Severe asthma represents a major public health challenge and a socio-economic burden. Currently, there is a lack of a core outcome measurement set for severe asthma that is patient-centred, clinically meaningful to regulators and healthcare professionals. New-generation biological therapies are expensive and therefore it is imperative that the most relevant outcome measures are used to assess therapeutic response. As part of the 3TR consortium, we aim to develop a Core Outcome Measures set for children, adolescents, and adults with Severe Asthma (COMSA). The systematically reviewed evidence will be discussed within a modified GRADE Evidence to Decision framework to identify patient-centred outcome measures for COMSA. The results will inform the development of definitions of response and non-response to biological therapy for severe asthma.
Contributors- Professor Graham Roberts (PI), Professor Ratko Djukanovic, Dr Ekaterina Khaleva, Miss Anna Rattu from the University of Southampton, United Kingdom
- COMSA initiative members
- The European Lung Foundation (ELF)
- European Federation of Allergy and Airways Diseases Patients’ Associations (EFA)
- 3TR Adult Respiratory Patient Working Group (RPWG)
- 3TR Youth Respiratory Patient Working Group (YPWG)
- 3TR Respiratory Work Package 8 (WP8)
Disease Category: Lungs & airways
Disease Name: Severe asthma
Age Range: 6 - 120
Sex: Either
Nature of Intervention: Drug
- Clinical experts
- Consumers (caregivers)
- Consumers (patients)
- Families
- Journal editors
- Patient/ support group representatives
- Pharmaceutical industry representatives
- Regulatory agency representatives
- Researchers
- Service providers
- Service users
- Recommendations for outcome measures (measurement/how)
- Consensus meeting
- Delphi process
- Literature review
- Semi structured discussion
- Survey
- Systematic review
Objective 1: To identify patient-centred outcome measures for severe asthma.
• Part 1 – literature search to identify potential outcome measures (clinical including quality of life and patient reported outcomes, biological, and/or imaging) which could form the Core Outcome Measures (COM) set.
• Part 2 – modified Delphi exercise to decide which outcome measures are critical or important within clinical, patient, regulatory and pharmaceutical industry stakeholder groups.
• Part 3 – systematic review to describe the validation and reliability of selected critical and important outcome measures.
• Part 4 – ELF will undertake a narrative review of patients’ perceptions and opinions about selected outcome measures. If there is limited or a lack of data, we plan to conduct a survey among patients with severe asthma and parents/carers across Europe.
Objective 2: Define a response and non-response to biological therapy for severe asthma.
• A systematic review will be conducted to define a response and non-response to biological therapy for severe asthma.
• If there is lack of evidence, a multi-stage consensus process involving key stakeholders will be used to develop definitions of response and non-response to biological therapy for paediatric and adult severe asthma studies.
The findings from the systematic reviews and modified Delphi exercise will be discussed in a modified GRADE Evidence to Decision framework to make recommendations for a COM set and definition of response and non-response to biological therapy for severe asthma trials and clinical practice.