Establishing international standards in the analysis of patient reported outcomes and health-related quality of life data in cancer clinical trials

Project Information

SISAQOL-IMI

Grant agreement ID: 945052

DOI

10.3030/945052

Project closed

EC signature date 9 November 2020

Start date 1 January 2021

End date 31 December 2025

Funded under

• SOCIETAL CHALLENGES - Health, demographic change and well-being

Total cost

€ 5 944 754,75

EU contribution

€ 2 281 840,00

Coordinated by

EUROPEAN ORGANISATION FOR RESEARCH AND TREATMENT OF CANCER AISBL
Belgium

Periodic Reporting for period 4 - SISAQOL-IMI (Establishing international standards in the analysis of patient reported outcomes and health-related quality of life data in cancer clinical trials)

Reporting period: 2024-01-01 to 2024-12-31

Summary of the context and overall objectives of the project

Measuring and quantifying how a patient feels or functions during treatment is an important endpoint in cancer clinical trials. It is generally accepted that the collection of PRO data in cancer clinical trials allows the inclusion of the patient’s voice in the risk-benefit assessment of therapies. However, no standards exist on how to analyse, interpret or report health-related quality of life (HRQOL) and other patient-reported outcomes (PROs). SISAQOL-IMI’s goal is to pursue efforts in addressing the urgent need for standardization, by setting clear and robust standards that are tailored to and endorsed by all relevant stakeholders. With a strong international and multi-stakeholder Consortium, the initiative aims at finding consensus on suitable methods to analyse valid PRO objectives in cancer randomized clinical trials (RCTs) and ways to communicate these PRO findings in a standardized way that is understandable to all.

To achieve this aim, SISAQOL-IMI will identify valid PRO research objectives and match these with appropriate statistical methods for PRO analysis in cancer RCTs. Translation to the estimands framework will be provided. Furthermore, the possibility of extending these recommendations to single-arm trial designs will be explored. Recommendations on clinically meaningful change for PRO instruments, as well as design considerations and ways for assessing quality of collected PRO data will be developed, and tools and templates for presentation and visualization of PRO findings freely made available. Strong emphasis is put on continuous collaboration with patient advocacy representatives throughout the project.

Increased interpretability, adoption and full use of PRO outcomes for all stakeholders is expected by providing consensus-based and validated recommendations and communication tools for PRO data, ultimately resulting in better communication and shared decision making, improved outcomes, treatment satisfaction and care.

Work performed from the beginning of the project to the end of the period covered by the report and main results achieved so far

The key project output of the SISAQOL-IMI Consortium include over 140 recommendation statements on the design, analysis, visualization and interpretation of patient-reported outcomes and quality of life endpoints. The recommendations are accessible here: https://www.sisaqol-imi.org/2025/02/05/sisaqol-imi-launches-final-recommendations/

Other outputs are aimed at supporting the implementation and dissemination of these recommendations include:
-Journal publication for the release of the recommendations with highlights and link to the SISAQOL-IMI recommendations and supporting tools
-Interactive table
-Patient materials including patient checklist
-Glossary (with scientific and plain text versions)
-Guidebook
-Graphical templates

In 2024, the Consortium finalized all recommendations, and the supporting tools are also nearly final. The journal paper titled “SISAQOL-IMI consensus-based guidelines to design, analyse, interpret and present patient-reported outcomes in cancer clinical trials” has been drafted and approved by the SISAQOL-IMI publication review committee and is planned to be submitted to the Lancet Oncology within February 2025. The guidebook, graphical templates, interactive table and glossary are finalized in terms of content. The software for the interactive tool is being tested and will be finalized before submission of the journal paper.

The SISAQOL-IMI website was updated in 2024 to reflect the updates and to highlight the project outputs. The updated website is live, but supporting tools will be available only after the publication of the journal paper which is expected in the first or second quarter of 2025.

To disseminate and raise interest across different stakeholders, the Consortium organized a public launch event on 4 February 2025 to present highlights of the recommendations and to discuss how different stakeholders can implement them in their work. In this context, a panel discussion was organized with the involvement of representatives from regulatory and HTA agencies, industry, patient organizations, academic clinical trialists, and clinicians.

More than 130 people joined the SISAQOL-IMI public launch event from various organizations majority of which were from pharmaceutical industry. The executive summary and more information on the meeting can be found here: https://www.sisaqol-imi.org/2025/02/05/sisaqol-imi-launches-final-recommendations/

Progress beyond the state of the art and expected potential impact (including the socio-economic impact and the wider societal implications of the project so far)

NA

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