With the race for patients becoming increasingly competitive, building a clinical trial with a patient-centric approach can make all the difference. Relieving patient burden in clinical trials has been recognized as critical to effective trial planning and execution, since being defined more than 40 years ago.1,2 Integrating components of patient burden, such as pain and anxiety, into a quantitative measure can help save time and reduce costs associated with patient recruitment, retainment, and adherence issues.
A patient-centric approach to trial design
As the science associated with the clinical development effort evolves, so does the inclination of clinical scientists to expand the number of objectives, endpoints, and associated procedures. There are tremendous pressures for a trial to collect information that will lead to increased scientific understanding, especially in trials that employ novel therapeutic approaches.
While this goal is understandable, it can override consideration of the minimum requirements necessary to meet essential clinical and statistical outcomes. Recent research shows that "for a wide variety of reasons, research sponsors routinely add procedures because their marginal cost is a small fraction of the study budget."3
Unfortunately, when clinical activities are excessively burdensome for patients, it can hinder patient recruitment, retention, or adherence, leading to delay, or even worse—failure.4 Sometimes exceeding 30%, patient dropout rates can lengthen timelines, add costs, introduce risk to the interpretation and validity of study data, and delay or even derail trial completion and product approval.5
To minimize the potential negative impact of a trial on patient volunteers, clinical trial managers must create and incorporate a multi-faceted combination of patient insights. In addition, a lean study design that achieves the essential clinical and statistical outcomes, with a focus on minimizing site and patient burden, can lead to better, more positive patient experiences and better trial outcomes.
Data-driven decisions, improved trial outcomes
In today's trials, patient burden is typically assessed qualitatively. Yet quantifying a patient's experience can lead to greater insights. While protocols outline the number and types of testing procedures a patient undergoes during a clinical trial, it does not accurately reflect total patient burden. For instance, specific elements of patient hardship, including pain, anxiety, and harmful exposure, are generally not considered or incorporated into a clinical trial's protocol analysis.
To address this need, researchers have turned toward the development of quantitative solutions that can help improve patient-centricity. An example is Medidata's Patient Burden Index (PBI), which provides a quantitative framework of patient burden on a per-procedure basis. Based on the number of times each procedure is experienced by the patient over the course of the trial, the overall PBI can be quantified at the protocol level and benchmarked against studies of similar disease area and phase.
The patient and site burden of a trial can now be assessed quantitatively by evaluating the PBI and site burden scores at the study level. Combining the disparate components of patient burden into an overall quantitative PBI will help study designers and protocol managers achieve a better understanding of the patient’s overall experience participating in a clinical trial. Clinical operations professionals can now see a data-driven visualization of the patient experience and improve study operations by providing insights into protocol optimizations.
Finding, recruiting and retaining patients is only expected to become more competitive within the current clinical research landscape. By obtaining highly informative data and embracing data-driven insights, clinical trial sponsors and CROs are realizing the positive impact that a quantitative decision-making approach can have on better understanding patient burden and ultimately, improved trial planning and execution.
1 Sloan J. Asking the Obvious Questions Regarding Patient Burden, Journal of Clinical Oncology. 2002;20(1):4-6.
2 Bradburn NM, 1977, Respondent Burden, Health Survey Research Methods: Second biennial conference.
3 Getz K & Campo R. Drug Development Study Designs Have Reached The Danger Zone, Expert Rev Clin Pharmacol. 2013;6(6): 589–591.
4 Burgess LJ & Sulzer NU. Patient Retention Gifts In Clinical Trials—Undue Inducement Or Justified Motivational Tools? South African Medical Journal. 2011;101(9):640-641.
5 National Academies of Sciences, Engineering, and Medicine. Recruitment and Retention Issues: Patient, Provider, Institutional, and System Barriers. Strategies for Ensuring Diversity, Inclusion, and Meaningful Participation in Clinical Trials: Proceedings of a Workshop. Washington, DC: The National Academies Press. 2016. doi: 10.17226/23530