Juliet Moritz, vice president of patient and stakeholder engagement at Premier Research, called the trend toward patient-centric clinical trials as the logical evolution in how the industry approaches and conducts drug development. “It’s moving us from a purely scientific and clinical approach driven by researchers to one that is shaped by the needs of patients and their families and caregivers,” she told us.
In practice, patient centricity takes many forms, Mortiz said, for example, acknowledging the burden of participating in a clinical trial, designing studies with outcomes relevant to patients, and ensuring those involved have “realistic expectations about how they might, or might not, benefit.”
Moritz said the recent momentum is a reflection of the need for more participants to fill an increasing number of clinical trials. It also marks recognition on behalf of the industry that patients and caregivers who are better-informed and included in the process will be more engaged and less likely to drop out of a study.
Still, when the terminology emerged about five years ago, Clare Grace, vice president, site and patient access, Syneos Health, said many struggled to understand what patient centricity meant to their organization.
“What we have seen is an evolution of that understanding being developed, pilot projects being implemented to help organizations work through the ‘how’ within their organizations to today, where we are starting to see organizations actually adopting new processes routinely to ensure patient feedback is implemented across all development sectors from protocol development, endpoint development, and market access,” Grace told us.
The efforts to implement patient centric approaches are becoming more wide-ranging and sophisticated, noted Moritz, who said organizations are increasingly turning to patients to make sure they are ‘pursuing the right goals.’
“To discern what really matters to patients, we need to understand the burden of the disease and what living with the condition is really like,” she added.
This enables a better understanding and development of Target Product Profiles (TPP) and claims, said Grace. “This is then further refined with the development of the endpoints used to measure those product claims, with both patients and regulators working to support clinically meaningful endpoints, not just scientifically valid endpoints,” she explained.
As such, including patients in clinical protocol design is becoming more common, Grace said, and the practice will become the ‘norm’ with regulators wanting to ensure involvement.
The industry also is working with patients earlier in the development process. Said Moritz, “If you begin your patient engagement efforts in Phase II or Phase III, you’re too late.”
“If you’re going to design clinical development programs that address issues that are relevant to patients and families, and not just answer scientific questions, you need to move these engagement efforts up to the beginning of the process,” she explained.
“The patient voice can mean the difference between developing a therapy that changes a numerical value in a lab somewhere and one that produces improved medical or functional outcomes.”
“This knowledge is critical to our understanding how therapeutic changes affect patients in ways that are meaningful to them — and to translating biological change to real-world clinical benefit,” Moritz added.
That patients respond differently is important to recognize, even if the therapeutic shows significant benefit, so patients approach trials with realistic expectations, she added. This is particularly important with next-generation treatments, such as gene transfer therapy, which may halt disease progression, but not reverse damage or address all symptoms.
Involving sites and patient advocacy groups
Clinical research sites are able to provide meaningful insight into the collective patient experience, providing a window into issues that can be addressed to speed up clinical trial timelines, reduce costs and most importantly deliver medicines to patients.
They can pinpoint issues or trends and can share the practical challenges patients participating in research are facing. This is critical because we know that it is these practical challenges that most often result in patients declining to participate or dropping out of studies.
From Moritz’s perspective, the issue of patient-centricity can’t credibly be addressed without considering patient advocacy groups, who have a large role in clinical trials – one that is steadily growing.
“Regulators in the United States and Europe are actively encouraging the efforts of these groups, and we work hand and hand with many of them — from the largest and most well-known to those that consist of a worried mom working late into the night to raise awareness of a condition that has turned her family’s life upside down,” she said.
The impact of working these communities is exemplified by the industry’s work with muscular dystrophy (MD) patients. As Moritz explained, MD studies for a long time required patients who were able to walk, thus excluding those who had become non-ambulatory.
“But when an MD-focused advocacy organization talked to those patients, they generally understood that they were likely to lose ambulation, and what was most important to them was the ability to use their hands — and nobody was testing for that,” she said. Subsequently, the group of MD advocacy organizations, health care providers, and researchers worked with the FDA to develop new guidelines for conducting these clinical trials.
A challenge, though, is the number of these organizations. Said Moritz, “Some conditions have led to the development a dozen or more patient advocacy groups, and families looking to get involved can easily get overwhelmed.”
“We’re always mindful of the fact engaging in patient advocacy is an intensely personal decision,” she added, explaining that many families opt-out, most often due to privacy concerns.
“Many don’t want to share details about their private lives with strangers, especially at a time when it seems a new cybersecurity threat appears almost daily,” said Moritz. “Being truly patient-centric means we have to develop strategies to reach those families as well.”