Senior Scientific Advisor, Head of Health Outcomes
Research suggests that 20% of oncology trials do not recruit enough participants.1 Data also suggests that only three percent of patients with cancer participate in clinical trials.2 Considering the stats, it’s clear that there is significant work to be done to improve accessibility to clinical trials for cancer patients.
Over recent years there has been growing interest in how eCOA (electronic Clinical Outcome Assessments) solutions can be utilized within oncology clinical research to counteract many of the challenges faced by researchers in this area. There is encouraging evidence from the primary care setting about the benefit of deploying eCOA within this environment. According to The American Society of Clinical Oncology (ASCO) “in multiple prospective studies, integrating electronic Patient Reported Outcomes (ePROs) into routine cancer care has improved patient-clinician communication, patient satisfaction, symptom management and control, and quality of life. Nurses find symptom email alerts to be useful and actionable. Recent data have found reductions in emergency room visits, longer tolerability of chemotherapy, and improved survival. This evidence of benefits has led to growing interest in this approach.”3
Based on these encouraging observations, it is anticipated that eCOA could also help research professionals to overcome everyday data collection challenges in oncology clinical trials. Indeed, in clinical trials there is a growing expectation to understand the important components of disease and treatment impact from a patient perspective, and to ensure there are robust means to measure these.
The FDAs ongoing development of guidance relating to patient-focused drug development underline this. In the area of oncology, while tumor size and progression data remain vital to understand treatment effects, patient-reported outcomes are seen to be increasingly important to understand treatment effects and toxicity in a number of key areas including symptomatic adverse events, physical function and disease-related symptoms.3
The Challenges of Collecting Patient-Reported Outcome Measures (PROMs) in Oncology Clinical Trials
A patient-reported outcome (PRO) is an observation or symptom (e.g. pain or nausea) reported by the patient with no amendment or additional interpretation by anyone else, for example a clinician.
PROs can also assess the patient perspective on factors observable by others, such as quality of life, time to pain progression and time to worsening of physical symptoms. Traditionally, questionnaires collecting such data known as Patient Reported Outcome Measures (PROMs) have been collected via pen and paper, however, busy site teams often find this mode of collection cumbersome and antiquated and they are always looking for new solutions to support the challenges that clinical trials impose, to improve the quality of data collected.
Clinical trials in oncology are admin-heavy, they require site staff to manage busy visit schedules associated with complex protocols, and they demand finely tuned tracking of patients, symptoms and concomitant medications. These typical site challenges combined with traditional PROM data collection methods cause a huge burden for sites and can lead to poor data collection.
In addition to the site challenges, oncology patients face symptoms such as pain, fatigue, nausea, and dealing with other symptoms of the toxicity of treatment (e.g. chemotherapy) which can add further challenges to the management and running of oncology clinical trials. These symptoms can result in patients finding it difficult to report back PRO data and may even preclude patients from attending certain site visits. This can result in high degrees of missing data which can undermine the ability of clinical researchers to provide robust interpretation of the PRO data collected.
Improving Oncology Clinical Trials with Electronic Clinical Outcome Assessments (eCOA)
Regulators strongly favor including the patient perspective in oncology studies,3 however, with this interest in PRO data comes great scrutiny and the need for data that adheres to the highest of compliance standards. eCOA solutions support this by ensuring data integrity and quality and reducing the burden of data collection in oncology trials.
There are several ways that eCOA solutions improve data quality during oncology trials for patients, site staff, sponsors, and regulators. Using a digital technology such as handheld devices, tablets, and the web, makes reporting data and tracking the effects of a therapy easier than ever before.
Site Benefits
- Reduce the administrative burden of managing patients.
- Easily schedule patient visits.
- Automatically send reminders to alert patients about upcoming visits.
- Effortlessly monitor patient PRO compliance.
- Track patient pain, symptoms, and questionnaires all from one system.
- Present protocol-defined questionnaires in the correct order.
