DEVELOPMENT AND CONTENT VALIDITY OF THE HEMOPHILIA UNIVERSAL TREATMENT SATISFACTION PATIENT REPORTED OUTCOME (HUTS-PRO): A NOVEL PRO FOR MEASURING TREATMENT SATISFACTION ACROSS ROUTES OF ADMINISTRATION
Author(s)
Nuzhat Afroz1, Alexandros Georgiadis, PhD2, Brendan Christopher Jones, PhD, MD3, Michael Kostikas, Pharm D, MSc, MBA4, Peshawa Mahmood, MSc5, Andrea Navarria, MD6, Henrik Schyllert, MASc7, Elodie de Bock, MSc, PhD8.
1Associate COA Director, Novo Nordisk Denmark, 2IQVIA, Athens, Greece, 3Novo Nordisk A/S, Bagsvaerd, Denmark, 4IQVIA, The Hague, Netherlands, 5IQVIA, Amsterdam, Netherlands, 6Novo Nordisk Health Care AG, Zurich, Switzerland, 7Novo Nordisk A/S, Copenhagen, Denmark, 8IQVIA, Courbevoie, France.
1Associate COA Director, Novo Nordisk Denmark, 2IQVIA, Athens, Greece, 3Novo Nordisk A/S, Bagsvaerd, Denmark, 4IQVIA, The Hague, Netherlands, 5IQVIA, Amsterdam, Netherlands, 6Novo Nordisk Health Care AG, Zurich, Switzerland, 7Novo Nordisk A/S, Copenhagen, Denmark, 8IQVIA, Courbevoie, France.
OBJECTIVES: The hemophilia treatment landscape is evolving, with new administration routes entering clinical practice. This study aimed to develop the Hemophilia Universal Treatment Satisfaction Patient Reported Outcome (HUTS-PRO) tool to assess treatment satisfaction across current (e.g., intravenous, subcutaneous) and emerging (e.g., oral) treatments.
METHODS: Consistent with regulatory guidance, HUTS-PRO development included a targeted literature review followed by a four-stage process: concept elicitation (CE) interviews with US-based healthcare professionals (HCPs), adults (≥18 years) and adolescents (12-17 years) with hemophilia A; item generation; cognitive interviews; and iterative modification. Interviews were conducted online, transcribed verbatim, and analyzed using thematic and content analysis.
RESULTS: CE interviews were conducted with five HCPs and 20 adult and 10 adolescent (mean age: 44.5 and 14.6 years) patients. Treatment administration-related concepts reflected both positive and negative drivers. Among 26 participants receiving intravenous prophylactic or on-demand treatment, ease of administration was reported as a positive driver by half of them (n=13). Negative drivers included needle aversion (n=8), infusion difficulty (n=5), and preparation and administration time (n=4). Among 8 participants receiving subcutaneous prophylaxis, positive drivers were easy administration (n=5) and acceptable administration time (n=4). Injection site pain or soreness (n=3) and difficulty with administration method (n=1) were reported as negative drivers. Twenty concepts informed item generation. Cognitive interviews were conducted with 17 adults and 13 adolescents (mean age: 43.1 and 14.6 years) across three waves. Participants found the instructions, items, and response options easy to understand and complete, and the ≥4-week recall period appropriate. Minor terminology issues in 11 of 29 items were addressed to improve clarity.
CONCLUSIONS: The HUTS-PRO tool demonstrated content validity for assessing treatment satisfaction across intravenous, subcutaneous, and emerging oral therapies in adolescents and adults with hemophilia A, enabling assessment of changes in satisfaction across routes of administration. Further studies will be undertaken to evaluate its psychometric properties.
METHODS: Consistent with regulatory guidance, HUTS-PRO development included a targeted literature review followed by a four-stage process: concept elicitation (CE) interviews with US-based healthcare professionals (HCPs), adults (≥18 years) and adolescents (12-17 years) with hemophilia A; item generation; cognitive interviews; and iterative modification. Interviews were conducted online, transcribed verbatim, and analyzed using thematic and content analysis.
RESULTS: CE interviews were conducted with five HCPs and 20 adult and 10 adolescent (mean age: 44.5 and 14.6 years) patients. Treatment administration-related concepts reflected both positive and negative drivers. Among 26 participants receiving intravenous prophylactic or on-demand treatment, ease of administration was reported as a positive driver by half of them (n=13). Negative drivers included needle aversion (n=8), infusion difficulty (n=5), and preparation and administration time (n=4). Among 8 participants receiving subcutaneous prophylaxis, positive drivers were easy administration (n=5) and acceptable administration time (n=4). Injection site pain or soreness (n=3) and difficulty with administration method (n=1) were reported as negative drivers. Twenty concepts informed item generation. Cognitive interviews were conducted with 17 adults and 13 adolescents (mean age: 43.1 and 14.6 years) across three waves. Participants found the instructions, items, and response options easy to understand and complete, and the ≥4-week recall period appropriate. Minor terminology issues in 11 of 29 items were addressed to improve clarity.
CONCLUSIONS: The HUTS-PRO tool demonstrated content validity for assessing treatment satisfaction across intravenous, subcutaneous, and emerging oral therapies in adolescents and adults with hemophilia A, enabling assessment of changes in satisfaction across routes of administration. Further studies will be undertaken to evaluate its psychometric properties.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
PCR41
Topic
Methodological & Statistical Research, Patient-Centered Research, Real World Data & Information Systems
Topic Subcategory
Instrument Development, Validation, & Translation
Disease
Systemic Disorders/Conditions (Anesthesia, Auto-Immune Disorders (n.e.c.), Hematological Disorders (non-oncologic), Pain)