Development and Pretesting of a Questionnaire to Assess Patient Experiences and Satisfaction with Medications (PESaM Questionnaire)

Abstract

BackgroundThe aim of this study was to develop, together with the Lung Foundation Netherlands and Dutch Kidney Patients Association, patients and clinicians, a measure to evaluate patient experiences with the orphan drugs pirfenidone (for idiopathic pulmonary fibrosis [IPF]) and eculizumab (for atypical haemolytic uraemic syndrome [aHUS]), as well as a generic measure of patient experiences and satisfaction with medications.MethodsDevelopment of the Patient Experiences and Satisfaction with Medications (PESaM) questionnaire consisted of four phases: literature review (phase I); focus groups and individual patient interviews (phase II); item generation (phase III); and face and content validity testing (phase IV). Literature review aimed to identify existing disease-specific and generic patient experience measures to provide guidance on the domains of medication use relevant to patients, the number of items and type of response categories, and to generate an initial pool of items. Subsequent focus groups and patient interviews were conducted to gain insight into the perceived effectiveness of the therapies, the burden of side effects, and how the medication impacted on a patient’s daily life. Focus groups and interviews were recorded and transcribed verbatim. Coding was carried out by highlighting passages in the text and assigning each passage a code representing the following predefined categories: (1) perceived effectiveness; (2) side effects; (3) ease of use; and (4) impact of medication. Using data from phase I and II, a panel of experts selected items relevant for inclusion in the questionnaire. Individual patient interviews with IPF and aHUS patients (n = 18), using a retrospective verbal probing technique, were conducted to assess face validity, time needed to fill out the questionnaire, and content validity.ResultsThe PESaM questionnaire that was developed consisted of two disease-specific modules that assessed patient experiences with pirfenidone for the treatment of IPF, and eculizumab for the treatment of aHUS, a generic module, applicable to any medication, and a module to assess patient expectations. Review of the literature identified multiple disease- or medication-specific questionnaires and two generic patient satisfaction questionnaires. Common domains across most questionnaires were effectiveness, side effects, ease of use and overall satisfaction. Patient interviews revealed the social impact (e.g. unable to go outside) of side effects such as photosensitivity associated with pirfenidone and the risk of infection associated with eculizumab. Each PESaM module focuses on patients’ perceived effectiveness of the medication, side effects, and ease of use, and the impact these aspects have on physical and emotional health and daily life. The generic module additionally includes items related to satisfaction with the medication. Individual interviews with patients in phase IV confirmed, in general, that questions and response options of the modules were clear and content validity was good. The mean ...

<p>Assessing Preferences for Rare Disease Treatment: Qualitative Development of the Paroxysmal Nocturnal Hemoglobinuria Patient Preference Questionnaire (PNH-PPQ©)</p>

“…The main limitation of the study was sample size, which was constrained largely by the rare nature of PNH. 31 Moreover, we were limited in the number of patients for concept elicitation interviews because the optimal participant was one who had experience with both eculizumab and ravulizumab, and ravulizumab was only available through participation in the clinical trial. However, there was consistency among our study participants around the key issues for PNH treatment preferences, which were further confirmed by expert and existing patient ethnography data.…”

“…Eleven patient preference questionnaires that most closely aligned with the objectives of this project were identified from the literature; [26][27][28][29][30][31][32][33][34][35][36] however, none of them evaluated patient preference and dosing schedules in a manner suitable for our aims. After reviewing the literature and the 11 questionnaires, the following concepts were identified as potentially relevant to our measure: symptom relief; effectiveness; ease of use/convenience/bother; impact on daily life; and medication preference.…”

Patients' perceptions and patient-reported outcomes in progressive-fibrosing interstitial lung diseases

“…Because each patient is an individual, the effects of a disease or its treatment may vary from person to person, depending on several factors. Investigators in the Netherlands have developed the patient experiences and satisfaction with medications measure [42] to assess tolerance and perceived effectiveness of pirfenidone in IPF.…”

Validity of the Patient Experiences and Satisfaction with Medications (PESaM) Questionnaire

“…The Patient Experiences and Satisfaction with Medications (PESaM) questionnaire is a recently developed patient-reported outcome measure (PROM) for quantitative assessment of patient experiences and satisfaction with (novel) drug therapies [ 21 ]. It was developed in response to the request of two patient organizations that wanted to better capture patients’ experience with expensive orphan drugs that had been granted conditional approval for reimbursement by the Dutch government.…”

“…It comprises two disease-specific modules evaluating drug treatment for IPF and aHUS, a generic module applicable to any medication and a module focused on patient expectations of the medication. The format and content of the PESaM questionnaire were based on a formal conceptual framework [ 29 ], a literature review and input from patients through focus groups and individual interviews [ 21 ]. Development and pre-testing of the questionnaire has been described extensively elsewhere [ 21 ].…”