Ultra Market Research | United States Sickle Cell Disease Treatment Market
United States Sickle Cell Disease Treatment Market
Report ID : 872
Category : Pharmaceuticals,United-States(US)
No Of Pages : 184
Published on: November 2024
Status: Published
Format :
Key Question Answer
Global Market Outlook
In-depth analysis of global and regional trends
Analyze and identify the major players in the market, their market share, key developments, etc.
To understand the capability of the major players based on products offered, financials, and strategies.
Identify disrupting products, companies, and trends.
To identify opportunities in the market.
Analyze the regional penetration of players, products, and services in the market.
Comparison of major players financial performance.
Evaluate strategies adopted by major players.
Recommendations
United States Sickle Cell Disease Treatment Market
Introduction
The United States sickle cell disease market focuses on developing effective treatments and management solutions for sickle cell disease (SCD), a genetic disorder impacting hemoglobin production. Affecting approximately 100,000 individuals in the U.S., SCD leads to complications such as anemia, pain crises, and organ damage. Recent advancements, including gene therapies and innovative drugs, are driving the SCD treatment market USA. In 2023, the market was valued at approximately $1.2 billion and is poised for robust growth due to increased healthcare investments and R&D activities. The emergence of novel therapies and strategic collaborations among pharmaceutical companies highlight a transformative phase for the sickle cell anemia market US.
Segmentation
By Therapeutics
Disease-Modifying Drugs
Hydroxyurea
L-glutamine
Others
Gene Therapies
CRISPR-Cas9 Therapies
Lentiviral Vector Therapies
Curative Therapies
Hematopoietic Stem Cell Transplantation
Bone Marrow Transplants
By Diagnostics
Screening Tests
Newborn Screening
Prenatal Testing
Blood Tests
Imaging Techniques
Doppler Ultrasound
Magnetic Resonance Imaging (MRI)
Others
By End-Users
Hospitals
Specialty Care Centers
Emergency Departments
Clinics
Hematology Clinics
Genetic Counseling Centers
Research Institutes
Academic Research Labs
Clinical Trial Centers
By Distribution Channels
Hospital Pharmacies
Inpatient Pharmacies
Specialty Pharmacies
Retail Pharmacies
Chain Pharmacies
Standalone Pharmacies
Online Pharmacies
List of Market Players
Novartis AG (Switzerland)
Vertex Pharmaceuticals (United States)
CRISPR Therapeutics (Switzerland)
Global Blood Therapeutics (United States)
bluebird bio (United States)
Editas Medicine (United States)
Sangamo Therapeutics (United States)
Pfizer Inc. (United States)
Acceleron Pharma (United States)
Emmaus Life Sciences (United States)
Sanofi (France)
Bioverativ (United States)
Merck & Co. (United States)
Celgene Corporation (United States)
Apellis Pharmaceuticals (United States)
Drivers
The SCD therapeutics market USA is propelled by increasing awareness of sickle cell disease and advancements in genetic research. Government programs, such as newborn screening initiatives, enhance early detection and treatment efficacy. Breakthrough therapies like Oxbryta (Voxelotor) and gene-editing technologies are transforming disease management. Rising healthcare investments and collaborations between biopharma companies and research institutions accelerate innovation. Moreover, advocacy for health equity has heightened focus on improving access to quality care for underserved populations, bolstering the sickle cell healthcare market USA.
Restraints
The SCD drugs market growth USA faces barriers such as high treatment costs, limiting access for uninsured populations. Gene therapies, while promising, involve complex procedures and require specialized infrastructure, making them less accessible. Limited awareness in rural areas and disparities in healthcare delivery create adoption challenges. Regulatory hurdles, including lengthy approval processes for emerging therapies, slow market expansion. Additionally, the chronic nature of SCD demands lifelong management, straining resources for patients and caregivers.
Opportunity
Significant opportunities exist in the United States sickle cell disease market due to advances in precision medicine and gene-editing technologies like CRISPR. Expanding research into novel drugs and curative therapies opens avenues for innovation. Collaborations among biotech companies and academic institutions foster the development of affordable and effective solutions. The integration of digital health tools and remote monitoring enhances disease management, particularly in underserved regions. Increased funding and grants for SCD research by organizations such as NIH amplify growth prospects.
Trend
Key trends in the SCD treatment market USA include the rise of personalized medicine and precision therapies. Pharmaceutical companies are increasingly adopting value-based care models, focusing on cost-effectiveness and patient outcomes. AI-powered diagnostic tools and wearable devices for real-time symptom tracking are transforming care delivery. Furthermore, the emphasis on home-based treatments and patient-centric care aligns with the growing preference for convenience and accessibility. The approval of once-daily oral therapies signals a shift toward simplified treatment regimens.
FAQs about United States Sickle Cell Disease Market
Increased awareness, advanced therapies, and government screening programs.
Novartis, Vertex Pharmaceuticals, and Global Blood Therapeutics.
High costs, healthcare disparities, and complex therapy requirements.
