In The News: Patient-Centric Approach in Clinical Trial Design: Involve Patients Early
Published by AJNU Life Sciences Software
Patient-Centric Approach in Clinical Trial Design: Involve Patients Early
Clinical trials function more efficiently and produce better results when patients are engaged in the process. Increasingly, the industry is moving towards a patient-centric approach to clinical research and the design of trials. Reducing patient burden is a big part of this as it helps to boost recruitment and retention rates. But there is also value in engaging patients in the design of the trial itself.
We explore how trial design can include patients’ perspectives and why this is a valuable approach to take.
Design Benefits of Including Patients
There’s an argument that patient and public involvement (PPI) matters because those that pay for or participate in research should have a say as to how it’s conducted. Beyond this more practical reasons abound, says Emmy Racine, Ph.D., a researcher at University College Cork.
PPI may help improve research quality; however, evidence supporting this claim is lacking. Indeed, the evidence shrinks further when it comes to trials of rare diseases, explain Kevin Marsh, Ph.D. and Margaret Vernon, Ph.D. at pharmaceutical product development company Evidera. Still even that limited available evidence is promising: A review of 26 studies of rare diseases showed that when patients and the public were engaged, patient enrollment improved.
Additionally, the researchers say that the benefits outweigh the costs of investing in patient and public engagement processes.
Evidence Versus Anecdote
Perhaps the lack of hard data demonstrating the value of patient engagement isn’t such a hindrance. No one’s ever been persuaded to ride a rollercoaster because of the physics involved has been explained to them, writes neuroscientist and analyst Kristina Staley. People try new experiences based on what those they trust and respect tell them.
Done right, storytelling could be more persuasive — and keep researchers’ expectations in check. After all, patient involvement won’t suddenly simplify trial design, but it will help researchers to think in fresh ways, identify unseen problems and even motivate them.
FDA Says Patient Voice is Essential
Without patient involvement, designs of trials testing medical devices could be of a poorer quality than those that engage patients. Former Acting FDA Commissioner Ned Sharpless, M.D. says patient involvement reduces the burden they bear and helps to enroll participants.
The information from patients about what it is like to live with a disease or require the use of a medical device is vital to successful treatment. It helps researchers determine what matters most to those who will ultimately rely on the devices.
Work With a Patient Adviser
Commenting on the FDA’s call for patient involvement, pharmaceutical and biotech writer Nick Paul Taylor says sponsor companies should work with organizations that train patient advisers. They should look for advisers experienced in clinical trials and disease management.
Patient advisers can help identify solutions to relieve patient burden, such as scheduling site visits on weekends or opting for more digital and remote processes. Taylor notes the FDA recommendation that collaboration between sponsors and patient advisers happen early in the design process of a trial.
Patients Know Best
When it comes to the experience of living with a disease, patients know best. Their involvement should be seen as a boon to researchers. Indeed, it can improve their methods and study designs, writes Angela Wipperman at BMC. But it can also boost recruitment — especially in rare disease trials.
She points to a rare disease study that began in 2014. Researchers doubted whether they could recruit the necessary number of patients given the limited benefits the trial would offer participants. After consulting a patient group, however, they discovered patients were more eager to participate when they learned they could help young people avoid experiencing the same severe symptoms they had lived with.
Start Engagement Long Before Recruitment
The U.S. has launched a bold campaign. The All of Us Research Program from the National Institute of Health aims to enroll one million people in the country to handover their medical data to improve the development of precision medicine.
To get this level of buy-in from the public, engagement is key, write Bobby Clark and Ronnie Tepp, principals in the health innovation team at HCM Strategists. Their organization has been helping the NIH achieve its goal. The pair notes that patient engagement should happen long before recruitment for a trial begins. Doing so will help researchers understand what matters most to patients.
