Patient and public involvement is suboptimal in randomized controlled trials addressing a chronic condition

Mathilde Husson; Agnès Dechartres; Bruno Ramdjee; Maëlle Diverres; Tarik Zejli; Marianne L'Hénaff; Eric Jehanno; Florence Tubach; Agnès Caille

doi:10.1016/j.jclinepi.2023.06.012

Patient and public involvement is suboptimal in randomized controlled trials addressing a chronic condition

J Clin Epidemiol. 2023 Aug:160:71-82. doi: 10.1016/j.jclinepi.2023.06.012. Epub 2023 Jun 24.

Authors

Mathilde Husson¹, Agnès Dechartres², Bruno Ramdjee¹, Maëlle Diverres¹, Tarik Zejli³, Marianne L'Hénaff⁴, Eric Jehanno⁵, Florence Tubach², Agnès Caille⁶

Affiliations

¹ Département de Santé Publique, AP-HP, Hôpital Pitié-Salpêtrière, Paris 75013, France.
² Département de Santé Publique, INSERM, Institut Pierre Louis d'Epidémiologie et de Santé Publique, AP-HP, Hôpital Pitié-Salpêtrière, Sorbonne Université, Paris 75013, France.
³ INSERM, SPHERE U1246, Université de Tours, Université de Nantes, Tours, France; INSERM CIC 1415, CHRU de Tours, Tours Cedex 9 37044, France.
⁴ Association de Recherche, de Communication et d'Action pour l'accès aux Traitements, Paris, France.
⁵ Association Nationale de Défense contre la Polyarthrite Rhumatoïde, Paris, France.
⁶ Département de Santé Publique, INSERM, Institut Pierre Louis d'Epidémiologie et de Santé Publique, AP-HP, Hôpital Pitié-Salpêtrière, Sorbonne Université, Paris 75013, France; INSERM, SPHERE U1246, Université de Tours, Université de Nantes, Tours, France; INSERM CIC 1415, CHRU de Tours, Tours Cedex 9 37044, France. Electronic address: agnes.caille@univ-tours.fr.

PMID: 37356637
DOI: 10.1016/j.jclinepi.2023.06.012

Abstract

Objectives: To describe patient and public involvement (PPI) in randomized controlled trials (RCTs) addressing a chronic condition and to analyze whether PPI is associated with trial characteristics.

Study design and setting: We used PubMed search to identify RCTs addressing a chronic condition and published in journals with a mandatory PPI statement.

Results: Across 101 RCTs; 40 (40%) reported PPI at any stage of the research process. PPI contribution occurred mostly at the design stage of RCTs (n = 36), especially for assessing the burden of the intervention (n = 24), and at the conduct stage (n = 21), with the elaboration of communication materials (n = 14). Less than one-third (13/40) of RCTs included PPI in the development or choice of outcome measures. As compared with non-PPI RCTs, PPI RCTs more frequently were published in The BMJ, had a corresponding author from the United Kingdom, reported a public funding source, had a higher inclusion rate, used usual care as a control and evaluated a digital intervention. PPI RCTs were associated with less frequent use of placebo as a control group.

Conclusion: Our results underline that PPI is not uncommon in RCTs of chronic conditions but infrequently occurred at a key stage. Yet, the engagement of patients as a real partner in RCTs of chronic conditions should be enhanced.

Keywords: Chronic conditions; Methodological review; Patient and public involvement; Randomized controlled trials.

MeSH terms

Chronic Disease
Humans
Outcome Assessment, Health Care*
Patient Participation*
Randomized Controlled Trials as Topic
United Kingdom