title: Development and Pretesting of a Questionnaire to Assess Patient Experiences and Satisfaction with Medications (PESaM Questionnaire).
abstract: BACKGROUND
  The 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.
  
  
  METHODS
  Development 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.
  
  
  RESULTS
  The 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 time to complete the modules ranged from 6 min for the disease-specific (aHUS) module to 9 min for the generic module.
  
  
  CONCLUSIONS
  We developed the PESaM questionnaire to quantitatively assess patient experiences and satisfaction with medications. A validation study is currently underway to examine the psychometric properties of the PESaM questionnaire.
authors.affiliation: Department of Clinical Epidemiology and Medical Technology Assessment, Maastricht University Medical Centre, Maastricht, The Netherlands. merel.kimman@mumc.nl.
authors.forename: Merel L
authors.initials: ML
authors.lastname: Kimman
authors.affiliation: Department of Clinical Epidemiology and Medical Technology Assessment, Maastricht University Medical Centre, Maastricht, The Netherlands.
authors.forename: Adrienne H
authors.initials: AH
authors.lastname: Rotteveel
authors.affiliation: Department of Respiratory Medicine, Erasmus Medical Centre, University Hospital Rotterdam, Rotterdam, The Netherlands.
authors.forename: Marlies
authors.initials: M
authors.lastname: Wijsenbeek
authors.affiliation: Department of Pulmonary Medicine, Zuyderland Medical Centre, Heerlen, The Netherlands.
authors.forename: Rémy
authors.initials: R
authors.lastname: Mostard
authors.affiliation: Department of Respiratory Medicine, Erasmus Medical Centre, University Hospital Rotterdam, Rotterdam, The Netherlands.
authors.forename: Nelleke C
authors.initials: NC
authors.lastname: Tak
authors.affiliation: Lung Foundation Netherlands, Amersfoort, The Netherlands.
authors.forename: Xana
authors.initials: X
authors.lastname: van Jaarsveld
authors.affiliation: Kidney Patients Association, Bussum, The Netherlands.
authors.forename: Marjolein
authors.initials: M
authors.lastname: Storm
authors.affiliation: Department of Paediatric Nephrology, Radboud University Medical Center, Amalia Children's Hospital, Nijmegen, The Netherlands.
authors.forename: Kioa L
authors.initials: KL
authors.lastname: Wijnsma
authors.affiliation: Department of Internal Medicine, Division of Nephrology, Maastricht University Medical Centre, Maastricht, The Netherlands.
authors.forename: Marielle
authors.initials: M
authors.lastname: Gelens
authors.affiliation: Department of Paediatric Nephrology, Radboud University Medical Center, Amalia Children's Hospital, Nijmegen, The Netherlands.
authors.forename: Nicole C A J
authors.initials: NCAJ
authors.lastname: van de Kar
authors.affiliation: Department of Internal Medicine, Division of Nephrology, Radboud University Medical Center, Nijmegen, The Netherlands.
authors.forename: Jack
authors.initials: J
authors.lastname: Wetzels
authors.affiliation: Department of Clinical Epidemiology and Medical Technology Assessment, Maastricht University Medical Centre, Maastricht, The Netherlands.
authors.forename: Carmen D
authors.initials: CD
authors.lastname: Dirksen
mesh_terms: D000368:Aged; D061067:Antibodies, Monoclonal, Humanized / Q000008:administration & dosage / Q000009:adverse effects*; D000970:Antineoplastic Agents / Q000008:administration & dosage / Q000009:adverse effects; D065766:Atypical Hemolytic Uremic Syndrome / Q000188:drug therapy; D005260:Female; D006801:Humans; D054990:Idiopathic Pulmonary Fibrosis / Q000188:drug therapy; D007407:Interviews as Topic; D008297:Male; D008875:Middle Aged; D009426:Netherlands; D009965:Orphan Drug Production; D057240:Patient Preference / Q000523:psychology*; D017060:Patient Satisfaction; D011594:Psychometrics; D011728:Pyridones / Q000008:administration & dosage / Q000009:adverse effects*; D011788:Quality of Life; D015203:Reproducibility of Results; D012189:Retrospective Studies; D011795:Surveys and Questionnaires / Q000592:standards*
chemical_list: D061067:Antibodies, Monoclonal, Humanized; D000970:Antineoplastic Agents; D011728:Pyridones; C481642:eculizumab; C093844:pirfenidone
keywords: Eculizumab; Focus Group; Idiopathic Pulmonary Fibrosis; Orphan Drug; Patient Experience