The need for patient-centred clinical research in idiopathic pulmonary fibrosis

Abstract

Patient-centredness is an accepted term and is perceived by healthcare professionals to be morally and ethically desirable. We are motivated by the belief that this approach will improve the patient-professional experience of the decision-making process and improve health outcomes. We acknowledge that patients, either as participants or as co-investigators, have positive contributions to make to research. As the idiopathic pulmonary fibrosis (IPF) community enters a new era of clinical research activity we consider that there is greater capacity for patient involvement and partnership.Patient involvement in research can be optimised through collaborations in the research design, study conduct, and dissemination. There is increasing interest in using patient- reported outcomes (PROs), such as health-related quality of life, and symptoms measures to inform decision-making and ensure patient perspectives are taken into account. PROs are an essential component of specialist IPF services, to monitor and improve care delivery and to measure and benchmark performance. In clinical trials, PROs can additionally be used to define entry criteria, evaluate efficacy of an intervention, and evaluate adverse events. We suggest that there is a much wider scope for including patient-centred PROs in clinical research and for creative thought in developing patient co-investigator roles.Participation in research activity requires highly refined decision-making processes, particularly in a condition such as IPF, which has an often unpredictable trajectory. The IPF research landscape has changed and the design and conduct of clinical trials in IPF requires some radical rethinking. It is accepted that involving patients in the role of co-investigators will impact the research questions we ask and result in study designs that are patient-centred. IPF clinical trials have been hindered by the lack of availability of validated, disease-specific questionnaires. A conservative approach appears to have been taken to the inclusion of generic symptom or quality of life measures as PRO endpoints. Thus, the impact of new drugs on the quality of life of research participants demonstrates only minimal benefit. It is time to refocus on a patient-centred approach with regards to the co-investigator role, PRO development, and research participants.

Therapy for idiopathic pulmonary fibrosis: lessons from pooled data analyses

“…In an era of increasingly personalised medicine and with an evolving understanding of specific biological mechanisms, it is likely that combination therapy will be the way of the future. Patient-driven outcomes, including cough, dyspnoea and quality of life, will also require focused attention if we are to make further inroads in this disease [22]. In the real word, physicians are still facing a series of unanswered questions.…”

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

“…Cough and activity-limiting dyspnoea have immense impact on the everyday lives of patients with IPF and other ILDs [2,[10][11][12]. Cough can affect sleep, willingness to participate in social activities [9] and thus, physical and emotional well-being.…”

“…Cough can affect sleep, willingness to participate in social activities [9] and thus, physical and emotional well-being. Fatigue can be equally debilitating and lead to decreased social participation, physical deconditioning, low mood and isolation [2,10,12,13].…”

“…Besides posing a substantial threat to patients' physical well-being, PF-ILD can negatively impact patients' mental well-being. Patients may suffer from bouts of anxiety and/or depression and develop feelings of grief and anger [9,12]. Living with, and coming to terms with a terminal illness, is remarkably difficult [6].…”

Best supportive care for idiopathic pulmonary fibrosis: current gaps and future directions

“…Although based on expert opinion with very few studies supporting this approach, it is recommended that access to specialised healthcare professionals should be regulated via referrals from primary care to specialists and thereafter to specialised centres [19]. At the tertiary level, a very important role is played by nurses specialising in the care of patients with ILDs [21,22]. Indeed, as the main patient contacts in the healthcare system, they play a pivotal role in addressing a patient's needs and in coordinating a multidisciplinary team of other professionals around the patient.…”