Recording patient’s perspectives on their own health and quality of life while taking treatments for cancer is clearly important. Cancer treatments can be gruelling, and the impact on daily life is often significant.
But up until now, patient-reported outcome measures – PROMs – have been used in cancer clinical trials in a very inconsistent way, making it difficult to compare PROMs from one study to another. Researchers all over the world have used various ways to assess quality of life, and this has led to confusing and conflicting results. As a result, studies involving PROMs have not always been taken seriously, and it has been difficult to obtain meaningful insights into how a patient’s quality of life might be affected by a new cancer drug.
“When I first joined SISAQOL and I was reading through a review of a huge number of single arm trials (60 plus), I was totally surprised at the complete laissez-faire attitude which seemed to prevail throughout, with companies and researchers choosing what to test, what to report and what to ignore; including aspects of treatment or PRO results or side-effects I thought vital to patients. It was a real eye opener for me,” says David Ness, a patient who took part in SISAQOL. “Quality of life issues and overcoming side effects are vital aspects of daily life [for cancer patients].”
SISAQOL focused on how to define well-defined endpoints for PROMs, how to ensure that high-quality and targeted measures for quality of life data were available to aid decision-making by various stakeholders ,and how to push researchers to evaluate quality of life according to meaningful criteria.
“When it comes to endpoints in clinical research, we’re more willing to accept endpointss like overall survival, progression-free survival and tumour response because they can be easily measured,” says Madeline Pe, Head of the Quality of Life Department at the European Organisation for Research and Treatment of Cancer (EORTC) as well as project coordinator and academic co-lead of SISAQOL.
“But something like the quality of life of patients, researchers tend to say it’s so subjective, how can you really measure it? The idea was to make these endpoints as relevant as the other clinical endpoints.”
Figuring out what was going on
In the beginning, a large part of SISAQOL’s work involved sifting through scientific literature and finding all the different ways that cancer researchers were evaluating patient-reported outcome measures. Often, they found commonalities which could form part of an established standard. Where they found differences, they tried to identify why those differences were happening and what was the reasoning behind the differing approaches. In some cases, the project found that researchers were not using the right endpoint to answer the question that they were asking.
Patient-reported outcome measures come with their own unique complications which makes them different from other kinds of clinical endpoints. For instance, collecting PROMs often involves patients filling out questionnaires, but the questionnaires are not always completed, resulting in missing data. There were other hurdles too, like the fact that many studies considered "we will measure quality of life" as an appropriate way to define an endpoint, which it is not.
Tools for researchers that will yield better insights into quality of life
SISAQOL developed an interactive table designed to make researchers running clinical trials reflect more on the research questions that they are asking to understand exactly which endpoints related to quality of life they should be considering.
The researcher inputs information relating to the question that they want to ask and the interactive table indicates what’s needed in terms of statistical analysis.
“When the statisticians analyse the data, the conclusion they come to actually responds to the original research question,” says Pe. “It guides researchers to be more specific in regards to what it is about the patient's quality of life you want to know because of the initiation of treatment.”
The interactive table was developed with input from all of SISAQOL’s partners, including patient representatives, cancer researchers, industry partners, research organisations, regulators and health technology assessment (HTA) bodies.
Empowering patient advocates using checklists
Patient advocates are increasingly called on to check clinical trial protocols to ensure that they are addressing the needs of patients. However, there can be a knowledge gap – sometimes advocates don’t necessarily know how to ensure that clinical trials are addressing quality of life concerns properly. SISAQOL has developed a checklist to help with that.
“Patient advocates are now part of reviewing protocols,” says Pe. “It was important for us that they understood that the quality of life endpoints are meaningful and they know how to look at them. So when they advise, they know what they're looking for.”
In addition to the checklist for clinical trial protocols, SISAQOL also developed checklists for visualisations, both scientific and plain-language.
Scientific visualisations are used in peer-reviewed papers and reports that are written up after trials take place, containing the findings. Where they cover quality of life aspects, SISAQOL has written a set of recommendations on how they should be presented.
The visualisations should be transparent and easy to interpret for researchers and experts in the field, and the checklist allows patients and healthcare professionals to evaluate whether the scientific visualisation reaches those goals. Likewise, the plain-language visualisation checklist allows patients and healthcare professionals to evaluate whether a visualisation depicting quality of life results that is targeting a general audience is fit-for-purpose.
“Visualisations are a standard way to report results. But they have to be interpreted, so it’s really important to make sure that you have the full picture. It needs to be reported in a specific way because you can misrepresent it,” says Ahu Alanya, Team Leader at the Quality of Life department of EORTC and project manager of SISAQOL.
SISAQOL also built graphical templates into the interactive table so that researchers have a blueprint to help them to design visualisations that are easily understandable and communicate the findings properly.
The rest of SISAQOL’s toolbox includes a glossary for patients, a guidebook and on a regular basis, SISAQOL will run training programmes. The next session will be September 22-23 2026 in Croatia – more information is available here: https://www.sisaqol.org/sisaqol-training/
SISAQOL’s outputs are relevant for all clinical trials for cancer treatments, and the methodology could be applied in the future to create resources to help standardise and organise quality of life data collection for other fields.
SISAQOL is supported by the Innovative Medicines Initiative, a partnership between the European Union and the European pharmaceutical industry.