Patient engagement means opening the door to patients for meaningful dialogue and contributions. A recent op ed in In Vivo explored several ways in which patient access can truly permeate the entire patient journey, especially in relation to clinical trial participation.


Peyton Howell, chief commercial and strategy officer at PAREXEL, outlined how there are many opportunities to enhance patient access and engagement.


One of these was harnessing the power of patient advocacy groups, which Howell says “play a key role in generating awareness of the need for new treatments and the importance of patient access to treatments, including coverage and reimbursement”. The insight and perspectives these groups can offer can have a significant impact on clinical trial participation and access, he advises.
“Patient advocacy groups can have an impact on reducing the hurdles to patient access, including insurance coverage and reimbursement. They can articulate the unmet need and urgency of access to a new treatment. And they can also support documentation of the impact and value of treatment, which is the ultimate real-world evidence (RWE).”


Advocacy groups serve as a well-informed and tightly knit network that can raise awareness of clinical studies, as well as sharing information about potential new treatment options and trials with their patient communities, he says.


Powell also articulates how including patient access as part of overall product launch planning is making a difference, saying more biopharma companies have “over-invested” to support launches and have been innovating with access services to smooth patient and prescriber access to treatments. Ultimately, he says, these actions have accelerated product use and success.


Access to clinical trials is problematic for both patients and trial sponsors. According to Howell, there is a growing awareness that patient access and participation in clinical studies is impacted by a wide range of issues; he says everything from the detail of the study protocol to the related travel and costs of study participation can affect patient participation. He also cites growing evidence that patients of lower income, rural geographical locations, and minorities are less likely to participate in clinical studies.


Research organizations must find ways to ease the burden for patients, beginning at the very start of the trial process with protocol development. Logistical barriers must also be eliminated. Simple but important factors like transportation and cost can dissuade patients from taking part in a trial. By tying the patient journey in with protocol design and taking into consideration how patients experience their disease, how they define success and the real-world implications of the study, they can optimise patient participation.


Howell also stresses that pharmaceutical companies must consider particularly vulnerable communities; for example, the small patient numbers with rare diseases means trial participants may be faced with a heavy travel burden due to geographic dispersion. “Virtual trials may provide some of the answers in this regard, he notes. Some trials cannot be made virtual, however, and still present challenges for participants; exploration of how challenges such as childcare, for example, for patients have not traditionally featured in trial feasibility.


Engaging with the patient community directly to understand first-hand what key challenges and barriers to participation they foresee can help better shape the study strategy and proactively mitigate potential issues, says Howell. “This minimal upfront investment in time and money can reap great rewards by avoiding protocol amendments due to a misunderstanding of patient burden,” he advises. “It could also advise the removal of endpoints that are not meaningful to patients but may add a significant time burden at visits, which would otherwise have negatively impacted dropout rates.”


Howell concludes by noting that a recent Economist Intelligence Unit (EIU) report commissioned by PAREXEL found that trials using patient-centric designs were 19% more likely to be launched compared than those that did not.


“Continuous and meaningful dialogue with patients is critical to achieving more streamlined processes that get patients treated more efficiently and effectively; both within clinical trials and throughout the patient journey.”

PFMD believes that patient engagement should become the cornerstone of successful research and development. This article raises a number of important issues that must be addressed before true patient engagement can be realised.