Patient-reported outcomes in vaccines research: relevance for decision-making

Figures & data

Table 1. Additional uses of PROs

Use of PRO	Example	Details
As an inclusion/exclusion criteria^Citation32	The GOLD initiative for COPD established the GOLD “ABCD” assessment tool to classify COPD patients combining spirometry data, risk of exacerbation and symptoms assessment based on PROs.^Citation33 The CAT is the PRO instrument used to support the GOLD “ABCD”.	The CAT total score is comprised of eight items assessing cough, phlegm, chest tightness, breathlessness, activities, confidence, sleep and energy. The GOLD classification is widely used to define the study population within COPD trials and as inclusion/exclusion criteria.
In the case definition^Citation19	An ARI case identification was triggered using the FLU-PRO. If subjects experienced 1 day of any respiratory symptom from a pre-defined list of respiratory symptoms (e.g. runny/stuffy nose, sore throat, earache or pain) they recorded symptoms daily using the Flu-PRO, an existing PRO instrument.	ARI (primary endpoint): detection of RSV in at least 1 respiratory sample at the time of illness plus ≥1 symptom from any 2 of 3 locations (upper respiratory tract, lower respiratory tract, systemic) from the FLU-PRO instrument.
To define severity of cases^Citation18	HZ patients were classified as developing PHN based on the ZBPI scale, an existing PRO instrument. Suspected HZ cases were asked to complete the ZBPI at home every day from rash onset daily for 28 days, and weekly thereafter until either they had been painfree for 4 consecutive weeks or 90 days had elapsed.	PHN was defined as a ‘worst’ pain score ≥ 3 on the ZBPI scale persisting or appearing 90 days or more after rash onset. The duration of PHN is calculated similarly as the period that pain persists using the worst ZBPI pain score.
Virtual Studies^Citation34	Patients with CKD were followed up using a PRO-based approach to evaluate the effectiveness of the quality of care, use of resources and patient outcomes associated with CKD. A battery of self-assessment questionnaires including the SF-36 domains, EQ-5D and the BIPQ.	The primary endpoint was defined as non-inferiority in loss of renal function (eGFR) comparing patients in the (1) PRO-based remote follow-up and (2) PRO based telephone consultations versus (3) Usual outpatient follow-up (control group)
Surveillance systems to monitor patients^Citation35	Memorial Sloan Kettering Cancer Center performed a randomized controlled trial, in patients receiving chemotherapy, comparing a surveillance system reporting 12 common symptoms via tablet computers versus usual care consisting of symptom monitoring at the discretion of clinicians, A web-based interface STAR previously established for patients with cancer with high symptom burdens was used in association with the EuroQol EQ-5D instrument.	For those individuals using the surveillance system, automated alerts for severe or worsening symptoms were e-mailed to their nurses prompting intervention. The PRO intervention was associated with significantly fewer emergency department visits and improved overall survival, as well as improvements in quality of life.
In Frailty assessment^Citation36,Citation40	A Frailty Index was constructed using an accumulation of deficits approach, incorporating medical comorbidities and PROs. The PRO assessment incorporated a baseline assessment of the SF-36 and EQ-5D instruments.	Study subjects were classified as non-frail; pre-frail; or frail based on the constructed frailty index. Vaccine immunogenicity, efficacy, and safety were evaluated stratified by frailty status.
Public health impact and Cost-Effectiveness modeling^Citation37,Citation38	A multi-cohort Markov model was used to estimate the potential public health and economic impact of the new RZV in the German population ≥ 60 YOA. A prospective study assessing the impact of HZ and PHN on the quality of life of individuals ≥ 50 YOA in Germany was used to estimate utility inputs using the EQ-5D questionnaire.	The QALY loss associated with a HZ only episode (i.e. no PHN) was estimated to be 0.018 in subjects 50–69 YOA and 0.019 in subjects ≥ 70 YOA, and 0.158 for a HZ episode involving PHN.

ARI, acute respiratory illness; BIPQ, brief illness perception questionnaire; CKD, chronic kidney disease; COPD, chronic obstructive pulmonary disease; CAT, chronic obstructive pulmonary disease assessment test; EQ-5D, EuroQol- 5 Dimension; eGFR, estimated glomerular filtration rate; GOLD, global initiative for chronic obstructive lung disease; HZ, herpes zoster; PRO, patient-reported outcome; PHN, post-herpetic neuralgia; RSV, Respiratory Syncytial Virus; RZV, Recombinant Zoster Vaccine; STAR, symptom tracking and reporting; SF-36, Short Form 36; QALY, quality-adjusted life year; YOA, years of age; ZBPI, zoster brief pain inventory

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