Identifying the Benefits and Risks of Emerging Treatments for Idiopathic Pulmonary Fibrosis: A Qualitative Study

Idiopathic pulmonary fibrosis (IPF) is a rare, progressive, and fatal disease, with very few therapeutic options. Given a paucity of qualitative research to the perspective of patients and other stakeholders in IPF, we sought to identify issues associated with the benefits and risks of emerging treatments and other issues relevant to design of a survey for assessing patient preferences for IPF treatments. Semi-structured key informant interviews were conducted, predominately via telephone, with a range of stakeholder perspectives identified through partnership with a national advocacy organization using a combination of purposive and snowball sampling. Stakeholders were asked guiding questions related to emerging trends impacting IPF patients, likely benefits and risks of emerging treatments, and the outcomes most relevant to patients. Detailed and de-identified field notes were analyzed using interpretive phenomenological analysis (IPA), and a taxonomy of key themes was developed. A total of 20 interviews (participation rate 63%) were conducted with patients/advocates/caregivers (n = 7), providers/researchers (n = 8), and experts associated with policy/industry (n = 5). All interviewees expressed great hope with regards to emerging treatments. Three super-ordinate themes emerged: impact of emerging therapies (spanning the benefits, risks, and unintended consequences of emerging therapies); documenting the patient experience (spanning measuring patient-reported outcomes and quality of life and understanding the burden of disease); and coping with disease progression (including symptom mitigation, lung transplantation, and end-of-life considerations). In identifying issues associated with emerging IPF treatments, we demonstrate the value of qualitative research in understanding the views of diverse stakeholders and in providing a basis for future survey research. As such, qualitative methods should play an important role in understanding the benefits and risks of emerging therapies and in promoting patient-centered drug development.