Protocol design has historically been the domain of clinical research scientists. But that’s changing. Once seen primarily as ‘study subjects’ who had research performed on them, the culture is shifting towards engaging patient partners who contribute across the spectrum of clinical development, including in the design of trial protocols. The early adopters are reaping the benefits. The imperative for the rest of the industry is to embed patient experience as a must-have input sooner rather than later.

Simple innovation

When we hear the word innovation we often leap to thinking about things like artificial intelligence, blockchain and other cutting-edge technologies that – let’s be honest – most of us are in the dark about. But innovation can be so much simpler than an app or other tech-centric solutions. Innovation is about thinking differently. Patients can help you do that, if you let them.

In a 68-country survey examining patients’ attitudes, perceptions and experiences of clinical research, three quarters of respondents listed ‘types of medical procedure required by the protocol’ as a very important consideration that influenced their decision on whether to participate or not.

And yet it’s been estimated that as many as one-third of clinical trial procedures – such as blood tests or biopsies – are not crucial to the applications for drug approval. The involvement of patients in trial protocol design can shine a light on these superfluous tests and help shape trial protocols that are more convenient and therefore more likely to recruit.

Convenience factor

Convenience is hugely important to patients. An evaluation of 30 patient advisory boards found that most were making recommendations about the convenience and feasibility of study visits, and the schedule of procedures performed. This shows that focussing on patient experience goes beyond simply thinking about disease factors. It’s about immersing yourself in a patient’s disease as well as their everyday life and viewing the impact of clinical trial interactions on this level.

As Michael Mittelman, a rare disease patient, puts it: “The decision to enter a clinical trial isn’t simply binary – ‘yes I want to’ or ‘no I don’t want to’ – the practicality of it isn’t as simple as the industry makes out.” The problem, according to Shwen Gwee from Novartis, is that we’ve been focussing on the wrong customers. “Oftentimes pharma only talks to healthcare professionals (investigators) to understand patients, which is an important, but skewed perspective,” he says.

Kelly McKee from Vertex agrees: “We must design clinical trials to fit the needs of patients and not just researchers and clinicians.” Patient advocate Lilly Stairs gives an example of home infusion as a way to improve patient experience. “If a medicine needs to be delivered, then why can’t we send an at-home infusion nurse so that patients don’t have to travel when they’re already not feeling well?”.

While strides are being made, we need to accelerate and spread the change throughout the industry. Clinical trials are about improving patient outcomes, and convenience shouldn’t be a barrier for them to overcome so they can participate and help pursue those outcomes.

User experience mentality

Adapting to the increasingly competitive age of clinical trial recruitment means adapting to a user experience mindset, seeing patients not as an amorphous group to be targeted by mass marketing, but as individuals or sub-groups of people with particular shared characteristics and needs.

For example, how do patients who need to visit a dialysis centre a few times a week become aware, motivated and enrolled in a study? And how does that differ from people who participate in an outpatient, acute-disease study?

As Shwen Gwee from Novartis puts it: “We’ve been too transactional up until now in our approach to clinical trials, and tactical in execution, rather than thinking in terms of service design and changing the overall experience for every patient touchpoint along the journey.”

Thinking in this more nuanced and strategic way is part and parcel of the consumer advertising world, which is replete with examples of how to effectively use digital tools and social media to engage different types of customers.

“We need to start thinking about taking the consumer tech approach rather than the scientific approach to how we present information, how we engage with patients, and how we create the overall experience for what patients go through in a clinical trial,” Gwee says. He adds that we need to dramatically shift how we think about clinical trials. “The traditional model of handing off to the sites is more a business-to-business type transaction rather than a business-to-consumer, which means we’ve been focussing on site experience more than patient experience,” he says. This needs to change, though we still need to continue to engage and support sites to help drive better experiences through and for them as well.

Kelly McKee from Vertex says: “We need to establish value propositions for our studies. If you wouldn’t participate in a clinical trial, how can you expect anyone else to participate?”

Designing clinical trials with patients as partners, providing educational materials and communications using health literacy principles, eliminating unnecessary visits and procedures, incorporating telemedicine and technology, and providing study results to patients, are all tangible tactics that can be employed by study teams and will, ultimately, improve the understanding and appreciation of clinical trials.

Lindsey Wahlstrom-Edwards from Antidote adds that we need to stop trying to find a “one size fits all” solution. She says that patient motivators participating in research and how they would like to receive information about research vary greatly by condition and demographics.

Eligibility frustrations

Eligibility criteria are guidelines describing characteristics of all participants of a clinical trial. They help make the trial a fair test. The specific characteristics will vary from trial to trial and often include patient age, gender and medical history.

Such criteria are often there to protect either the patients or the study. But are all these eligibility criteria needed, or are they a symptom of a cut ‘n’ paste mentality, leading researchers to use previous trial protocols as templates for their next studies?

Whatever the reason, patients – and their doctors – often grow frustrated when they encounter the sometimes-overwhelming requirements to join a study.

Sharon Allin works on clinical trials at Biogen. When asked what she thinks is holding back clinical trials, she lamented the industry’s habit of “writing protocols for the perfect patient that doesn’t exist”. Clearly the issue of inclusion and exclusion criteria isn’t a simple case of removing all those that are a barrier to patient participation – they are intended to avoid harm to patients or confounding factors for trials. But an open and honest interrogation of the often-lengthy eligibility criteria – with patients – could at least sometimes lead to an easing of criteria, giving more patients the chance to try new treatments and a greater chance of a clinical trial meeting recruitment targets. Surely that’s worth the effort?

For those who are sceptical, “run an A/B test”, suggests patient advocate Lilly Stairs. “Run one trial where you work with patients from day one, then pick another trial in a similar therapeutic area and don’t involve patients. See what the difference is. I guarantee you the one where you work with the patient is going to enrol faster, it’s going to have a much higher retention rate and you’re going to see a better patient experience,” she says.

Financial value of patient engagement

The choice of inclusion and exclusion criteria can affect the duration and cost of a clinical trial, not to mention the likelihood of the trial meeting desired enrolment levels and retaining patients. A study of thousands of clinical trials showed that more than 40% had amended protocols prior to the first patient visit, delaying trials by an average of 4 months. Of course, some protocol amendments can’t be avoided. But the potential for amendments can be reduced with better planning, with and for patients.

And the evidence shows just how valuable this patient involvement can be. A recent study that looked at return on investment for engaging patients in trial design showed eye-opening results. Researchers looked at an oncology research project in which a patient review of the pre-phase II protocol had avoided one amendment to the trial, increased enrolment and adherence, and reduced dropout rates. They then used a financial modelling technique called expected net present value (ENPV), which is a cumulative measure of cost, time, revenue, and risk.

The ENPV was found to be an astonishing US$35M, rising to US$75M by Phase III. Depending on the scenario, the researchers conclude that a US$100K investment in patient engagement in the design of a trial could produce an ENPV 500 times that figure. Clearly, understanding patient experience pays.

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