International PRO Guidance and Regulatory Science

The SPIRIT-PRO guidelines provide recommendations for items that should be addressed and included in clinical trial protocols in which Patient Reported Outcomes (PROs) are a primary or key secondary outcome. Improved design of clinical trials including PROs could help ensure high-quality data that may inform patient-centred care.

Related links:

• Guidelines for Inclusion of Patient-Reported Outcomes in Clinical Trial Protocols

• ‘Give Us The Tools!’: development of knowledge transfer tools to support the involvement of patient partners in the development of clinical trial protocols with patient-reported outcomes (PROs), in accordance with SPIRIT-PRO Extension

SISAQOL-IMI (Setting International Standards in Analysing Patient-Reported Outcomes and Quality of Life Endpoints in Cancer Clinical Trials – IMI) provides recommendations to standardize the use, analysis, and interpretation of patient reported outcome (PRO) data in cancer clinical trials.

Related links:

• SISAQOL-IMI website

The aim of this work is to develop an electronic patient-reported outcomes platform to collect symptom, adverse event and tolerability data in an early phase trial, and to generate international consensus guidelines for use of patient-reported outcomes in early phase trials. 

Related links:

• The value of patient-reported outcomes in early-phase clinical trials | PubMed

• Statistical methods and data visualisation of patient-reported outcomes in early phase dose-finding oncology trials: a methodological review | eClinical Medicine

The PRO ethics guidelines provide recommendations for ethical issues that should be addressed in PRO clinical research. Addressing ethical issues of PRO clinical research has the potential to ensure high-quality PRO data while minimizing participant risk, burden, and harm and protecting participant and researcher welfare.

Related links:

• Ethical Considerations for the Inclusion of Patient-Reported Outcomes in Clinical Research | The Jama Network

Despite their benefits, there are concerns that the potential burden on respondents may reduce their willingness to complete Patient Reported Outcomes (PROs), with potential impact on the completeness and quality of the data for decision-making. The consensus statement provides 19 recommendations to address potential PRO-related respondent burden in healthcare research and clinical practice.

Related links:

• Recommendations to address respondent burden associated with patient-reported outcome assessment | Nature Medicine

Global regulators, payers, and policy makers have indicated that PRO data can provide valuable information on the effectiveness, safety and tolerability from the patient perspective. Currently, there are no regulatory, health technology appraisal guidance nor publications pertaining to the standardization of patient reported outcome (PRO) data usage in RW studies.CPROR team contributes to the PROs in Prospective Real World Study Design ISPOR Task Force. The task force’s goal is to develop emerging good practice recommendations for designing prospective real-world studies that collect patient reported outcome (PRO) data.

Related links:

• Patient Reported Outcomes (PROs) in Prospective Real World Study Design

Real-world evidence (RWE) plays an increasingly important role within global regulatory and reimbursement processes. RWE studies are used to assess the real-world long-term effectiveness and safety of health interventions in diverse patient populations. Unlike the highly controlled environment of registration trials, which are usually limited to specialised health care providers, characterised by artificially high patient compliance and close patient monitoring.

RWE generation can be enhanced by the collection and use of PROs. PROs can provide valuable information on the effectiveness, safety and tolerability of health interventions from the patient perspective. However, currently, the collection of PRO data in the real-world setting is restricted. Researchers have limited guidance to support the use of PROs in RWE generation. Available recommendations are fragmented and there is a lack of international guidelines for the collection and utilisation of PROs in this context.

This project aims to characterise the use of PROs in RWE generation and identify challenges and opportunities to implement PROs into RWE studies successfully.

This projects aims at understanding the importance of incorporating PRO instruments in AI health technologies in clinical trials and their use in AI models. Our ongoing work and published articles in The Lancet Digital Health consideres challenges and solution to facilitate the inclusion of PROs in AI health technologies to ensure that the patient perspective is not lost in the rush to advance digital health care. 

Related links:

• The role of patient-reported outcome measures in trials of artificial intelligence health technologies: a systematic evaluation of ClinicalTrials.gov records (1997–2022)

• Embedding patient-reported outcomes at the heart of artificial intelligence health-care technologies