Accelerating Equitable Access to Gene Therapies for Sickle Cell Patients

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Gene Therapy

By Pharm. Maryam Imam

Sickle Cell Disease (SCD) is a major public health challenge in Africa, with the continent accounting for approximately 75 per cent of the global burden of this hereditary blood disorder.In Nigeria alone, an estimated 150,000 children are born with SCD annually, making it the country with the highest prevalence worldwide. Despite the magnitude of SCD, access to effective treatments remains limited; while emerging therapies like gene editing also pose additional challenges due to their high costs.

Gene therapies, particularly those based on CRISPR technology, offer hope for a potential cure for SCD by targeting and correcting the genetic mutations responsible for the disease. These advancements have transformed treatment landscapes in high-income countries, yet their benefits remain largely out of reach for many African nations. The estimated cost of a single treatment course for gene therapies often exceeds $1 million, a price tag that is simply unaffordable for the majority of affected individuals diagnosed with SCD in Africa.

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In countries like Nigeria, where healthcare systems are already overburdened and underfunded, the prohibitive costs of gene therapies highlight the glaring disparities in access to healthcare.

Most SCD patients rely on symptomatic management, such as blood transfusions and hydroxyurea therapy, which are themselves often difficult to access consistently due to infrastructural and financial barriers. The economic toll of SCD on affected families is staggering.

Many households face significant financial strain due to recurring hospital visits, out-of-pocket payments for medications, and the inability to work caused by the frequent pain crises and complications of the disease. This strain is compounded by inadequate health insurance coverage and the lack of robust government support for SCD treatment programmes.

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To address these challenges, a multifaceted approach is needed. First, efforts should be intensified to negotiate with pharmaceutical companies for differential pricing models that consider the economic realities of low- and- middle-income countries. Partnerships between African governments, international health organisations, and private stakeholders are essential to subsidise the costs of gene therapies and make them accessible to individuals in need.

Additionally, research should prioritise culturally relevant, cost-effective alternatives, such as community-based interventions and local production of existing therapies like hydroxyurea. Expanding newborn screening programmes and improving early diagnosis of SCD can also reduce complications and lower long-term healthcare costs.

Furthermore, public awareness campaigns are significant to destigmatise SCD and advocate for policy changes that prioritise the disease in national health agendas. SCD research is underfunded. Policy makers must allocate resources to support SCD research, infrastructure development, and the training of healthcare professionals equipped to manage this complex condition.

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While gene therapies hold immense promise for transforming the lives of individuals with SCD, their current costs exacerbate health inequities, leaving many African families without hope for a cure. Bridging this gap requires a collective effort to prioritise equitable access to life-saving innovations, ensuring that advancements in medicine benefit all, regardless of geography or socioeconomic status.

By Pharm. Maryam Imam

PhD Candidate, Medical College of Wisconsin.

Wisconsin, USA

 

 

 

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