The market for treating sickle cell disease is projected to expand significantly, increasing from US$ 1,160.16 million in 2022 to US$ 4,691.87 million by 2030, reflecting a compound annual growth rate (CAGR) of 19.1% during this period.
This growth in the sickle cell disease treatment sector is primarily driven by the rising incidence of the disease and various initiatives from both governmental and private organizations aimed at enhancing awareness about sickle cell disease. Additionally, the introduction of innovative diagnostic tools and an uptick in research efforts aimed at developing effective treatments are further propelling market expansion. Nevertheless, the limited availability of treatment options for sickle cell disease poses a challenge to market growth.
The management of sickle cell disease (SCD) can be categorized into two main approaches: health maintenance and complication management. Sickle cell anemia presents both genotypic and phenotypic variations, which arise from specific mutations in hemoglobin genes. The disease is notably prevalent and its severity is increasing globally. The World Health Organization (WHO) estimates that around 5% of the global population carries genes that predispose them to hemoglobin disorders, including sickle cell anemia and thalassemia. A study published in the National Library of Medicine indicates that over 300,000 infants are born annually with severe hemoglobin disorders, with projections suggesting that 400,000 newborns will be diagnosed with sickle cell disease by 2050. Furthermore, a report from the American Society of Hematology in 2023 estimates that between 70,000 and 100,000 individuals in the United States are affected by SCD. Consequently, the increasing rates of malaria and sickle cell disease in various regions worldwide are contributing to the growth of the treatment market for these conditions.
In recent years, focused pediatric programs and research initiatives have greatly enhanced patient care and increased life expectancy for those affected by SCD. Below are several organizations that have mobilized resources to improve treatment for sickle cell disease in the United States and globally.
• The American Society of Hematology Foundation aims to raise US$ 500,000 in private charitable contributions to develop targeted programs that focus on:
o Promoting awareness and encouraging proactive measures against SCD
o Training and educating hematologists and healthcare providers who care for SCD patients
o Funding SCD programs
o Enhancing care standards, research initiatives, and access to clinical trials for individuals with SCD
o Supporting the workforce of hematologists and healthcare professionals to effectively treat SCD patients throughout their life stages?from early diagnosis in childhood to adulthood.
• The Doris Duke Foundation provides grants to support innovative treatment methods for SCD, including gene therapy and drug treatments aimed at restoring hemoglobin function. The annual funding for these projects ranges from US$ 150,000 to US$ 300,000, plus 10% indirect costs for a duration of three years. This initiative seeks to advance clinical research that will:
o Promote advanced gene therapies, such as gene addition and genome editing for sickle cell disease in clinical settings
o Enhance globin regulatory mechanisms to restore red blood cell functionality
o Improve bone marrow transplant techniques to reduce mortality and enhance patient outcomes.
• In August 2021, the Ministry of Tribal Affairs, in partnership with the Indian Council of Medical Research (ICMR), allocated US$ 7.2 million to states for initiatives aimed at addressing sickle cell anemia, including screening programs. The Tribal Research Institute (TRI) under the Ministry of Tribal Affairs sanctioned a research project at Sir Ganga Ram Hospital focused on sickle cell disease. Additionally, a project titled "Diagnosis, IEC and Nutritional Support for Patients Affected by Sickle Cell Anemia and Thalassemia" was initiated.
• In February 2021, Novartis collaborated with the Bill & Melinda Gates Foundation to develop an accessible in vivo gene therapy for sickle cell disease. This partnership, funded with US$ 7.28 million, aims to create a treatment that is affordable and simple enough for use in low-resource settings with high SCD prevalence, particularly in sub-Saharan Africa, where approximately 80% of affected individuals reside. The goal is to develop a ready-to-use treatment that circumvents some of the complex and costly steps involved in current gene therapy methods.
• In March 2022, a team of international researchers received a US$ 3 million grant from the National Institutes of Health (NIH) to sequence the complete genetic code of children with sickle cell disease in Ghana. By analyzing the DNA of 500 Ghanaian children with SCD, the researchers aim to identify potential genetic modifiers that could enhance disease management and patient care. These children are part of the Sickle Cell Disease Genomics Network of Africa (SickleGenAfrica), a US$ 5.4 million NIH-funded international project led by Ofori-Acquah, which focuses on understanding the genetic factors influencing SCD progression in African populations.
Insights on Treatment
The sickle cell disease treatment market is categorized based on treatment types into generic drugs and originator drugs. The originator drugs segment held the largest market share in 2022. Sickle cell disease is a hereditary blood disorder characterized by abnormal hemoglobin, which impairs the ability of red blood cells to transport oxygen effectively.
Insights on Route of Administration
The treatment market is also segmented by route of administration into oral and parenteral & intravenous methods. The oral segment represented the largest market share in 2022; however, the parenteral segment is anticipated to exhibit the highest CAGR during the forecast period. Oral drug delivery is favored due to its high patient compliance, non-invasive nature, minimal sterility requirements, cost-effectiveness, flexibility in dosage form design, and ease of manufacturing. The advantages of simple administration and long-term cost efficiency are key factors driving the preference for oral medications.
Insights on Distribution Channels
The treatment market is segmented by distribution channels into direct tender, hospital pharmacies, retail pharmacies, online pharmacies, and others. The direct tender segment accounted for the largest market share in 2022; however, the online pharmacies segment is projected to experience the highest CAGR during the forecast period.
Key primary and secondary sources utilized in the preparation of the report on the treatment market include the World Health Organization (WHO), the US Census Bureau, and the Centers for Disease Control and Prevention (CDC), among others.