Trial design
problems in the healthcare sphere. “Insights from patients are often unexpected, but that’s exactly why they add value. It’s a perspective that is missing around the table, but helps improve the outcome of clinical trials.”
Research backs him up. For example, the Economist Intelligence Unit compared 4,000 clinical trials with significant patient-centred elements with 20,000 traditional trials (all were run between 2012 and 2017). Of the patient-centred trials, 87% had positive results, a much higher rate than 68% of traditional trials. Clearly, there are pockets of good practice. The problem, according to Brooke, is that’s just what they are: pockets. He remembers talking to patient leaders back in the mid-2010s and finding that there were many good stories to tell. “But they were far from the norm,” he says. “We wanted to be a spark or contributor to the shift from anecdotal to something replicable or scalable that can then become the norm.” The ‘we’ he’s referring to is Patient Focused Medicines Development (PFMD), a Synergist programme that was formed in 2015, following these conversations. “What we have done is really look at these pockets of good patient engagement, identify the good practices and work out how we can turn that into best practice,” he explains. PFMD’s Patient Engagement Management Suite is the foundation of their work. It includes over 150 activities that can – and should – be carried out with the help of patients over the life cycle of drug development. For each activity, there are seven criteria that PFMD says must be respected by sponsors and other stakeholders. The team is also developing how-to modules for each of the most critical decision points, from clinical trial design to writing plain language summaries. “We are only a small piece of the puzzle though,” Brooke is keen to stress. Regulators are also investing resources in clarifying the role of patients in drug development. The FDA, for example, is developing a series of four patient-focused drug development (PFDD) guidance documents, with the aim of making patient involvement systematic
Clinical Trials Insight /
www.worldpharmaceuticals.net
rather than anecdotal. “It’s a work in progress, but I’d say we are at least halfway to the culture shift. We’re putting in the processes, tools and relationships to make that happen,” says Brooke.
Bring the trial to the patient Kenneth Getz is deputy director and research professor at The Tufts Center for the Study of Drug Development (CSDD) and an internationally recognised expert on clinical trial management practices and trends. He agrees that much progress has been made over the past 12–15 years. “For many years, pharma would look to the literature to see what had been written about a given disease or talk to a medical expert, but never to the patient,” he says.
“Insights from patients are often unexpected, but that’s exactly why they add value. It’s a perspective that is missing around the table, but helps improve the outcome of clinical trials.”
Nicholas Brooke
“A big part of the patient engagement movement is the recognition that the patient’s view and the clinician’s view is not always aligned, and that what matters to patients has to be factored into the design of our studies, how they’re conducted and, ultimately, in developing a clinically meaningful new therapy.” One of the most important milestones in the movement has been the development – and increasing popularity of – patient advisory boards. These are listening sessions where patients with a given disease and their caregivers are presented with a draft protocol and asked to comment on it, helping sponsors determine what the primary end points or goals of a study should be and what patients’ practical considerations are. “Patients will be very quick to tell you there are too many visits or that they would never go to a research centre and spend eight hours in and out of the lab or the pharmacy,” Getz says. More sponsors are also holding patient journey workshops, where researchers meet with groups of
Clinical trials are working to increase patient engagement to improve trial processes and outcomes.
20,000
Traditional trials with no significant patient-centred
elements run between 2012 and 2017.
4,000
Clinical trials with significant patient- centred elements run between 2012 and 2017.
Economist Intelligence Unit 15
Frogella/
Shutterstock.com
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