In the Patient Advocacy Track, the importance and challenges around placing the patient at the centre of clinical trials
In my experience, patients are aware of the importance of being involved in clinical trials and they are highly motivated, both for their own benefit and the future benefit of others. Ideally, patients are involved in the design of a clinical trial in which they can provide meaningful input to ensure that the endpoints are relevant and important to them; this may vary between patients according to tumour type, age and other factors. For example, when we asked patients with sarcoma and advanced/metastatic disease about their priorities prior to starting palliative chemotherapy, we found that younger patients all prioritised length of life, whereas two-thirds of elderly patients prioritised quality of life (QoL), or found QoL as important as length of life (ESMO Open. 2021;6:100258).
Data from elderly patients treated with palliative chemotherapy for metastatic soft tissue sarcoma are scarce. We demonstrated previously that in a retrospective analysis of 2810 adults with advanced/metastatic soft tissue sarcoma from 12 European Organisation for Research and Treatment of Cancer (EORTC) trials only 12% were older than 65 years and 1.1% older than 75 years, while almost half of patients diagnosed with soft tissue sarcoma are over the age of 65 years (Oncologist. 2018;23:1250–1259). Hence, this study had a clear underrepresentation of a clinically and biologically very heterogenous group of patients. We therefore designed within EORTC, with important input from patients, the phase III Tolerance study in this age group (≥65 years) in which we compare standard 3 weekly chemotherapy with metronomic treatment, with health-related QoL (difference in physical and role functioning at 12 weeks) as the primary endpoint (NCT04780464). Patients can thus advise on much more than the patient information leaflet alone, and their input is welcomed for study design, for example when discussing the tolerability of the intervention and practicalities around study participation.
In general, several challenges need to be addressed and may prevent patients from participating in clinical trials. First, patients need to know whether the aim of a clinical trial fits with what they expect it to be and what will be involved. Other important factors include comorbidities, logistics and communication. Differences between funding sources and healthcare systems may further affect participation, as a pharmaceutical company-led trial usually pays transport costs, whereas funding can be limited for investigator-led trials. In the latter case, patients may receive funding from their healthcare provider, but in countries where costs may be borne by the patient, these may be prohibitive. Geography is another important issue, especially for patients with rare conditions who may have to travel long distances to a trial centre. Elderly patients often rely on family members or caregivers for transport, and support for the caregiver’s employer – for those of working age – is also important. People living in socially deprived areas and those with lower levels of education also tend to be missed from clinical trials.
Communication is another challenge. Patients may vary in their understanding of medical terminology and care is needed to ensure that processes and procedures are fully explained and understood. Language barriers and cultural differences also need to be considered. As well as affecting participation, communication and logistical issues can affect compliance and, ultimately, the outcome of the trial. Some patients may need to receive treatment for several years, during which circumstances and social support could change and affect compliance in the long-term. For this, we need to consider factors that may prevent patients from remaining in a study, as well as those that prevent their initial recruitment.
Less stringent inclusion criteria could improve patient participation in trials and help rectify the current situation where trials tend to be overly homogeneous, which can limit the generalisability of the data. With an aging population, we need more elderly patients to participate in clinical trials, but many enrolment criteria exclude patients with comorbidities, so this needs further attention. An example of this will be featured in my presentation, ‘The importance of joining clinical research to improve cancer research outcomes’ at ESMO Congress 2022. We also need to improve on informing patients of the results of clinical trials in a meaningful and understandable way. Lay summaries could help patients understand the outcomes and side-effect profiles presented from clinical trials and put the data into context.