Development and First Use of the Patient’s Qualitative Assessment of Treatment (PQAT) Questionnaire in Type 2 Diabetes Mellitus to Explore Individualised Benefit–Harm of Drugs Received During Clinical Studies

Abstract

Introduction Individualised benefit-harm assessments can help identify patient-perceived benefits and harms of a treatment, and associated trade-offs that may influence patients' willingness to use a treatment. This research presents the first use of a patient-reported outcome measure designed to assess patient-perceived benefits and disadvantages of drugs received during clinical studies. Methods The Patient's Qualitative Assessment of Treatment (PQAT) was developed in English and cognitively tested with US (n = 4) and Canadian (n = 3) patients with type 1 and type 2 diabetes mellitus (T2DM). The revised version of the PQAT comprises three qualitative open-ended questions focused on the benefits and disadvantages of treatment and reasons why patients would choose to continue/discontinue treatment. A final quantitative question asks patients to evaluate the balance between benefits and disadvantages using a 7-point scale. The revised version of the questionnaire was administered as an exploratory endpoint in a phase II clinical trial for a new injectable treatment for T2DM. Qualitative data were analysed using thematic analysis, and relationships between qualitative and quantitative data were identified. Results Patient-reported benefits of treatment administered during the clinical trial included clinical markers of efficacy and subjective markers. Disadvantages reported by patients were mainly related to drug adverse effects or to the mode of administration. Of the 57 patients completing the PQAT, 70.2% reported being willing to continue treatment, with 59.6% reporting that the benefits outweighed the disadvantages. The reported benefits of feeling better and improved energy levels were more likely to be associated with a more positive ratio (70% and 71.4%, respectively), while the disadvantages of fatigue, headaches, and stomach pain were associated with a negative ratio and patients not being willing to continue the treatment. Conclusions The PQAT is a unique patient-reported outcome tool designed to aid understanding patients' real experience of benefits and disadvantages of a treatment. It combines the richness of qualitative data with quantitative data-information valuable for various stakeholders to make well-informed treatment decisions. Trial Registration ClinicalTrials.gov identifier: NCT02973321.

Patient-Reported Questionnaires to Identify Adverse Drug Reactions: A Systematic Review

“…After screening 1563 studies once 1171 duplicates were removed, 78 full-text articles were assessed for eligibility. A total of 19 patient-reported questionnaires met the inclusion criteria for this systematic review [7,15,16,[27][28][29][30][31][32][33][34][35][36][37][38][39][40][41][42]. The PRISMA flowchart for study selection is presented in Figure 1.…”

“…Table 2 shows a breakdown of the different characteristics of each of the questionnaires such as which medication classes they focus on and the number of side-effect-related items and domains or sections within the questionnaire. Out of the 19 questionnaires, eight (42%) focused on mental health medications [15,27,31,35,36,39,41,42], four (21%) were general questionnaires applicable to any medication [7,16,28,38], two (11%) focused on antiepileptics [30,40], two (11%) focused on inhaled medications for asthma and COPD [29,33], one (5%) focused on diabetes medications [34], one (5%) focused on chemotherapy [37], and one (5%) focused on triptans [32] (Table 1). Table 2 shows a breakdown of the different characteristics of each of the questionnaires such as which medication classes they focus on and the number of side-effectrelated items and domains or sections within the questionnaire.…”

“…Table 2 shows a breakdown of the different characteristics of each of the questionnaires such as which medication classes they focus on and the number of side-effectrelated items and domains or sections within the questionnaire. Antiepileptics, n = 2 [30,40] Side-Effect Checklist Assessment of Side-Effects in AED treatment (SIDAED) Asthma and chronic obstructive pulmonary disease, n = 2 [29,33] Satisfaction with Asthma Treatment Questionnaire Inhaled Corticosteroids Questionnaire (ICQ) Diabetes, n = 1 [34] Patient's Qualitative Assessment of Treatment (PQAT) Chemotherapy, n = 1 [37] Common Terminology Criteria for Adverse Events Side-Effects Questionnaire Triptans, n = 1 [32] Triptans Questionnaire Only nine questionnaires (47%) used a scoring system, with six out of the nine questionnaires having a scoring system with a minimum and a maximum score. Out of these questionnaires mentioning the range of potential scores, the GASS questionnaire was the only tool to provide explicit advice regarding interpretation of scores.…”

Patient Global Impression of Benefit–Risk (PGI-BR): Incorporating Patients’ Views of Clinical Benefit–Risk into Assessment of New Medicines

“…The WIWI questionnaire endeavors towards assessing patients' decision-making regarding treatment in a clinical trial [21]. The PQAT was designed to assess the benefits and disadvantages of new treatments for type 2 diabetes mellitus, and was recently used as part of a clinical study [24]. It comprises a question with fixed response options on patients' overall treatment experience and open-ended questions for free-text responses.…”

“…It comprises a question with fixed response options on patients' overall treatment experience and open-ended questions for free-text responses. In contrast with the current questionnaire, the content of the PQAT was developed without direct patient input, and only underwent cognitive testing in seven patients with diabetes mellitus [24]. By comparison, the current instrument is a patient-driven, patient-centric PRO questionnaire with established content validity using best practices for instrument development [6][7][8][9], and assesses the benefit-risk of 'good' and 'bad' experiences with an individual treatment across a range of therapeutic areas.…”