On 26th January, the FDA released a guidance document for industry, detailing how best to involve patients in medical device clinical trials. The guidance, which follows several years of FDA discussions, recommends that patients should be involved in an advisory capacity, not just as research participants. The upshot would be more patient-centric trials, and ultimately more user-friendly devices.
“Efforts like this are long overdue given that healthcare lags far behind other industries when it comes to incorporating consumer feedback into the product design process,” comments Dr Soheila Borhani, physician and cancer researcher at the University of Illinois. “The good news is that we do not have to reinvent the wheel. There is much that can be learned and adopted from the success of the user-centered design frameworks in other industries.”
Joe Muldoon, CEO of Fast BioMedical, adds that it is monumentally important for any medical technology to incorporate the voices and priorities of the patients.
“Their journeys are very heterogeneous and are often confusing, terrifying and exhausting,” he says. “They need to be heard, and they often are not. It is easier than most think to get very focused on the extremely challenging work of moving a medical technology forward, and in the fog of activity, lose sight of the human inside the patient.”
When sponsors don’t listen to patients
There are various ways that failing to involve patients can pose problems. In some cases, focusing on some groups of patients at the expense of others can mean bias is encoded in the design. Borhani cites assistive devices designed for patients with spinal cord injuries. Because more than 80% of these patients are male, she says women may be forced to use devices with male-oriented designs and features. A similar issue applies to pulse oximeters, which notoriously work less well in patients with darker skin tones.
In other cases, it can mean the device just isn’t user friendly – and the consequences here can be far more damaging than with, say, poorly designed consumer goods. Borhani notes that many women skip their routine mammograms because of concerns about the discomfort they may feel.
“Patient engagement during the device development process can lead to manufacturing user-friendly mammogram machines that are accepted and used by more women,” she remarks. “For example, a new generation of self-controlled mammograms give the patients the ability to adjust the pressure applied to the breasts, which gives the patients more control over their experience. The same principle holds true with colonoscopies, cystoscopies, and other intrusive cancer screening methods.”
Devices for rare diseases are another category in which patient engagement is often lacking. Because these conditions affect only a small group of people, trial recruitment can be challenging – a quarter of rare disease trials were terminated between 2016 and 2020 because of low patient numbers, according to GlobalData. This means patients often lack devices that are designed specifically for their condition. In a 2016 FDA survey, 37% of clinicians said they had repurposed an existing medical device for patients with rare diseases.
“Children are another example,” says Borhani. “Many pediatric patients require devices and implants that should grow with them… [this is] a problem that is not shared by adult patients, and can thus be easily overlooked without active participation from this demographic of patients during the design process.”
Patient advisors key in trial planning
As per the FDA’s recommendations, sponsors should consider involving patient advisors from the very early planning phases of a trial. Sponsors might want to seek their input on barriers to recruitment, data collection techniques, and meaningful trial endpoints, among other talking points.
The advisors in question might themselves live with the disease in question. Perhaps they didn’t meet the criteria to be involved as research subjects, but are still interested in participating. Or, as per more generic devices like cancer screening tools, they might be healthy volunteers belonging to the at-risk demographic.
The FDA also notes that patient advisors should be drawn from racially and ethnically diverse populations, which in turn might spur trial recruitment from these populations.
“As a general rule, the sooner patient advisors can be involved the better because errors or misjudgments can be fixed more easily early on in the process,” says Borhani. “For instance, engaging with patients only at the eleventh hour on a project involving a large team of doctors, engineers, and designers is not likely to produce any meaningful improvements.”
Putting it into practice
How all this works in practice will really depend on the sponsor. FAST Biomedical, which is developing an innovative technology for kidney function, got deeply involved in patient advocacy groups and medical societies prior to conducting its four clinical trials, as well as pursuing nurses’ input.
“We decided early on we needed the voice of the patient at the center of what we did. This is hard to do,” says Muldoon. “You have to get them involved often and early, along with their caregivers. I’ve also been fortunate two serve two terms on the Kidney Health Initiative Board, which gave me a front row seat to best practices in working with patient advisory councils. I met many patients through my role, and they were a source of both keen insights for our product development and motivation for our entire team.”
He remarks that while patients are already living full lives, and may be too busy to participate, patient advocacy groups are an important first point of contact.
“There are wonderful patient ambassadors out there who are more than willing to share their story, give you input, and connect you with other patients with experience directly relevant to what you are working on,” he says. “Companies that do this need to be more vocal on the benefits they have received from doing it.”
Nottingham Spirk, a product innovation firm that creates consumer and medical device innovations, believes lessons taken from the former can apply to the latter.
“During the initial discovery and design phases, Nottingham Spirk leverages recruitment vendor partners specialising in healthcare to identify our relevant patient targets,” says Amanda Beacher, director of insights for Nottingham Spirk. “This is very similar to how we locate pertinent research audiences during consumer product development. Virtual insights methodologies allow a broader geographic net to be cast during recruitment, so if a medical condition is rare we have a greater chance of locating affected individuals.”
The push towards patient-centricity
Beacher applauds the FDA’s recent guidance, and believes that infusing the patient voice into medical device development will eventually shift from a voluntary to a mandatory practice.
“Data linking increased patient acceptance and improved outcomes will catalyse this transition. Nottingham Spirk has been consumer-centric for the past 50 years, so the recent FDA guidance regarding patient-centricity feels familiar and prudent to us,” she says.
Borhani believes that AI systems will play a critical role in this transition, by enabling sponsors to sift through massive data sets in order to match trials up with patients.
“A recent study of more than 60,000 patients with lung cancer showed that the pool of eligible patients can be doubled using data-driven approaches, which drastically improves the odds for the successful completion of the trial,” she says. “Another study reported a near 60% increase in enrollment in a lung cancer trial as a result of employing AI-based clinical trial matching.”
While there is clearly work to be done here, many medical device companies are making an increased push towards patient-centricity. Recruiting patient advisors in clinical trials will no doubt become an important piece of the puzzle.
“I think most technologies are sincerely being developed with the patient in mind,” says Muldoon. “But I also think most could benefit greatly by a more intimate and iterative relationship with the patient’s journey from the patient’s and their caregiver’s perspective.”