SYSTEMATIC LITERATURE REVIEW OF TREATMENT PREFERENCE ELICITATION STUDIES IN SICKLE CELL DISEASE
Author(s)
Emily Back, MSc1, Dick Nchang, BSc2, Brendan Mulhern, PhD3, Sarah Brighton, MSc1, Maisie Robinson, BSc4, Samuel Bristow, BSc, MSc1, George Morgan, MSc1.
1DHT.health, Clitheroe, United Kingdom, 2Sickle Cell International Foundation, Douala, Cameroon, 3Centre for Health Economics Research and Evaluation, Sydney, Australia, 4NHS Business Service Authority, Newcastle, United Kingdom.
1DHT.health, Clitheroe, United Kingdom, 2Sickle Cell International Foundation, Douala, Cameroon, 3Centre for Health Economics Research and Evaluation, Sydney, Australia, 4NHS Business Service Authority, Newcastle, United Kingdom.
OBJECTIVES: Sickle cell disease (SCD) is an inherited blood disorder associated with substantial clinical and treatment burden. As treatment options expand, including disease-modifying, emerging, and potentially curative therapies, understanding treatment preferences among patients, caregivers, and healthcare professionals is increasingly important. This systematic literature review aimed to identify and summarise published preference elicitation research evaluating treatment preferences in SCD.
METHODS: A systematic literature review (SLR) was conducted in MEDLINE® and the Cochrane Library to identify studies published from January 2010 to May 2026. Relevant conference proceedings were searched from January 2020 to May 2026. Eligible studies included preference elicitation research and mixed-methods studies incorporating preference elicitation or treatment-attribute development. Two reviewers independently double-screened studies against pre-defined eligibility criteria. Data extracted included study characteristics, perspective, methods, treatment, and preference outcomes.
RESULTS: Of 479 records screened, 6 articles and 2 conference abstracts met the inclusion criteria. Of the 8 studies identified, 6 were conducted in the United States. Studies, excluding one systematic review, were categorised as formal preference elicitation studies or preference-related surveys. Three formal elicitation studies were identified, including one discrete choice experiment. The remaining 4 were preference-related surveys, including 2 conference abstracts, which explored treatment priorities without formal elicitation. Study perspectives included patients (7 studies), caregivers (5 studies) and healthcare professionals (1 study). Common attributes included safety and side-effect profile (6 studies), treatment effectiveness (5 studies) and curative potential (4 studies) while disease severity, autonomy/control and knowledge/familiarity were rarely addressed. Overall, participants prioritised clinical benefit, although this was balanced by safety concerns.
CONCLUSIONS: Published treatment preference research in SCD remains sparse and methodologically heterogeneous. Despite an expanding treatment landscape, few studies have formally quantified treatment preferences, attribute importance, or benefit-risk trade-offs. Further robust preference elicitation research is needed to inform patient-centred treatment development, shared decision-making, and value assessment in SCD.
METHODS: A systematic literature review (SLR) was conducted in MEDLINE® and the Cochrane Library to identify studies published from January 2010 to May 2026. Relevant conference proceedings were searched from January 2020 to May 2026. Eligible studies included preference elicitation research and mixed-methods studies incorporating preference elicitation or treatment-attribute development. Two reviewers independently double-screened studies against pre-defined eligibility criteria. Data extracted included study characteristics, perspective, methods, treatment, and preference outcomes.
RESULTS: Of 479 records screened, 6 articles and 2 conference abstracts met the inclusion criteria. Of the 8 studies identified, 6 were conducted in the United States. Studies, excluding one systematic review, were categorised as formal preference elicitation studies or preference-related surveys. Three formal elicitation studies were identified, including one discrete choice experiment. The remaining 4 were preference-related surveys, including 2 conference abstracts, which explored treatment priorities without formal elicitation. Study perspectives included patients (7 studies), caregivers (5 studies) and healthcare professionals (1 study). Common attributes included safety and side-effect profile (6 studies), treatment effectiveness (5 studies) and curative potential (4 studies) while disease severity, autonomy/control and knowledge/familiarity were rarely addressed. Overall, participants prioritised clinical benefit, although this was balanced by safety concerns.
CONCLUSIONS: Published treatment preference research in SCD remains sparse and methodologically heterogeneous. Despite an expanding treatment landscape, few studies have formally quantified treatment preferences, attribute importance, or benefit-risk trade-offs. Further robust preference elicitation research is needed to inform patient-centred treatment development, shared decision-making, and value assessment in SCD.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR83
Topic
Health Policy & Regulatory, Patient-Centered Research, Study Approaches
Topic Subcategory
Patient Engagement
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)