PATIENT AND CAREGIVER TREATMENT PREFERENCES IN DUCHENNE MUSCULAR DYSTROPHY: A TARGETED LITERATURE REVIEW
Author(s)
Anoushka Pathak, MSc1, Raju Gautam, PhD2, Rosalind Augustine, MSc1, Tushar Srivastava, MSc2, Radha Sharma, PhD3.
1ConnectHEOR, Delhi, India, 2ConnectHEOR, London, United Kingdom, 3Director, ConnectHEOR, Edmonton, AB, Canada.
1ConnectHEOR, Delhi, India, 2ConnectHEOR, London, United Kingdom, 3Director, ConnectHEOR, Edmonton, AB, Canada.
OBJECTIVES: Duchenne muscular dystrophy (DMD) is a progressive X-linked neuromuscular disorder causing ambulation loss and premature mortality. Treatment preferences in DMD have primarily been collected using best-worst scaling (BWS), and limited evidence exists on more advanced stated preference methods. We conducted a targeted literature review (TLR) focusing on conjoint analysis and discrete choice experiments (DCEs) of treatment preferences in DMD.
METHODS: A TLR was conducted in Embase® and supplemented with a desk search (up to June 2026), using disease, study design, and patient/caregiver preference terms. Studies reporting data on patient and/or caregiver preferences for DMD treatments using DCE or conjoint analysis were included.
RESULTS: In total, 74 records were screened, of which 3 studies (2 US, 1 global) were included. Two studies employed DCEs, and one used conjoint analysis and BWS. Across studies, attributes were developed via literature review, advisory board, and stakeholder collaboration. In a US-based study using DCE (9 patients, 87 caregivers, 65 healthcare professionals), participants favoured an improvement in muscle strength. The maximum acceptable increase in risk for muscle improvement was 26% for fracture risk, 9% for kidney damage, or 22% for drug-benefit uncertainty (all p<0.001). In another US-based conjoint analysis (119 caregivers), the conditional attribute importance was highest (26.9%) for effects on muscle function, followed by risk of arrhythmia (23.9%). In the global study (8 countries; 77 patients, 77 caregivers), participants preferred treatment that slowed progression for 3 years and had a drug failure rate of 25%; the preference models for patients and caregivers were relatively similar, with no differences in scale.
CONCLUSIONS: Treatment efficacy, particularly improvements in muscle function and slowing disease progression, are important to patients and caregivers. Future studies should employ advanced stated preference methods and increase the representation of patient perspectives, particularly as advances in treatment have increased survival into adulthood.
METHODS: A TLR was conducted in Embase® and supplemented with a desk search (up to June 2026), using disease, study design, and patient/caregiver preference terms. Studies reporting data on patient and/or caregiver preferences for DMD treatments using DCE or conjoint analysis were included.
RESULTS: In total, 74 records were screened, of which 3 studies (2 US, 1 global) were included. Two studies employed DCEs, and one used conjoint analysis and BWS. Across studies, attributes were developed via literature review, advisory board, and stakeholder collaboration. In a US-based study using DCE (9 patients, 87 caregivers, 65 healthcare professionals), participants favoured an improvement in muscle strength. The maximum acceptable increase in risk for muscle improvement was 26% for fracture risk, 9% for kidney damage, or 22% for drug-benefit uncertainty (all p<0.001). In another US-based conjoint analysis (119 caregivers), the conditional attribute importance was highest (26.9%) for effects on muscle function, followed by risk of arrhythmia (23.9%). In the global study (8 countries; 77 patients, 77 caregivers), participants preferred treatment that slowed progression for 3 years and had a drug failure rate of 25%; the preference models for patients and caregivers were relatively similar, with no differences in scale.
CONCLUSIONS: Treatment efficacy, particularly improvements in muscle function and slowing disease progression, are important to patients and caregivers. Future studies should employ advanced stated preference methods and increase the representation of patient perspectives, particularly as advances in treatment have increased survival into adulthood.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR33
Topic
Patient-Centered Research
Disease
Rare & Orphan Diseases