PIONEER

Out of all the 2.7 million new cases of cancer diagnosed per year in the European Union, 12.1% are prostate cancer, making it the fourth most common cancer in the world.

Although prostate cancer is highly prevalent, we still know relatively little about who is most likely to develop it and which risk factors play the biggest role. PIONEER leveraged real-world data to stratify the patient population and to identify some shared characteristics of prostate cancer patients. This will speed up the diagnosis of prostate cancer and improve care for patients.

Every day around the world, data on prostate cancer patients is collected from registries, electronic health records, epidemiological cohorts and clinical trials, but it is stored in inconsistent formats. PIONEER has harmonised data on 3.5 million prostate cancer patients and it is available for use in the PIONEER platform. By applying big data analytics and a common data model framework, PIONEER transformed these heterogeneous real-world data into high-quality real-world evidence that  researchers can trust and use reliably.

PIONEER experimented with innovative ways of working, organising three international study-a-thons. The first was held virtually during the COVID-19 pandemic, followed by two successful hybrid events.  Each study-a-thon brought together clinicians, researchers, data scientists, patients and other stakeholders to tackle various research questions across 5 days. Several peer-reviewed publications arose as a result of these events.

Through interviews and other forms of engagement with patients, the researchers discovered that a cure wasn’t necessarily the only outcome that interested men with prostate cancer. Many wanted to be able to avoid treatment-related side effects, especially since prostate cancer doesn’t always present with symptoms and  curative therapies sometimes pose greater risk than the disease itself. To address this, PIONEER analysed data from over 500 000 patients to pinpoint when active intervention is warranted and when ‘conservative management’– monitoring the disease without immediate treatment – is preferable.

PIONEER used big data to sift through the existing information available about prostate cancer patients, their characteristics and their responses to treatment. With this, they were able to identify several factors that could indicate a person’s suitability either for treatment or for taking the conservative approach.

PIONEER delivered a clear picture of the profile of the patient who is likely to benefit from prostate cancer treatment, taking into account age, hypertension, obesity and type 2 diabetes, amongst other factors. They found that the typical profile of a patient in the real world differed substantially from those enrolled in clinical trials. For instance, the typical patient that could benefit from treatment is over the age of 68 and has several comorbidities, especially heart issues and type 2 diabetes. In contrast, most clinical trials for prostate cancer treatments enroll patients younger than 65 with few serious health issues.

The PIONEER study showed that strict exclusion criteria in randomised clinical trials limited the applicability of their findings.  Clinicians should account for these limitations when applying trial results to patient care. The study further recommends that future randomised clinical trials adopt broader enrollment criteria to better serve the most needy patients.

To ensure that interventions and treatments are effective and safe, researchers need to look at the effects they have on patients. Researchers do this by measuring ‘outcomes’, which are variables measured during clinical trials that demonstrate whether an intervention or treatment was effective.

For example, in a study of how well a prostate cancer treatment works outcomes might include: overall survival; progression free survival; sexual, bowel and urinary function. A core outcome set is an agreed standardised group of outcomes that researchers should measure and report if they are conducting a clinical trial in a particular disease area.

PIONEER developed Core Outcome Sets that can be used to measure outcomes in prostate cancer patients. The Core Outcome Sets are the result of a methodological rigorous systematic review and consensus finding process with a diverse range of prostate cancer stakeholders including clinicians, nurses, researchers, industry partners and most importantly patients, and they have been endorsed by the European Association of Urology, Europe’s leading medical authority on urological practice, research and education.

While measuring core outcomes is important, it is also critical to evaluate the impact of side effects and health-related quality of life on patients with prostate cancer. It is especially important because prostate cancer patients may have to choose between health-related quality of life or length of life.

PIONEER systematically reviewed both clinician reported outcomes and Patient Reported Outcome Measures (PROMs) used in localised, locally advanced, and metastatic prostate cancer. Based on the results of the reviews and subsequent consensus processes, PIONEER recommended minimum sets of clinician and patient reported outcomes, provided definitions or each, and recommended PROMs, for use in routine practice and research settings.

PIONEER also developed an online search tool to help clinicians choose which biomarkers they want to use to determine a course of treatment for a patient with prostate cancer.