, Rare disease, Substrate replacement therapy (SRT), Uplyso, VPRIV, Zavesca
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