Researchers advocate involving patients as active, engaged stakeholders in the design and implementation of clinical trials.

By: Joseph Vandigo and Malissa Carroll
Monday, May 12, 2014

Baltimore, MD, USA – In a new study published in Value in Health, the journal of the International Society for Pharmacoeconomics and Outcomes Research (ISPOR), researchers from the University of Maryland School of Pharmacy and PatientsLikeMe – a patient network that allows people to share and learn from real-world, outcome-based health data – suggest patient participation in clinical trials could be improved if trials were designed to be more patient-centered.

“Many people are reluctant to participate in clinical trials,” says C. Daniel Mullins, PhD, professor and interim chair of the Department of Pharmaceutical Health Services Research (PHSR) at the School of Pharmacy and lead author of the study. “This reluctance can stem from the perceived risks that people associate with participating in these trials, in addition to individuals not seeing any clear benefit to their participation. Oftentimes, the only exception is when a person has a condition for which there is no effective treatment. In this instance, he or she may feel that there is no other option but to participate in a clinical trial.”

The study, titled “Patient-Centeredness in the Design of Clinical Trials”, argues that, while individuals might initially feel comfortable with their participation in a clinical trial, they often become unsure of their involvement as the trial progresses. This hesitation can result from a number of factors, including participants’ mistrust of medical research, perceived risk of harm, or inconvenience associated with the trial’s protocol.

“Randomized controlled trials have traditionally been designed with the science in mind,” says Paul Wicks, PhD, vice president of innovation at PatientsLikeMe and co-author of the study. “But, as patients become increasingly empowered through digital information, and engaged in managing their own condition, the traditional trial models need to evolve, too.”

Mullins and Wicks recently collaborated with other partners to launch the Patient-Centered Involvement in Evaluating the Effectiveness of Treatments (PATIENTS) program at the School of Pharmacy, which aims to reduce health disparities by leveraging relationships with patient communities and health care systems to ensure that patients, health care providers, and other partners are actively engaged in research. Both researchers are advocates for patient-centered outcomes research (PCOR), which looks at the benefits and harms of a wide range of treatment options to help patients, their families, and health care providers make better decisions about their care.

Although traditional trials typically recruit patients from a specialist environment following a rigorous screening process, Mullins and Wicks suggest three approaches -- pragmatic, adaptive, and Bayesian trial designs – that could enhance patient-centeredness within the clinical trial setting if properly implemented.

Unlike traditional trials, pragmatic trials recruit participants from a wide range of clinical settings. They are designed to evaluate the effectiveness of certain treatments in real life situations. Adaptive trials allow researchers to alter certain features of the trial as it progresses as well as evaluate findings in real-time, instead of waiting for the trial to end. The results produced by Bayesian trials are more easily interpreted by patients and their health care providers than results produced by traditional trials.

“The way in which a clinical trial is designed can provide an opportunity to empower patients in the research process,” says Mullins. “By applying different aspects of these trial designs to their research, investigators can improve the quality of care and safety for their participants as well as improve trial recruitment and retention.”

Wicks adds, “Each of these approaches gives patients a higher chance of maximizing their outcomes and avoiding harms, as well as helps integrate trials into every day practice so that research becomes a part of care.”

Though additional research is needed to help determine which trial design works best to actively involve participants in clinical trials, Mullins and Wicks emphasize that trial design is not the only barrier that prevents people from engaging in the research process.

“Making clinical trials more patient-centered extends beyond implementing more pragmatic, Bayesian, and adaptive trials,” says Mullins. “Investigators need to involve informed participants and patient advocates in the study design, promote continuous engagement and feedback among participants, and better address the information needs of diverse populations to truly ensure the trial is patient-centered and improve recruitment and retention.”

Please visit the International Society for Pharmacoeconomics and Outcomes Research (ISPOR) website to view the official press release.