A Needs-Finding Framework for understanding gaps in Sickle Cell Disease Care Pathway

Background: Sickle Cell Disease (SCD) is a genetic blood disorder caused by a point mutation in the HBB gene that replaces glutamic acid with valine, altering the structure of hemoglobin. Unlike healthy, flexible red blood cells, these sickled cells are rigid and prone to clumping in small blood vessels, blocking blood flow and causing pain, tissue damage, and oxygen deprivation. As a result, individuals with SCD experience chronic anemia, fatigue, and delayed physical development. SCD diagnosis and treatment advancements have not been implemented equitably across all settings. Innovation priorities may be weakened when solutions are selected before population needs are defined and prioritized.Objective: The current studies in SCD available in public domain lack a systematic and structured approach to distill key reasons/themes why SCD diagnosis, treatment and management have not achieved substantial success when public health is concerned, even though there have been significant scientific advances in understanding the disease pathophysiologically. The main objective of this study is to identify and prioritize solution-neutral unmet needs in SCD diagnosis, management and treatment by adapting the Stanford Biodesign needs-finding framework to the systematic review of research publications on SCD.Study Design: The Biodesign framework consists of 3 phases: Identify, where needs finding and needs screening occurs; Invent, where the concept generation and filtering occurs; and Implement, where stages for strategy development and prototyping occur prior to the project launch. Needs Finding in Stanford Biodesign framework consists of three primary components: secondary background research, clinician and stakeholder discussions, and on-field observations. In this phase of research, we focused on the first part - secondary background research. The research involved identifying published literature on SCD, with a focus on gaps and unmet needs in SCD care in underserved settings. Deeper review with thematic analysis led to identification of key challenges and gaps in SCD care which formed the basis for writing structured need statements following the Stanford Biodesign template. Framework Results: The study produced 12 priority needs that fall under 4 major themes namely barriers to care, trust and emotional support, early diagnosis / screening and education and awareness. Increased screening needs received the highest total score (20.0) across the 4 criteria. Barriers to care and trust and emotional support received the second highest total score of 19.0. Education and awareness received a total score of 15.0. Conclusion: The Biodesign framework enabled a structured review to convert research evidence into prioritized SCD need statements that warrant solutions. The study identified screening needs, overcoming barriers to care, building trust and emotional support and education and awareness initiatives as the prioritized unmet needs in SCD patients.