“Patient Reported Outcomes in Severe Asthma: Fit for Purpose?” presented by Prof. Janelle Yorke
As part of our ongoing seminar series, the Centre of Research Excellence in Severe Asthma hosted Prof. Janelle Yorke, for a webinar on “Patient-Reported Outcomes In Severe Asthma: Fit For Purpose?” on 21 March, 2018.
Presentation Summary:
Patient reported outcome measures (PROMs) assess the impact of disease from a patient perspective. PROMs are increasingly used to assess the effects of disease and treatment. However, PROMs are rarely used as primary outcomes in clinical trials.
A range of health-related quality of life PROMs have been used in asthma studies. However, no PROMs have been developed specifically for severe asthma. Existing PROMs fail to assess factors that are unique to severe asthma. These include frequent hospitalisations and frequent use of oral corticosteroids (OCS).
PROM development is a lengthy and rigorous process. Questionnaire development involves initial item generation, refinement, reduction, validation and re-testing. Careful consideration must be made when framing questions and developing answer options. The intended PROM use (e.g. clinical utility vs. research) also shapes development.
Prof. Yorke presents preliminary findings on the development of a severe asthma PROM. This project is a collaboration with Prof. Vanessa McDonald. Preliminary data from patient interviews about disease exacerbations highlights a range of domains relevant to patient experience. Further, findings highlight the importance of terminology with patients predominantly using the term “attack”.
Key Points:
- Patient reported outcome measures (PROMs) assess disease experiences and outcomes from a patient perspective
- PROMs provide insight into patients’ experiences related to quality of life, symptoms and other healthcare outcomes
- Health process metrics (e.g. exacerbations, health care utilisation) are commonly included in clinical trials
- Objective measures are also used to quantify disease-relevant metrics (e.g. lung function)
- PROMs are increasingly being used to assess patient experience of living with disease and effects of treatment
- There is often a disconnect between treatment effects on objective disease measures and patient reported outcomes
- PROMs are rarely included as primary outcomes in clinical trials
- PROM development is a lengthy and rigorous process, which can be informed by FDA guidelines (FDA 2009)
- PROM development involves creating an initial conceptual framework, item generation, design choices and refinement, item reduction, reliability and validity testing and questionnaire re-testing
- Healthy-related quality of life is complex, including a range of variables and domains
- Quality of life PROM questionnaires can be generic, condition/organ-specific or disease-specific
- Health-related quality-of-life PROMs assess whether treatment is worthwhile from the patient perspective and provide information on illness trajectory
- Asthma PROMs should assess both the burden of disease and the burden of treatment
- PROMs currently used in to assess asthma include generic, respiratory and asthma-related quality of life assessments
- However, no PROMs have been developed specifically for severe asthma
- Severe asthma is increasingly recognised to be different from mild-to-moderate asthma, in particular the impact of frequent exacerbations and oral corticosteroids (OCS)
- Existing PROMs fail to assess factors unique to severe asthma
- In preliminary analysis, patients with severe asthma describe a broad range of domains when discussing asthma exacerbations
- Patients predominantly use the term “attack” to describe disease exacerbations (93%), while very few use the terms “exacerbation” or “flare-up”
- When developing PROMs, it is important to consider the intended use (e.g. clinical utility versus research applications)
- To be useful in clinical practice, questionnaires must be shorter but also maintain sensitivity
- When assessing disease symptoms, it is important to consider the framing of questions, options for answers and the time-frames being assessed
- Existing asthma PROMs may be providing an overly positive estimate of quality of life in patients with severe asthma
- They lack assessment of the impact of treatment, particularly frequent exposure to oral corticosteroids (OCS) and disease-specific symptoms
About Prof. Janelle Yorke:
Prof. Janelle Yorke is the founding Chair of Christie Patient Centred Research (CPCR) and Deputy-lead of the Supportive Palliative Care (SuPaC) group at University of Manchester.
A primary objective of her work has been to improve symptom experience and quality of life for people living with a long-term illness, through the development of measurement tools, better understanding of the illness experience and palliative care needs of patients and their families, and the testing of multifaceted non-pharmacological interventions.
She has particular expertise in the development and validation of Patient Reported Outcome (and Experience) Measures (PROMS/PREMS) using advanced psychometric techniques, including Rasch analysis and is collaborating with Prof Vanessa McDonald to develop a severe asthma-specific quality of life tool.
