The development of a measure to better understand patient experiences with pirfenidone and nintedanib for the treatment of idiopathic pulmonary fibrosis (IPF)

Background: Optimal treatment of IPF remains challenging. Recent guidelines recommend two anti-fibrotic agents: pirfenidone and nintedanib. Besides traditional physiological end-points such as forced vital capacity, the patient9s perceived effectiveness, influence of treatment on daily life, and satisfaction are crucial outcomes that impact clinical management choices and might support reimbursement decisions. Aim: To develop a patient-reported outcome measure to evaluate Patient Experiences and SAtisfaction with Medication in IPF – PESaM questionnaire. Methods: We followed recognised methodology involving literature review, patient interviews (n=2), two focus groups (n=13), expert opinion, pre-testing of face and content validity using retrospective verbal probing (n=9), and evaluation of psychometric properties (n=100). Results: Two complementary measures were developed; a generic measure that can be used for any medication (16 items), and a measure focused on pirfenidone and nintedanib (10 items). The measures cover the following domains: perceived effectiveness , perceived side-effects, ease of use and satisfaction. Face and content validity were considered good. Psychometric properties are under evaluation. The PESaM questionnaires is completed every 3 months as part of the Dutch Registry for treatment of IPF. Conclusion: We developed two patient-reported measures to assess experiences with medication in general and with pirfenidone and nintedanib specifically. In future these tools could be used in clinical practice to assist shared-decision making on treatment choices.