Chronic Obstructive Pulmonary Disease (COPD)

Chronic Obstructive Pulmonary Disease (COPD) is a common inflammatory disease of the lungs, caused in particular by long-term smoking and resulting in breathing difficulties and other respiratory symptoms. Clinical trials in the past have focused on improving the lung function (breathlessness) or reducing the number of flare-ups (exacerbations) requiring hospital visits, resulting in a number of medications to treat these aspects of the disease. 

Whilst there are a number of medications which help with the breathlessness and reduce exacerbations, COPD patients also suffer from other symptoms on a daily basis, like chronic cough and excess mucus secretion, which greatly affect their lives and are poorly managed by the current therapies. The purpose of the present study was to quantify the relative importance that patients place on alleviating different COPD symptoms, with a view to informing the design of clinical trials in COPD, such that endpoints that matter most to patients are taken into account in future trial designs.

We performed qualitative research analyzing social media and online community discussions to highlight the burden of cough and mucus for COPD patients, revealing that these symptoms can lead to disturbed sleep, fatigue, urinary incontinence and social anxiety/embarrassment. We then conducted a  5-country patient preference study with 1050 COPD patients,  to show just how important these other symptoms are to patients, compared to breathlessness and exacerbations, and how important it would be to them to be able to alleviate these different consequences of their COPD. Patients found relevance in all six attributes tested - exacerbations, sleep disturbance, breathlessness, incontinence, mucus clearance effort, and cough. Preference share analysis showed the significant value to patients of health states where the novel clinical endpoints (cough, mucus, incontinence, sleep) were improved.

Publications

Cook NS, Criner GJ, Burgel P-R et al. People living with Moderate-to-Severe COPD Prefer Improvement of Daily Symptoms Over the Improvement of Exacerbations: A Multi-Country Patient Preference Study. European Respiratory Journal – Open Research (2022) In Press

Cook NS, Kostikas K, Gruenberger J-B, et al. Patients’ perspectives on COPD:
findings from a social media listening study- ERJ Open Res 2019; 5: 00128-2018 

Cook N, Gey J, Oezel B, et al. Impact of cough and mucus on COPD patients: primary insights from an exploratory study with an Online Patient Community. Int J Chron Obstruct Pulmon Dis. 2019;14:1365-1376. Published 2019 Jun 24. doi:10.2147/COPD.S202580

Patalano F, Gutzwiller FS, Shah B et al. Gathering Structured Patient Insight to Drive the PRO Strategy in COPD: Patient-Centric Drug Development from Theory to Practice. Adv Ther 2019;37:17–26

Fact sheet

Therapeutic area	Chronic Obstructive Pulmonary Disease (COPD)
Study led by	Novartis
PREFER leads team	Nigel Cook
MPLC decision point of interest	Input to phase III clinical trial design
PREFER case study acronym	COPD
Clinical objectives	Quantify the relative needs and preferences of COPD patients regarding symptoms, the impact on their QOL, and evaluate whether preferences vary with certain respondent characteristics.
Patients from	Five countries (US, UK, France, Australia, Japan) recruited through patient groups and supplemented through patient panels
Methods in Qualitative Study	Literature review, social media listening, online bulletin boards, qualitative in-depth telephone interviews
Methods in Quantitative Study	Discrete Choice Experiment (DCE) for the preferences elicitation Various PRO instruments and other questions also included in survey to enable sub-group analyses
End-date qualitative data collection	June 2019
End-date quantitative data collection	January 2020