Introduction
Methods
Search strategy
Systematic review
REF 2014 impact case studies
Units of assessment | ||
---|---|---|
Main panel A | 1 | Clinical medicine |
2 | Public Health, Health Services and Primary Care | |
3 | Allied Health Professions, Dentistry, Nursing and Pharmacy | |
4 | Psychology, Psychiatry and Neuroscience | |
5 | Biological Sciences | |
6 | Agriculture, Veterinary and Food Science |
Eligibility criteria
Data screening
Systematic review
REF 2014 impact case studies
Data extraction/coding
Systematic review
REF 2014 impact case studies
Results
Systematic review
Included studies
Study characteristics
PRO impact types and pathways to impact
PRO impact metrics
Barriers to impact
Trial design
Conduct and analysis
Reporting
Use of PRO data in practice
Facilitators to impact
Barriers to impact | Impact Facilitators |
---|---|
PRO trial design | |
●SPIRIT ●SPIRIT-PRO Exta | |
●SPIRIT ●SPIRIT-PRO Exta | |
Failure to define PRO/HRQL endpoints [47] | ●SPIRIT-PRO Exta |
●SPIRIT-PRO Exta ●ISOQOL Minimum Standards for PRO Measures in patient-centered outcomes and comparative effectiveness researcha | |
●SPIRIT ●SPIRIT-PRO Exta | |
●SPIRIT | |
●SPIRIT-PRO Exta | |
PRO trial conduct and analysis | |
●SPIRIT-PRO Exta ●SISAQOLa | |
●SPIRIT-PRO Exta ●SISAQOLa | |
Lack of communication between researchers and administrators regarding PRO questionnaires involved in the trial [44] | ●SPIRIT-PRO Exta ●SISAQOLa |
●SPIRIT-PRO Exta ●SISAQOLa | |
●SPIRIT-PRO Exta ●SISAQOLa | |
PRO trial reporting | |
●CONSORT ●CONSORT-PRO Ext | |
●CONSORT ●CONSORT-PRO Ext ●SPIRIT-PRO Ext | |
●CONSORT | |
●CONSORT-PRO Ext ●SPIRIT-PRO Ext | |
●CONSORT-PRO Ext ●SPIRIT-PRO Ext | |
●CONSORT ●SPIRIT-PRO Ext | |
●CONSORT ●CONSORT-PRO Ext | |
CONSORT-PRO Ext | |
●CONSORT ●CONSORT-PRO Ext | |
●CONSORT ●SPIRIT-PRO Ext | |
CONSORT-PRO Ext | |
●CONSORT ●SPIRIT-PRO Ext | |
Discrepancies between PRO protocol and PRO trial report [44] | ●CONSORT ●SPIRIT-PRO Ext |
●Journals should allow space to report HRQL data alongside other clinical outcomes [50] | |
Barriers to uptake of PRO trial results in practice | |
●PROlearna ●SPIRIT-PRO Ext ●Provide training to clinicians to gain confidence regarding the validity and reliability of HRQL instruments [67] | |
●PROlearna ●Clinician’s checklist for reading and using an article about patient-reported outcomesa | |
Clinicians concerns about the PRO results being biased by missing data [77] | ●PROlearna ●Provide training to clinicians to gain confidence regarding the validity and reliability of HRQL instruments [67] ●Clinician’s checklist for reading and using an article about patient-reported outcomesa |
●CONSORT ●Clinician’s checklist for reading and using an article about patient-reported outcomesa | |
Concerns that the PRO results were chance findings arising from multiple testing [77] | ●PROlearna ●Provide training to clinicians to gain confidence regarding the validity and reliability of HRQL instruments [67] ●Clinician’s checklist for reading and using an article about patient-reported outcomesa |
●Stakeholder-driven, evidence-based standards for presenting PROs in clinical practicea | |
Lack of time to discuss PRO outcomes with patients [67] | ●PROlearna ●Provide consistent and improved HRQL data reports and a summary of the clinical implications of the HRQL results [67] ●Provide training to clinicians to gain confidence regarding the validity and reliability of HRQL instruments [67] |
●SPIRIT-PRO Exta |
REF 2014 impact case studies
Included studies
PRO clinical trials characteristics
Trial characteristics | Number of trials, (%) |
---|---|
Trial phase | |
I | 0 |
I/II | 1 (1.4) |
II | 1 (1.4) |
III | 24 (34) |
Other | 3 (5.7) |
Not specified | 40 (57) |
Leading study centre | |
UK | 62 (89) |
International | 7 (11) |
Trial design | |
International multicentre study | 21(30) |
PRO outcome | |
Primary outcome | 17 (24) |
Secondary outcome | 35 (50) |
Both | 11 (15) |
PRO measures used | |
SF-36 | 17 (24) |
EQ-5D | 12 (17) |
HADS | 9 (13) |
VAS | 9 (13) |
EORTC QLQ-C30 | 3 (4) |
Other | 70a |
Real-world evidence of PRO impact
Direct PRO impact
Indirect PRO impact
Absence of evidence around PRO impact
Discussion
The Cardiac Resynchronisation — Heart Failure (CARE-HF) trial demonstrated that the cardiac resynchronisation therapy reduced the risk of complications and death among patients with left ventricular systolic dysfunction and cardiac dyssynchrony who had moderate or severe heart failure [85, 86]. In addition, as measured with the EQ-5D and Minnesota Living with Heart Failure Questionnaire (MLWHF), the therapy was associated with quality of life and symptoms improvement [86]. ●The main trial publication was characterised for complying with the different facilitators identified by the systematic review and for adhering to the SPIRIT PRO Extension and CONSORT PRO Extension guidelines despite these guidelines being published subsequently. ●Low rates of PRO missing data (8%) and statistical methods for dealing with missing data were reported. ●The PRO data was included in the main RCT report and alongside other clinical data [87]. In addition, there were detailed and timely secondary PRO publications [86, 88, 89]. Attributing impact directly to PRO data is difficult given the survival benefit; however, this well designed, conducted, analysed and reported trial led to impact that could be measured through the following impact metrics: ●In the short term, PRO results were included in the main trial publication, [87] which led to 4927 citations by January 2018. At least 4 additional PRO trial publications are available. ●In the mid-term, PRO trial findings were incorporated in clinical guidelines and health policy at national and international level: NICE in the UK, [90] the European Society of Cardiology, [91] the European Society of Cardiology in Canada, [92] Brazil, [93] and USA [94]. Therefore, the use of CRT influenced the healthcare practice at national and international level by providing the CRT to patients with heart failure and dyssynchrony. ●In the long-term, an additional study assessing the effects of the CARE-HF trial on quality of life demonstrated that the device improved quality of life and symptoms and improved survival among the users [88]. In addition, PRO results informed the cost-effectiveness analysis of the intervention and the production of the device, [89, 95] which led to increased income from industry: ‘the world market for CRT devices is projected to grow to $2.8 billion annually by 2015’. [81] The cost-effectiveness analysis demonstrated that CRT is cost-effective when compared with medical therapy alone (MT). In the same way, CRT plus cardioverter-defibrillator is more cost-effective when compared to CRT + MT.[95] |