Take the following example. Two years ago, Janssen researchers had patients carry out mock visits to a trial site, says Katherine Capperella, global patient engagement leader at Janssen Pharmaceuticals. Doing so showed researchers they needed to reduce the length of patients’ site visits and provide transportation for improved trial participation. And it worked. A year into the trial, no one had dropped out of the study compared with an average attrition rate of around 18 percent.
Engagement is Desirable But Can Be Difficult
Most in the industry would agree that patient engagement is fundamental to successful trials. At the very least, researchers would see engagement as desirable. Yet few organizations have formal patient engagement processes in place, according to Merck associate director Mary Elmer.
Indeed, an article published in the Therapeutic Innovation & Regulatory Science reports that of the 14 companies surveyed in the study, none had regular processes for engaging patients in trial design and conduct. Elmer says there’s a disconnect between appreciating the need for patient engagement and the practical steps to making that engagement happen.
This disconnect needs to be rectified but it won’t be easy. Elmer notes that patient engagement requires sponsor companies to increase budgets, engage in institutional review board and ethics committee reviews, create patient-led roles and organize engagement activities. Successful patient engagement requires much in the way of cross-stakeholder collaboration.
Sponsors can start by accessing patient platforms and networks, carrying out patient focus groups and interviews with patients or collaborate with patient advisory boards.
Develop Trust With Patients
Historically, patients and the public have been mistrustful of pharma companies and more generally of clinical research. This is changing as patients and pharma work together, says Keith Wilson, patient research ambassador for Liverpool Heart and Chest Hospital NHS Foundation Trust. “Through my work representing patients, I’ve learned life sciences companies are not just driven by profits; they want that next breakthrough, they want to make a good product, and they want to improve quality of life for patients.”
And patients can help make all of this possible. When pharma engages patients, they learn what matters in ways that researchers won’t have considered, Wilson explains. Recruitment and retention challenges — and how to fix them — can be made clear by patients. He gives the example of a trial that handed out contact cards to patients. But there were no pockets on their hospital gowns, so most patients would leave the cards behind.
Plain Language Communication is Required
For any patient engagement to succeed, communication is key. The trouble with most communication regarding scientific matters is that it may not connect with the lay person. The researchers worry that plain language may affect accuracy, which they can guarantee by using scientific language. But that can be hard for a patient to parse. A balance is needed.
Sponsors will succeed in recruitment and boost its brand through engagement based on trust. Likewise, patients and their families and caregivers will feel more empowered to discuss their health and how it relates to the trial in question.
Balance the Needs of Researchers and Patients
Patient involvement is advantageous but understanding the motivations of researchers and patients will help to make the process better. Researchers published in Research Involvement and Engagement investigated these different attitudes towards patient and public involvement.
Looking at a urinary tract infection (UTI) study, researchers found that patients were motivated to improve UTI treatment for the benefit of others, interact with peers and scientists, as well as by the remuneration involved. But they were also insecure about their roles and the subject matter. They expected their role to help further science.
Researchers wanted to improve research, grow professionally, help patients and generally support PPI. They expected their roles to range from being a passive support to patients to being an active participant on the board.
Understanding these motivations and expectations will help to use the PPI process for better trial outcomes.
Clinical Trials Will Be Human-Centered
As treatment becomes more personalized and the clinical research industry shifts closer to a mindset of patient as a consumer, involving patients will be essential to trial design. Adam Berman and Kelsey Jakee at PA Consulting, say companies will seek the competitive edge by providing value to patients through personalized, seamless and information-rich treatment. Ultimately, trials will be designed to accommodate patients’ lifestyles rather than around site and sponsor infrastructure.
But endpoints will also be developed with patients and regulators, which will require human-centric attitudes and approaches to patients. Part of this shift towards personalized, patient-friendly trials will involve more virtual trials. This will help to overcome common patient burdens such as transport and accommodation.
Engaging patients is key to improving the patient experience. Researchers who want to achieve better patient participation will need to design trials with patients’ real requirements in mind.
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