Finding the Patient Voice in Clinical Trials
The gold standard for evaluating risks and benefits of pharmacologic and non-pharmacologic therapies in an unbiased manner is a randomized, controlled clinical trial. However, despite the advancement of clinical trials, many lack one essential component to success: Patient voices. How can pharmaceutical companies claim to truly understand the risks and benefits of their therapies without fully engaging with the targeted patient base?
A survey conducted by the Clinical Trials Transformation Initiative and the Drug Information Association found that only 45% of 119 predominantly large pharmaceutical companies engage with patient groups. More surprising, 41% said they had no plans to engage with patient groups in the future. A thorough understanding of disease burden, risks, benefits, and therapeutic outcomes from the patients’ perspective should serve as a foundation for the development of clinical trials. However, the patient voice is often added after trial design has been completed; or in some cases, not at all. This begs the question: Why are patients’ voices not being heard sooner? Is there an opportunity to engage patients before and after clinical trials to understand their drivers and barriers for participation, desired outcomes and insights into the patient perspective on clinical trials?
A wide variety of barriers exist that prevent the inclusion of patient ideas and opinions. Barriers specific to trial design include enrollment criteria, poor site selection, improper planning of recruitment processes, and suboptimal protocol design. Patient-specific barriers include lack of education and awareness of disease states, lack of financial compensation, dropouts, and patient-perceived benefits of participation. These factors point to barriers on both sides of the trial.
Another barrier lies in clinical trial design. The development of a clinical trial is initiated within the pharmaceutical company through clinical research and the development of a methodology, which consists of enrollment criteria, recruitment processes, and site selection. If proper research has not been conducted, the enrollment criteria may exclude those patients most affected by the studied disease states, therefore excluding those who could provide valuable insight. Many times, the sites selected for study can lead to the recruitment of only a specific and limited patient demographic. Additionally, sites selected may lack the resources for adequate and unbiased recruitment.
A greater problem is the fact that patients often do not perceive a benefit to participating in clinical trials. Treatment outcomes are usually measured by negative means, such as progression of disease or death. Rarely, positive patient reported outcomes, such as improvement in quality of life after treatment, are reported. Many times, patients are not fully knowledgeable about their disease and may not understand the outcomes being investigated by the study. Lack of compensation for their absences from work, transportation expenses, and other related expenses also lead to patient reluctance.
Pharmaceutical companies should make it a priority to develop patient-centered clinical trials by establishing more patient partnerships, hiring more culturally-competent staff, and investing in patient services. Clinical trial design needs to allow for the inclusion of patients during the clinical development phase, rather than the commercialization phase. The development of patient engagement platforms, such as Novartis’ Blood Pressure Success Zone, will provide an understanding of the patients’ perspective and insight into methods to achieve optimal health outcomes. Rather than focusing on negative measures of success that don’t tap into the needs and opinions of patients, these trials should be more focused on understanding patients and reaching them in a more positive way. Only then will clinical trials become truly unbiased by reaching a broader population.