Personalized medicine, AI-based diagnostics, and wearable health monitors
1. Introduction 1.1 Report Description 1.2 Research Methodology 1.2.1 Data Mining 1.2.2 Market Modeling and Forecasting 1.2.3 Data Validation 1.2.4 Industry Analysis 2. Market Dashboard 3. Market Overview 3.1 Market Definition and Scope 3.2 Market Segmentation 3.2.1 By Therapeutics 3.2.2 By Diagnostics 3.2.3 By End-Users 3.2.4 By Distribution Channels 3.3 Market Dynamics 3.3.1 Drivers 3.3.2 Restraints 3.3.3 Opportunities 3.3.4 Challenges 4. United States Sickle Cell Disease Treatment Market by Therapeutics 4.1 Introduction 4.2 Market Size and Growth Rate by Therapeutics (2024-2030) 4.2.1 Disease-Modifying Drugs 4.2.1.1 Hydroxyurea 4.2.1.2 L-glutamine 4.2.1.3 Others 4.2.2 Gene Therapies 4.2.2.1 CRISPR-Cas9 Therapies 4.2.2.2 Lentiviral Vector Therapies 4.2.3 Curative Therapies 4.2.3.1 Hematopoietic Stem Cell Transplantation 4.2.3.2 Bone Marrow Transplants 5. United States Sickle Cell Disease Treatment Market by Diagnostics 5.1 Introduction 5.2 Market Size and Growth Rate by Diagnostics (2024-2030) 5.2.1 Screening Tests 5.2.1.1 Newborn Screening 5.2.1.2 Prenatal Testing 5.2.1.3 Blood Tests 5.2.2 Imaging Techniques 5.2.2.1 Doppler Ultrasound 5.2.2.2 Magnetic Resonance Imaging (MRI) 5.2.2.3 Others 6. United States Sickle Cell Disease Treatment Market by End-Users 6.1 Introduction 6.2 Market Size and Growth Rate by End-Users (2024-2030) 6.2.1 Hospitals 6.2.1.1 Specialty Care Centers 6.2.1.2 Emergency Departments 6.2.2 Clinics 6.2.2.1 Hematology Clinics 6.2.2.2 Genetic Counseling Centers 6.2.3 Research Institutes 6.2.3.1 Academic Research Labs 6.2.3.2 Clinical Trial Centers 7. United States Sickle Cell Disease Treatment Market by Distribution Channels 7.1 Introduction 7.2 Market Size and Growth Rate by Distribution Channels (2024-2030) 7.2.1 Hospital Pharmacies 7.2.1.1 Inpatient Pharmacies 7.2.1.2 Specialty Pharmacies 7.2.2 Retail Pharmacies 7.2.2.1 Chain Pharmacies 7.2.2.2 Standalone Pharmacies 7.2.3 Online Pharmacies 8. Competitive Landscape 8.1 Company Profiles 8.1.1 Novartis AG (Switzerland) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.2 Vertex Pharmaceuticals (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.3 CRISPR Therapeutics (Switzerland) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.4 Global Blood Therapeutics (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.5 bluebird bio (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.6 Editas Medicine (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.7 Sangamo Therapeutics (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.8 Pfizer Inc. (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.9 Acceleron Pharma (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.10 Emmaus Life Sciences (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.11 Sanofi (France) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.12 Bioverativ (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.13 Merck & Co. (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.14 Celgene Corporation (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 8.1.15 Apellis Pharmaceuticals (United States) • Business Overview • Product Portfolio • Strategic Developments • Financial Overview 9. Conclusion and Recommendations 10. Appendix 10.1 List of Tables Table United States Sickle Cell Disease Treatment Market Size by Therapeutics (2024-2030) Table Disease-Modifying Drugs Market Size by Sub-type (2024-2030) Table Gene Therapies Market Size by Sub-type (2024-2030) Table Curative Therapies Market Size by Sub-type (2024-2030) Table United States Sickle Cell Disease Treatment Market Size by Diagnostics (2024-2030) Table Screening Tests Market Size by Sub-type (2024-2030) Table Imaging Techniques Market Size by Sub-type (2024-2030) Table United States Sickle Cell Disease Treatment Market Size by End-Users (2024-2030) Table Hospitals Market Size by Sub-type (2024-2030) Table Clinics Market Size by Sub-type (2024-2030) Table Research Institutes Market Size by Sub-type (2024-2030) Table United States Sickle Cell Disease Treatment Market Size by Distribution Channels (2024-2030) Table Hospital Pharmacies Market Size by Sub-type (2024-2030) Table Retail Pharmacies Market Size by Sub-type (2024-2030) 10.2 List of Figures Figure United States Sickle Cell Disease Treatment Market Size and Growth Rate by Therapeutics (2024-2030) Figure Disease-Modifying Drugs Market Growth Rate by Sub-type (2024-2030) Figure Gene Therapies Market Growth Rate by Sub-type (2024-2030) Figure Curative Therapies Market Growth Rate by Sub-type (2024-2030) Figure United States Sickle Cell Disease Treatment Market Size and Growth Rate by Diagnostics (2024-2030) Figure Screening Tests Market Growth Rate by Sub-type (2024-2030) Figure Imaging Techniques Market Growth Rate by Sub-type (2024-2030) Figure United States Sickle Cell Disease Treatment Market Size and Growth Rate by End-Users (2024-2030) Figure United States Sickle Cell Disease Treatment Market Size and Growth Rate by Distribution Channels (2024-2030) Tables Financial Overview of Key Companies Table Novartis AG Sickle Cell Disease Treatment Financial Overview Table Vertex Pharmaceuticals Sickle Cell Disease Treatment Financial Overview Table CRISPR Therapeutics Sickle Cell Disease Treatment Financial Overview Table Global Blood Therapeutics Sickle Cell Disease Treatment Financial Overview Table bluebird bio Sickle Cell Disease Treatment Financial Overview Table Editas Medicine Sickle Cell Disease Treatment Financial Overview Table Sangamo Therapeutics Sickle Cell Disease Treatment Financial Overview Table Pfizer Inc. Sickle Cell Disease Treatment Financial Overview Table Acceleron Pharma Sickle Cell Disease Treatment Financial Overview Table Emmaus Life Sciences Sickle Cell Disease Treatment Financial Overview Table Sanofi Sickle Cell Disease Treatment Financial Overview Table Bioverativ Sickle Cell Disease Treatment Financial Overview Table Merck & Co. Sickle Cell Disease Treatment Financial Overview Table Celgene Corporation Sickle Cell Disease Treatment Financial Overview Table Apellis Pharmaceuticals Sickle Cell Disease Treatment Financial Overview
