Collaborating for consensus: Standardizing the design, analysis and interpretation of patient-reported outcomes in cancer clinical trials

12th April 2021

Patient-reported outcomes, or PROs, are important considerations when new cancer therapies are being investigated, including in benefit and risk assessments. PROs include how patients themselves experience and report their symptoms, as well as other health-related quality of life (HRQOL) issues.

The importance of patient involvement in the development of cancer therapies must not be underestimated if we are to truly transform the lives of people living with cancer.

Over the past few years, we have seen an increase in the collection of PROs in cancer clinical trials. However, to date, a set of robust, international recommendations to standardize the design, analysis, presentation and interpretation of these data has been lacking. Individual researchers and organizations currently have their own standards, which can lead to varying conclusions being drawn from the same trials, and different decisions being made across stakeholders. Ultimately, this lack of consistency could negatively impact patient care and outcomes.


The SISAQOL (Setting International Standards of Patient-Reported Outcomes and Quality of Life Endpoints in Cancer Clinical Trials) Consortium was first convened by the European Organisation for Research and Treatment of Cancer (EORTC) in 2016, to engage a range of international stakeholders to address the issues of inconsistency in using PRO data.

In February this year, the launch of SISAQOL-IMI was announced – a four-year project with the ambition of improving how PROs are used in cancer clinical trials. Co-led by my colleagues and I at Boehringer Ingelheim as the Project Lead, and the EORTC as the Project Coordinator, SISAQOL-IMI will develop a consensus-based set of best practice recommendations for the design, analysis and interpretation of PRO endpoints in cancer clinical trials. This will be done with the view that some of these findings may also be relevant to other therapeutic areas.

The launch of this project signifies the SISAQOL Consortium responding to the Innovative Medicines Initiative’s (IMI) call to broaden stakeholder engagement work, harmonize available recommendations, and update them based on stakeholder needs and recent developments in the methodological literature.

SISAQOL-IMI’s ambition is to create recommendations that:

·        address the interests and needs of a broad range of international stakeholders, based on a consensus among them,

·        are of acceptable and high methodological quality, and

·        are able to be interpreted by stakeholders both with and without statistical backgrounds, including lay people.

Although there have been other large-scale initiatives of this nature in the past, we have rarely seen any other broad, global and multi-stakeholder outreach projects on the same scale as SISAQOL-IMI. This is one of the largest initiatives to date in terms of integrating valuable input from a wide range of relevant decision-makers and – importantly – the cancer patient perspective.

Key stakeholders and how the recommendations will benefit the cancer community

As a public-private collaborative project, SISAQOL-IMI brings together 41 stakeholder groups, including researchers from the pharmaceutical industry, academia, cancer institutes, PRO experts, regulators, non-profit associations, and patient representatives and advocacy organizations.

Buy-in of the final consensus recommendations by all stakeholders will be critical to the success of this project and to ensure this happens, we are involving them throughout the process.

To begin with, stakeholders’ needs are being assessed, before their role turns to being advisors who shape the recommendations and methods of standardization. A crucial part of SISAQOL-IMI will be the consensus meetings, which will be held at key milestones over the four years of the project to allow partners to discuss recommendations, resolve areas of disagreement, and agree on next steps. Each milestone will result in a consensus report, which when collated will form the basis of the standards.

The final recommendations will benefit the cancer community by improving their understanding of the role of PRO measures in academic research, drug development, and drug approval by regulators and Health Technology Assessment (HTA) bodies. It will also guide the community in using PROs in a methodologically robust manner, analyzing them in a statistically adequate way, and presenting findings intelligibly. This will ensure a high study quality and better comparability of results across trials.

A project designed with patients in mind

Patients bring a deep understanding of their experience with cancer and its treatments, and this allows for more impactful delivery of patient-relevant outcomes. Therefore, patients’ insight on the research objectives, study design and interpretation of PRO findings must be highly valued.

In the SISAQOL-IMI project, patient representatives and clinicians will be involved throughout the development of the recommendations, including contributing to a layperson version for patients and the wider public. This will ensure that the outputs from SISAQOL-IMI are understandable and meaningful to stakeholders with a non-statistical background.

In the long run, we would like to see these standards provide a more robust and reliable basis for joint decision-making between patients and physicians, which will undoubtedly lead to improved outcomes and treatment satisfaction.

What next?

SISAQOL-IMI is a strong example of how researchers from academia and industry can truly partner with regulators and patient organizations to develop useful recommendations for cancer clinical trials, and in doing so, address an unmet need.

The initial aim is to map and understand stakeholders’ needs in terms of standardizing the use of PRO data. The end goal, four years from now, is to see conclusions based on PRO data from cancer clinical trials being reliable, robust, interpretable, clinically meaningful, replicable and trustworthy. SISAQOL-IMI will raise the standards and optimize the use of these valuable data, ensuring that cancer patients’ voices are accurately represented and efficiently communicated in the benefit and risk assessments of cancer therapies.

Launching the project is just the first step. There is a lot of work ahead of us, but we will collaborate for consensus, with the benefits this will provide to cancer patients at the core of our efforts.

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