The SISAQOL-IMI consortium will establish guidance on how to use patient-reported outcomes in cancer clinical trials so that they can be used in a methodologically sound way, analysed in a statistically adequate manner, and intelligibly presented to ensure a high study quality and a better comparability of results across clinical trials.

SISAQOL-IMI Work package 4

Communication tools for PRO
findings from cancer clinical trials

WP academic lead: Bernhard Holzner, Medical University of Innsbruck
WP pharma lead: Jane Chang, Pfizer

Project coordination: Lisa Wintner,

Further staff: Franziska Gross

SISAQOL-IMI Work package 6

Develop recommendations for the terminology and definitions of clinically meaningful change in cancer clinical trials

WP academic lead: Johannes Giesinger, Medical University of Innsbruck
WP pharma lead: Carla Mamolo, Pfizer

Project coordination: Micha Pilz

Summary

In drug development, it is important to find out how treatments affect how patients feel and function in their daily lives. This information is also essential when weighing up the benefits and risks of a medicine. In practice, it is rather difficult to obtain this information and communicate it clearly and simply.

The international, multidisciplinary SISAQOL-IMI consortium brings together 41 organizations, researchers and experts from academic institutions, medical facilities, non-profit associations, patient advocacy groups, regulatory authorities, small and medium-sized enterprises and the pharmaceutical industry. The goal is to establish a common understanding of scientific methods in the use of PRO instruments and data in oncology research. In the future, the newly developed standards will provide guidance on how to optimally define, collect and analyze PRO study endpoints and how to graphically present the results.

To do this, the project will seek to achieve consensus internationally and across stakeholder groups on the optimal use of PROs in cancer clinical trials, and gain clarity on the research objectives for the use of PROs in trials, including the definition of ‘clinically meaningful change’.

The project also aims to improve the statistical analysis of PROs in cancer clinical trials, and standardise the way findings are reported, presented and visualised. The standards should be endorsed by all relevant stakeholders.

Furthermore, the project plans to develop educational tools based on the standards; these tools will hopefully help to improve patients’ understanding and empower shared decision making.

Ultimately, the tools and resources developed by SISAQOL-IMI should ensure that cancer clinical trials accurately capture how patients feel or function during treatment. This in turn will aid in decision making for regulators, health technology assessment bodies, and, crucially, improve patient satisfaction. The findings may also be applicable to clinical trials in other disease areas.