Global Prevalence: Sickle Cell Disease (SCD) is a widespread genetic blood order, affecting millions worldwide. The majority of cases are found in sub-Saharan Africa and Southeast Asia.

Impact in the US: In the United States, SCD predominantly affects individuals of African or African-American descent, with approximately one out of every 365 births in this population affected.

Life Expectancy: Despite advancements in care, individuals with SCD in developed countries have significantly lower life expectancy compared to the general population.

Raising Awareness: There is a crucial need to raise awareness about SCD among policymakers, healthcare professionals, and the general public. Advocacy efforts can promote research, improve access to care, and address the social and economic challenges faced by individuals with SCD.

Collaboration is Key: Collaboration between high-income countries (HICs) and low-and middle-income countries (LMICs) is vital to facilitate the delivery of effective treatments, including future gene therapies, to regions with the highest disease burden. Emerging gene therapies (GTx) could potentially lead to complete remission.

Prime HCD’s research explores the potential health impacts and value of a future GTx for SCD in HICs and LMICs.

Morgan, G., Back, E., Besser, M. et al. The value-based price of transformative gene therapy for sickle cell disease: a modeling analysis. Sci Rep 14, 2739 (2024).

We developed a Markov model to estimate the health benefits and costs of a hypothetical GTx for SCD from a multi-country perspective, compared to standard care, considering factors like remission probability, treatment durability, and uptake over time.

Our findings suggest that hypothetical GTx could reduce the number of people symptomatic with SCD over time, leading to fewer disability-adjusted life-years (DALYs). Across countries, value-based prices (VBPs) were primarily driven by willingness to pay per DALY averted and the rates of newborn screening. VBP ranged from $700 in Uganda to  $3.6 million in the US.

At Prime, we are driven by the urgent need for solutions to global healthcare challenges and are committed to fostering positive change. We hope the findings from our research:

  • Informs the development of payment models for treatment approval in the global market.
  • Highlights the need for strategies to ensure universal access to, and affordability of, GTx for SCD, such as differential pricing in each market or international collaborations to identify appropriate funding solutions.

For further information on our research and capabilities, please contact