Dataset Information

Concept confirmation of the Treatment Administration Satisfaction Questionnaire (TASQ) in rare paroxysmal nocturnal hemoglobinuria.

ABSTRACT:

Background

This study was conducted to evaluate content validity of the IntraVenous and SubCutaneous Treatment Administration Satisfaction Questionnaires (TASQ-IV and TASQ-SC), for use in a clinical trial population of participants with paroxysmal nocturnal hemoglobinuria (PNH) undergoing eculizumab treatment.

Methods

Participants underwent semi-structured combined brief introduction to disease history and full cognitive debriefing interviews to establish symptoms and key impacts of PNH and to explore the clarity and relevance of both sets of instructions (TASQ-IV and TASQ-SC). The clarity, relevance, response options, and recall period of the TASQ-IV items were also explored.

Results

Ten participants with PNH were recruited. Fatigue was the most commonly reported symptom (n = 7); the most commonly reported impact of PNH was on physical activity (n = 4). Nine participants indicated understanding and relevance of the TASQ-IV instructions; three participants suggested changes. Of the 20 TASQ-IV items, ≥ 15 were considered understandable, relevant and to have suitable response options (n ≥ 8). The TASQ-SC instructions were understood by all participants; seven participants indicated relevance. While a few participants suggested minor changes for the items, these reflected the one-off completion of the measure in an interview setting and were thus not considered sufficient to justify modification of the measure for clinical trial completion.

Conclusions

Most participants understood the TASQ-IV and TASQ-SC instructions (n = 9 and 10, respectively) and the TASQ-IV items were considered clear, relevant and to have suitable response options, demonstrating face and content validity of the instruments for the clinical trial setting.

SUBMITTER: Doll H

PROVIDER: S-EPMC8217362 | biostudies-literature |

REPOSITORIES: biostudies-literature

ACCESS DATA

Similar Datasets

Project description:PurposeTo develop a patient preference questionnaire (PPQ) assessing eculizumab and ravulizumab treatment for paroxysmal nocturnal hemoglobinuria (PNH).Patients and methodsThe development of the PNH-PPQ© was consistent with Food and Drug Administration guidelines for patient-reported outcome measure development, and included 1) a targeted literature review; 2) PNH expert clinician input on treatment preferences; 3) review of existing qualitative data on the PNH treatment and disease experience; 4) concept elicitation interviews with 8 PNH patients who received eculizumab and/or ravulizumab; 5) translatability review; and 6) cognitive debriefing with 5 patients. Interview participants were recruited through a United Kingdom PNH patient advocacy group and a Canadian clinical site involved in clinical trial ALXN1210-PNH-302.ResultsSix themes were identified as most relevant to the PNH treatment experience from the concept elicitation interviews: disease symptoms (n=8/8); treatment frequency (n=7/8); quality of life impact of treatment/disease (n=7/8); treatment burden (n=7/8); treatment efficacy (n=5/8); and treatment side effects (n=5/8). An initial list of 88 preference questions was reduced to 11 highly relevant and non-redundant questions reflecting the 6 themes. Cognitive interview participants unanimously agreed that the PNH-PPQ instructions were clear; response options were understandable, easy to use, and provided enough choices; and the questions captured the factors that inform treatment preferences.DiscussionWhen new drugs have similar efficacy to existing medications, documenting patient preferences is important for confirming patient benefit from the new medication. Understanding what matters most to patients is essential for delivering patient-centered care and may play a particularly significant role in treatment decision making. The availability of such a tool may be especially important as new orphan drugs are developed and patients with rare diseases have more than one treatment option to consider.ConclusionThe PNH-PPQ provides a patient-centered approach for evaluating preferences for the treatment of PNH. The PNH-PPQ has subsequently assessed patient preference in the clinical trial sub-study ALXN1210-PNH-302s.