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Table 4 Practical guide for researchers

From: The impact of patient-reported outcome (PRO) data from clinical trials: a systematic review and critical analysis

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]