Reporting Benefits - Adverse Events and Concomitant Medication Data
PROMs enabling the collection of adverse events can provide important insights into treatment tolerability, especially during treatment recovery phases at home. The PRO-CTCAE, for example, is a patient reported outcome instrument developed to evaluate symptomatic toxicity in patients on cancer clinical trials,5 and can be incorporated into the eCOA solution to enhance the monitoring and reporting of adverse events. Researchers select items to include based on the known profile of the test drug and any comparator treatments or use a standard list where little information is known a priori. eCOA solutions also support effective and accurate capture of concomitant medication usage by offering a predefined, personalized list of medications to choose from, as well as the ability to nickname medications for easier tracking by the patient.
Post-Progression Studies Benefits
With regulators driving sponsors to include post-progression/follow-up periods in their oncology trials, eCOA solutions can help reduce the burden on patients by remotely collecting data as site visits become less frequent, providing the option to complete questionnaires at home.
Patient and Caregiver Benefits
Patient-centric focus is one of the biggest benefits eCOA solutions provide. Patients who become too sick to complete the questionnaire at site visits can continue to report COA data because eCOA supports:
- Rescheduling PROM timings and site visits to provide flexibility for patients who may not be feeling well.
- Re-activating incomplete or interrupted visits so that patients can pick up where they left off.
- Adding unscheduled visits for patients who may need additional time at the site due to a change in their diagnosis.
- Extending data entry windows to accommodate the patient.
- Implementing a caregiver role to maintain data collection without adding burden to the patient.
Cancer patients that feel too unwell to complete PROMs at certain times or throughout the study may still provide this important data by electing to enable caregiver-completion on their eCOA device. This allows a caregiver, such as a family member, to input data on behalf of the patient.
Conclusion
Clinical oncology research is unique when compared to other therapeutic areas. So many types of cancers exist and therefore trials can vary greatly, especially in terms of scope, protocol, and methodology. Each individual trial and patient are different and therefore study teams need to make appropriate modifications during study implementation to ensure that patient centricity is the focus for optimal data collection. Sites and patients are the epicenter of clinical oncology trials. If something is amiss, they’ll be the first to feel the impact. Sites have so many administrative tasks that it’s sometimes difficult for them to manage everything and keep focus on the patient. Patients experience a range of challenges due to the nature of their condition, including a lack of tools and resources that would make contributing to clinical trials easier for them. By incorporating solutions to collect PRO and COA data electronically, many of these challenges can be addressed, simplifying and reducing the burden of data collection during oncology clinical trials.
References
- Bennette, C. S., Ramsey, S. D., McDermott, C. L., Carlson, J. J., Basu, A., & Veenstra, D. L. (2016). Predicting low accrual in the National Cancer Institute’s cooperative group clinical trials. JNCI: Journal of the National Cancer Institute, 108(2).
- English, R., Lebovitz, Y., & Giffin, R. B. (2010). Transforming clinical research in the United States: challenges and opportunities. In Workshop Summary.
- Kleutz PG, Slagle A, Papadopoulus EJ et al. (2016). Focusing on Core Patient-Reported Outcomes in Cancer Clinical Trials: Symptomatic Adverse Events, Physical Function, and Disease-Related Symptoms. Clin Cancer Res; 22(7): 1553-1558.
- Retzer, A., Kyte, D., Calman, L., Glaser, A., Stephens, R., & Calvert, M. (2018). The importance of patient-reported outcomes in cancer studies. Expert Review of Quality of Life in Cancer Care, 3(2-3), 65-71.
- Department of Health and Human Services FDA Center for Drug Evaluation and Research (2009). Guidance for industry: patient-reported outcome measures: use in medical product development to support labeling claims. https://www.fda.gov/downloads/drugs/guidances/ucm193282.pdf
- Basch, E., Campbell, A., Globe, D., Kluetz, P., Kozak, M., Minasian, L., ... & Spears, P. (2015). Capturing Symptomatic Adverse Events from the Patients’ Perspective: The Potential Role of the National Cancer Institute’s PRO-CTCAE Measurement System. Cancer Research.
Author Biography
Brad Sanderson is a Senior Scientific Advisor, Head of Health Outcomes at CRF Bracket. He is based in London, United Kingdom. Within this role he leads the Health Outcomes discipline and seeks to advance the company’s scientific and technical expertise in eCOA. Brad has over 15 years of industry experience in research across pharma, bio-tech and digital health.