Compared with the meticulous processes and state-of-the-art technology that goes into much of the drug development process, talking to patients is hardly rocket science. One might be forgiven for assuming that it is, however, given the scant focus that engaging the very people for whom drugs are being developed for has received, until very recent years.

The argument for patient involvement in clinical trials is simple. If sponsors understand what the people who are living with the burden of a particular disease need, clinical trials will run more smoothly, have better outcomes and the treatments that are ultimately developed will be more relevant and meaningful to patients’ lives. Historically, though, sponsors have engaged with clinicians to determine what protocols would be most suitable for clinical trials, rather than getting the information straight from the horse’s mouth. It’s hardly surprising then that around one in five trials is terminated because of under-recruitment, an average of 30% of participants drop out before the end of their trial and, even when they do see the process through to the end, only 46% of patients would encourage their peers to do the same, according to a whitepaper by Clinical Leader.

“What we see in practice is that every single time patients are involved – I’m talking about the right set of patients – I have yet to come across someone who is not happy about it,” says Nicholas Brooke, founder and executive director of The Synergist, an incubator that brings public and private players together to build programmes that solve societal 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 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


Traditional trials with no significant patient-centred elements run between 2012 and 2017.


Clinical trials with significant patient-centred elements run between 2012 and 2017.

Economist Intelligence Unit

“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 patients to understand what it’s really like to live with a particular disease, and carrying out participation burden assessments. “If the participation burden score exceeds a certain level, it signals to the protocol author that they need to go back and revise the design of the study,” Getz explains.

“We want to make clinical trials easier for patients to integrate into their lives and lifestyles. It’s really about bringing the trial to the patient rather than attracting the patient to the trial, and acknowledging that we should be designing studies in a way that gives the patient the opportunity to participate in whatever way is easiest for them.”

Demonstrate the ROI of patient engagement

One of the big hurdles to greater adoption of patient engagement initiatives is to convince sponsors that the immediate investment of time and resources is worthwhile in the long term. “Some approaches add time, which companies are racing against,” Getz acknowledges. “It already takes so long to write a protocol to now have to accommodate input from patients. Not every company has the persistence and appetite to deal with some of the short-term challenges that come with learning how to really use and integrate some of these approaches.”

That said, research CSDD has carried out with the Drug Information Association (DIA) shows that even the simplest changes can have a significant positive impact. After analysing 121 case studies by sponsor companies, they found that the patient engagement initiatives that offered the highest return on investment were patient advisory boards, professional panels, social media engagement and patient education programmes. Impacts included faster study planning, improvements in recruitment and retention rates, fewer disruptions and delays, and more positive feedback from study volunteers. In one case example, working with patient groups saved the organisation millions of dollars that otherwise would have been spent on patient recruitment and retention. Overall, patient input into protocol design resulted in between three and 17 changes to study requirements, including the number of required visits and procedures.

“We’ve been trying to gather hard evidence that would give companies a chance to see measurable return on their patient engagement investment, but that too is difficult, as it takes time to get the data,” Getz says. “If you’re looking at a large global trial with many sites around the world, sometimes what you’re measuring in one country is not being done systematically elsewhere. Nonetheless, I’m confident that over time we will get even more quantitative data that we can integrate into the narrative of the story.”

A new type of expertise

For Brooke, it’s clearly better to engage patients at the earliest possible stage of drug development – the later they are engaged, the more likely it is that some decisions will not be totally aligned with patient expectations or needs. The challenge with early engagement, however, is to maintain a continuous relationship with the patient while the molecule moves from department to department.

“The process, tools and skills to do that have simply never been discussed, but the companies that are making the best strategic choices are shifting from shorter relationships where they say, ‘We want to work with your patient organisation for this specific phase for 12 months’ to a long-term approach aligned better with the life-cycle time frame,” he says.

Takeda’s approach, which was cited in a recent Economist Intelligence Unit report on patient centricity, has been to build patient engagement into job requirements. In 2018, the company changed its employee key performance indicators to require every R&D employee to engage in three patient-themed activities of their choice. In 2019, the company went a step further by requiring all the R&D global programme study teams in every therapy area to conduct a direct patient engagement activity that informed drug development. By the end of 2020, all global programme teams were required to have a patient engagement plan that would map out how they are partnering with patients and the patient community in the development of new medicines.

“It’s a new type of expertise for pharma companies – it’s not about expertise in the science or the development process, its expertise in driving the relationship and aligning on a shared purpose,” Brooke says. “The reality of patient engagement is that, compared to the rest of the science in drug development, it is not rocket science.”


Clinical trials with significant patient-centred elements run between 2012 and 2017 that had positive results.


Traditional trials with no significant patient-centred elements run between 2012 and 2017 that had positive results.

Economist Intelligence Unit