Research Methodology
The process of market research at Ultra Market Research is an iterative in application and usually follows following path. Information from secondary used to build data models, then results from data models are validated from primary participants. Then cycle repeats where, according to inputs from primary participants, additional secondary research is done and new Information is again incorporated into data model. The process continues till desired level of Information is not generated
To calculate the market size, the report considers the revenue generated from the sales of Ultra Market Research providers. The revenue generated from the sales of Ultra Market Research has been calculated through primary and secondary research. The report also presents the key players operating in the Ultra Market Research market across the globe identified through secondary research and a corresponding detailed analysis of the top vendors in the market. The market size calculation also includes distribution channel segmentation determined using secondary sources and verified through primary sources.
Secondary Research
The secondary research source that are typically referred to include, but are not limited to:
Company websites, annual reports, financial reports, broker reports, investor presentations and SEC filings
Internal and external proprietary databases, relevant patent and regulatory databases
National government documents, statistical databases and market reports
News articles, press releases and web-casts specific to the companies operating in the market
The source for secondary research includes but is not limited to: Factiva, Hoovers and Statista
Inner Circle Represents – Stage of Research Process
Middle Circle Represents – Source of Information
Outer Circle Represents – Information Derived from that source
Primary Research We conduct primary interviews on an ongoing basis with industry participants and commentators in order to validate data and analysis. A typical research interview fulfills the following functions:
It provides first-hand Information on the market size, market trends, growth trends, competitive landscape, future outlook etc.
Helps in validating and strengthening the secondary research findings
Further develops the analysis team’s expertise and market understanding
Primary research involves E-mail interactions, telephonic interviews as well as face-to-face interviews for each market, category, segment and sub-segment across geographies
The participants who typically take part in such a process include, but are not limited to:
Industry participants: CEOs, VPs, marketing/ distribution channel managers, market intelligence managers and national sales managers
Purchasing managers, technical personnel, distributors and resellers
Outside experts: Investment bankers, valuation experts, research analysts specializing in specific markets
Key opinion leaders specializing in different areas corresponding to different industry distribution channels
Models Where no hard data is available, we use modeling and estimates in order to produce comprehensive data sets. A rigorous methodology is adopted in which the available hard data is cross referenced with the following data distribution channels to produce estimates:
Demographic data: Population split by segment
Macro-economic indicators: GDP, etc.
Industry indicators: Expenditure, distribution channel stage & infrastructure, sector growth and facilities.
Data is then cross checked by the expert panel.
2..1 Company Share Analysis Model
Company share analysis is used to derive the size of United States market. As well as study of revenues of companies for last three to five years also provide the base for forecasting the market size and its growth rate. This model is built in following steps: 2..2 Revenue Based Modeling
Revenue based models can be built in two ways – Top-Down or Bottom-Up irrespective of industry. Market size estimated from company share analysis acts as a validation point for bottom-up approach where as it acts as starting point for top-down approach.
2.1 Research Limitations Inflation is not a part of pricing in this report. Prices of United States Sickle Cell Disease Treatment Market and its derivatives vary in each region and hence similar revenue ratio does not follow for each individual region. The same price for each distribution channel has been taken into account while estimating and forecasting market revenue United Statesly. Regional average price has been considered while breaking down this market by application in each region. This report provides market size of United States Sickle Cell Disease Treatment Market for the past year and forecasts for the next six years. United States Sickle Cell Disease Treatment Market. Market size is given in terms of revenue. Market revenue is defined in USD Million. Market numbers are given on the basis of different United States Sickle Cell Disease Treatment Market categories. Market size and forecasts for each major application is provided in the context of United States market. The numbers provided in this report are derived on the basis of demand for United States Sickle Cell Disease Treatment Market from different application industries in different regions.
