Sickle Cell Disease – Landscape & Forecast – Disease Landscape & Forecast
Sickle cell disease (SCD) is a rare genetic blood disorder characterized by polymerization of hemoglobin (Hb) in red blood cells that distorts them into a sickle shape. This sickling leads to several complications, such as acute chest syndrome, anemia, and painful vaso-occlusive crisis (VOC). Despite the launch of novel therapies, hydroxyurea is the mainstay first-line treatment for SCD, in combination with prophylactic penicillin, analgesics, and blood transfusions. The FDA’s approval of Emmaus Life Sciences’ Endari (L-glutamine), Pfizer’s (formerly Global Blood Therapeutics’ [GBT’s]) Oxbryta (voxelotor), and Novartis’s Adakveo (crizanlizumab) gives patients additional treatment options. Allogenic hematopoietic stem cell transplantation is the only available curative therapy, although gene therapies such as Bluebird Bio’s lovo-cel and CRISPR / Vertex’s exa-cel have the potential to cure severe SCD. Therapies that can reduce or eliminate VOCs in the most severely ill HbSS and HbSβ0 patients and extend life expectancy are strongly needed. Recognizing the commercial opportunity, drug developers are focused on developing agents that target VOC pain and/or the underlying genetic defect.
Questions answered
- How large is the diagnosed prevalent SCD population in the United States and EU5? How will the population change over the forecast period?
- What is the current treatment landscape, and how will it change in the next 10 years? How will the launches of recently approved therapies (voxelotor, crizanlizumab, lovo-cel, and exa-cel) and late-phase therapies (Novo Nordisk’s [formerly Forma Therapeutics’] etavopivat, Pfizer’s [formerly GBT’s] inclacumab, Agios’s Pyrukynd [mitapivat], and Pfizer’s PF-07940367) impact SCD treatment?
- What sales / uptake could emerging therapies secure in the HbSS and HbSβ0 patient groups? How will new therapies be incorporated into medical practice?
Geographies: United States and EU5.
Primary research: Six country-specific interviews with thought-leading hematologists. Supported by survey data collected for this and other Clarivate research.
Epidemiology: Diagnosed prevalent and drug-treatable cases of sickle cell disease by country. Diagnosed prevalent cases of sickle cell disease segmented by clinical subtype.
Forecast: Drug-level sales and patient share of key sickle cell disease therapies in 2032.
Emerging therapies: Phase 3 / PR / approved: 9 drugs; Phase 2 and Phase 1/2: > 10 drugs.
Product description
Niche & Rare Disease Landscape & Forecast provides comprehensive market intelligence with world-class epidemiology, keen insight into current treatment paradigms, in-depth pipeline assessments, and drug forecasts supported by detailed primary and secondary research.
Solution enhancement
Niche & Rare Disease Landscape & Forecast introduces a Drug Pipeline chapter with real-time, global pipeline intelligence content directly from Cortellis. This chapter is updated daily and features interactive figures that can be easily downloaded for detailed analysis or presentations.
Table of contents
- Sickle Cell Disease - Landscape & Forecast - Disease Landscape & Forecast
- Key Findings
- Commercial Outlook
- Forecast
- Etiology and Pathophysiology
- Epidemiology
- Current Treatment
- Unmet Need Overview
- Drug Pipeline
- Emerging Therapies
- Key findings
- Key emerging therapies
- Notable developments among key emerging therapies for sickle cell disease
- Key therapies in development for sickle cell disease
- Estimated launch dates of key emerging therapies for the treatment of sickle cell disease
- Selectin inhibitors
- Pyruvate kinase-R activators
- Cell-based gene therapies
- HbS Polymerization inhibitors
- Early-phase pipeline analysis
- Patient registries
- Orphan drug designation
- Key findings
- Access & Reimbursement Overview
- Methodology
- Bottom-up forecasting overview
- Bottom-up forecast assumptions
- General sources of data
- Drug-treatable rate in sickle cell disease
- Drug-treatable rate assumptions in sickle cell disease
- Patient-share assumptions in sickle cell disease
- General statements about pricing
- Sources for drug prices
- 2022 exchange rates
- Price per treated day: 2032
- Dosing, days of therapy, and compliance
- Out-year forecasting
- Emerging therapy prices
- Primary market research
- Appendix
Shreaa, M.B.A., is an associate healthcare and data analyst on the Infectious, Niche, and Rare Diseases team at Clarivate. She conducts secondary and primary market research on U.S. and European biopharmaceutical markets. She holds an M.B.A. in pharmaceutical management from Jamia Hamdard in New Delhi.
Latashree Goswami, M.P.H., is an associate epidemiologist at Clarivate. Her areas of interest include oncology and autoimmune disorders. She holds an M.P.H. from the Indian Institute of Public Health, where she specialized in health economics, financing, and policy, and a B.D.S. from India’s ITS Dental College.