Sickle Cell Disease (SCD): Toward a Global Solution
SCD affects millions worldwide, with a particularly high prevalence in sub-Saharan Africa and Southeast Asia. In the U.S., African American communities are disproportionately affected, and despite healthcare advances, life expectancy for SCD patients remains significantly lower than average. Prime HCD’s study explored the potential impact of a gene therapy (GTx) for SCD, projecting major improvements in health outcomes and quality-adjusted life expectancy across income-diverse countries. With value-based price (VBP) estimates ranging from $3.6 million in the U.S. to $700 in Uganda, the study emphasized the urgent need for differential pricing and global collaboration to ensure accessibility for those most impacted.
HIV Curative Therapies (CTx): Envisioning a World Beyond antiretroviral therapy (ART)
ART transformed HIV into a manageable condition over the past three decades, and now new research into curative therapies (CTx) has accelerated. Prime HCD’s long-term modelling of CTx potential showed that curative treatments could dramatically reduce disability-adjusted life years (DALYs) and HIV cases, particularly in low- and middle-income countries. The value-based prices for a hypothetical CTx varied by country, ranging from $10,500 in South Africa to $8.7 million in the United States, highlighting the importance of country-specific economic models. This research underscores the need for innovative payment and access strategies to make CTx accessible and sustainable.
Gene Therapy for Hemophilia: A Roadmap for Decision-Making
Gene therapy (GTx) offers a transformative opportunity for hemophilia, a rare bleeding disorder affecting patients globally. Prime HCD led a quantitative survey across UK treatment centres, creating a roadmap for GTx consideration. Designed with patient centricity in mind, this roadmap includes key milestones, consent gateways, and support periods to facilitate informed decision-making for patients and their families. This framework not only fosters engagement but also supports healthcare providers in optimizing the pathway to GTx adoption and monitoring.
Systemic Lupus Erythematosus (SLE): Voices from Diverse Communities
SLE has high prevalence amongst patients particularly from black, African American and Asian backgrounds. Prime HCD’s focus was to capture patient reported outcomes (PROs) to measure the impact of living with SLE via a cross-sectional study in South Africa and the UK. Working with local country researchers, patient advocacy groups and in local dialects, the study successfully recruited over the target sample. Importantly, the study highlighted the continued toll of fatigue, joint pain, impact on family activities and work productivity persons from diverse ethnicities face associated with having SLE. With fatigue identified as the most burdensome symptom, the study underscores the pressing need for improved quality of life and highlights the value of inclusive research that considers patients’ ethnicity, socioeconomic and cultural contexts.
The Ageing Challenge in Rare Diseases: A New Focus on Geriatrics
Aging with a rare disease, once an improbable scenario, is now a reality for many due to earlier diagnoses and better care. However, elderly patients with rare diseases, such as hemophilia, face heightened risks of comorbidities, including cardiovascular and bone health issues, along with increased anxiety and depression. Research from the Prime HCD CHESS database highlighted the complex care needs of elderly hemophilia patients, emphasizing the importance of a multidisciplinary approach to manage their evolving health challenges. The study reinforces the need for specialized geriatric expertise to support quality of life in the aging rare disease population.
Looking Forward to 2025
The team at Prime HCD and all our colleagues across Prime are committed to driving innovation in health research, insights, understanding and communication. We will continue to promote inclusive research and advocate for collaborative strategies. We look forward to another exciting year in research, helping to transform health outcomes globally.