CONTENT VALIDATION OF THE NTDT-PRO IN ALPHA-THALASSEMIA: A STANDALONE HYBRID CONCEPT ELICITATION AND COGNITIVE INTERVIEW STUDY
Author(s)
Ali Taher, MD, PhD, FRCP1, Nicole Clarke, MPH, PMP2, Laurie Eliason, MPH2, Ahmed Hnoosh, MSc3, Matthew Dyer, Health Economist4, Gale Harding, MA5, Shweta Bapat, BS, PhD5, Khaled M. Musallam, PhD, MD6.
1American University of Beirut Medical Center, Beirut, Lebanon, 2Bristol Myers Squibb, Lawrenceville, NJ, USA, 3Bristol Meyer Squibb, uxbridge, United Kingdom, 4Mereo BioPharma, London, United Kingdom, 5Thermo Fisher Scientific, Waltham, MA, USA, 6Burjeel Medical City, Abu Dhabi, United Arab Emirates.
1American University of Beirut Medical Center, Beirut, Lebanon, 2Bristol Myers Squibb, Lawrenceville, NJ, USA, 3Bristol Meyer Squibb, uxbridge, United Kingdom, 4Mereo BioPharma, London, United Kingdom, 5Thermo Fisher Scientific, Waltham, MA, USA, 6Burjeel Medical City, Abu Dhabi, United Arab Emirates.
OBJECTIVES: This study evaluated the content validity of the Non-Transfusion Dependent Thalassemia Patient-Reported Outcome (NTDT-PRO), a validated measure of anemia-related symptoms in β-thalassemia, in individuals with non-transfusion-dependent α-thalassemia (HbH disease) by assessing its relevance, comprehensiveness, and interpretability.
METHODS: This qualitative study used a hybrid concept elicitation and cognitive interview design to evaluate the content validity of the NTDT-PRO in patients with non-transfusion-dependent α-thalassemia, HbH disease. The measure includes six items assessing anemia-related symptoms (tiredness, weakness, and shortness of breath, with or without physical activity). Establishing content validity is a key step in validating the NTDT-PRO for use in clinical trials. Twenty-three participants from the US, China, and Italy completed web-based interviews using semi-structured interview guides. Concept elicitation explored symptoms and disease impacts, while cognitive interviews assessed the clarity, relevance, recall period, and response options of the NTDT-PRO. Interviews were audio-recorded, transcribed, translated as needed, and analyzed using thematic analysis.
RESULTS: Twenty-three participants (mean age 32.7 years; 78% female) completed interviews (n=10 China, n=8 Italy, n=5 US). Concept elicitation findings indicated that tiredness (91%), breathlessness (87%), weakness (83%), headache (65%), and sleep problems (57%) were among the most frequently reported symptoms. Common impacts included physical activities (87%), work/school participation (83%), and social life/hobbies (83%), supporting the relevance of the concepts assessed by the NTDT-PRO. Participants generally demonstrated good understanding of the NTDT-PRO and found the items, instructions, recall period, and response options relevant and easy to use. Overall, findings supported the relevance, clarity, and usability of the NTDT-PRO in individuals with non-transfusion-dependent α-thalassemia, HbH disease.
CONCLUSIONS: This hybrid concept elicitation/cognitive interview study supports the content validity of the NTDT-PRO in adults with non-transfusion-dependent α-thalassemia (HbH disease), demonstrating that it captures symptoms and impacts meaningful to patients and supporting its use in future clinical research.
METHODS: This qualitative study used a hybrid concept elicitation and cognitive interview design to evaluate the content validity of the NTDT-PRO in patients with non-transfusion-dependent α-thalassemia, HbH disease. The measure includes six items assessing anemia-related symptoms (tiredness, weakness, and shortness of breath, with or without physical activity). Establishing content validity is a key step in validating the NTDT-PRO for use in clinical trials. Twenty-three participants from the US, China, and Italy completed web-based interviews using semi-structured interview guides. Concept elicitation explored symptoms and disease impacts, while cognitive interviews assessed the clarity, relevance, recall period, and response options of the NTDT-PRO. Interviews were audio-recorded, transcribed, translated as needed, and analyzed using thematic analysis.
RESULTS: Twenty-three participants (mean age 32.7 years; 78% female) completed interviews (n=10 China, n=8 Italy, n=5 US). Concept elicitation findings indicated that tiredness (91%), breathlessness (87%), weakness (83%), headache (65%), and sleep problems (57%) were among the most frequently reported symptoms. Common impacts included physical activities (87%), work/school participation (83%), and social life/hobbies (83%), supporting the relevance of the concepts assessed by the NTDT-PRO. Participants generally demonstrated good understanding of the NTDT-PRO and found the items, instructions, recall period, and response options relevant and easy to use. Overall, findings supported the relevance, clarity, and usability of the NTDT-PRO in individuals with non-transfusion-dependent α-thalassemia, HbH disease.
CONCLUSIONS: This hybrid concept elicitation/cognitive interview study supports the content validity of the NTDT-PRO in adults with non-transfusion-dependent α-thalassemia (HbH disease), demonstrating that it captures symptoms and impacts meaningful to patients and supporting its use in future clinical research.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PT14
Topic
Clinical Outcomes, Patient-Centered Research
Topic Subcategory
Instrument Development, Validation, & Translation, Patient-reported Outcomes & Quality of Life Outcomes
Disease
Rare & Orphan Diseases, Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)