Just Published: Systematic Review of PRO Use in Paediatric Populations with Vaccine-Preventable Infectious Diseases

This paper reports the results of a systematic review of the use and quality of Patient Reported Outcome (PRO) measures in paediatric populations with vaccine-preventable infectious diseases in developed countries.

The results of a systematic review on the use of patient reported outcomes (PROs) in paediatric populations with infectious diseases were recently published in Value in Health. 

The Patient-Reported Outcomes in Children with Infectious Diseases (PROCHID) study was performed in 2013 – 2014 by a team of researchers from OHE in collaboration with members of Sanofi Pasteur MSD’s HEOR team and focused on the use of PRO measures in any of 17 vaccine-preventable infectious diseases. 

To ensure feasibility, the review was restricted to the use of PROs in regions and countries where PROs are widely used and can contribute to decision making on the value of treatments (Europe, North America, Australia and New Zealand). 

Searches were performed in the Scopus and Psychlit electronic databases in conjunction with manual searching of reference lists in relevant articles.

Although a large number of titles and abstracts were reviewed, only a small number of full-text articles (n=17) were included for data extraction, indicating the dearth of research in this area. No PRO studies in paediatric populations were identified for 9 of the 17 conditions studied. 

PRO instruments used in the studies identified included a range of generic and disease-specific measures. The majority of the instruments used in the studies identified were of satisfactory or good quality in terms of their psychometric performance. 

In some instances, questionnaires for adults were used which might be unsuitable for younger populations, and there were other cases where proxy perceptions of children’s health were obtained when it would have been possible to obtain children’s perceptions directly. 

Only one questionnaire was identified which had been specifically designed for children with one of the diseases included, though it was still in the development stage.

The authors conclude that the lack of studies and disease-specific PRO instruments in paediatric populations with vaccine-preventable infectious diseases is a serious obstacle to the comprehensive assessment of the impact of those conditions or the benefits of their prevention and treatment. 

Access the full paper here.

This paper is one of two to be published in Value in Health stemming from the PROCHID study. The other is a review of the derivation and use of utility weights in paediatric populations with any of the same 17 vaccine-preventable diseases and has also been accepted for publication. 

In recent years, members of OHE have contributed to several studies involving the development and/or use of PRO measures in younger populations, including:

• development [open access] and validation [open access] of the EQ-5D-Y, the version of EQ-5D for children
• development and validation of a condition-specific measure of HRQOL for use in short stature children [open access]
• development and use of a widely used, generic measure of children’s HRQOL, the KIDSCREEN [open access]
• exploration of socioeconomic inequalities in HRQOL in children and adolescents across Europe.