Patient-centricity has become a buzzword in recent years, with companies touting more inclusive trial designs and bringing patients and advocates into early planning stages.
On Clinical Trials Day 2025, the Clinical Trials Arena team asks: has the push for patient-centricity truly changed the way trials are run?
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Most clinical trial conferences now have a stream dedicated to patient engagement or patient centricity, and these streams are always filled with delegates who are ready to openly speak about how they can improve designs for patients, showing that the industry is starting to consider this way of thinking. These approaches have been more heavily adopted in the rare disease space, where enrolment is already a challenge. Ensuring that patients’ opinions are considered in the protocols means it will likely be easier to enrol patients into the clinical trial.
Sponsors need to understand why patients take part in research to really focus on patient-centricity, says Dr. Dominique Demolle, CEO of Cognivia, a company that develops analytical tools to optimise and accelerate clinical development. “Patients who agree to participate in a clinical trial are doing it for the benefit of others and it has to represent what would be the real world,” Demolle explains.
Change has occurred
Demolle believes that change is happening, and this benefits both patients and stakeholders. In the past, strict protocols made finding eligible patients difficult. Today, sponsors are working with patient advocacy groups and patient leaders from the outset.
While this has been an issue, Dr. Stacie Bell, chief clinical research officer of Lupus Therapeutics, the clinical research affiliate of the Lupus Research Alliance, says she has seen a push by sponsors to get to know what patients need and want from clinical trials to improve enrolment.
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By GlobalData“Whether that be looking at a clinical trial protocol before it is actually finalised, or companies meeting those with a particular condition, that perspective can be incorporated into a clinical development programme,” Bells explains.
According to Medable’s Vice President of Patient Engagement, Jena Daniels, the onus cannot solely be on the sponsor. “It’s on every vendor, every partner, every CRO that they then choose to work with within their trial,” Daniels explains.
That includes the regulators. Rare-disease patient Suzanne Harris, who works for marketing in SubjectWell notes that regulators including the US Food and Drug Administration (FDA) are pushing patient-centricity but have a way to go. “The issue becomes, not many people know how to actually make it happen. I feel like companies are on the cusp, but the industry doesn’t know how to pull it all together.”
Not only in the US are regulators pushing this approach, but in Europe, advocacy groups and patient representatives have historically been included in European Medicines Agency (EMA) scientific review panels for protocols, showing the regulator’s desire for sponsors to adopt patient-centric designs. While this has primarily been in rare diseases, it is now also happening in oncology.
Not only is this something the regulators are providing guidance in, but in some cases will be considered in a drug approval, adds Cardiologist and CMO of Clinical Ink, an eSource solution company, Dr. Nicholas Alp.
“Many regulatory authorities now require evidence that you’ve done that as a sponsor – that you have had input from individual patients and advocacy groups and other experts to seek their feedback on what is proposed in the study, design the assessments that are planned to be used,” says Alp.
How sponsors can go further
Promising therapeutics have failed in the clinic not due to an initial lack of enrollment but due to patients dropping out of studies, highlighting the importance of retention when considering patient centricity.
“If we have poor adherence and poor retention, then we are at risk of having missing data, incomplete data or incorrect data and that is the focus of every scientific experiment,” Alp explains.
The key to continuing the positive trend is to engage patients even earlier – in the pre-protocol stage to learn what endpoints are meaningful for patients, according to Harris.
“An example that I like to use is, if it’s a headache trial and the medication makes you drowsy, but patients want to be able to live and work then sponsors are missing what a patient needs,” Harris says. “They need to understand patient motivation – that is critical.”
Bell agrees, adding that conversation with patients will help sponsors better understand how to design protocols. “One example in lupus is that patients suffer fatigue or brain fog which is something that unless you’re living with lupus, it’s difficult to understand. If you’re looking at a clinical trial endpoint that’s looking at joints or skin manifestations and those types of things, not that they’re not important, but sponsors need to understand what that lived experience is. There are things that are impactful and meaningful to those living with the disease.”
Sponsors also need to find more ways to connect to patients to make more people aware of clinical trials, Bell continues. “Sponsors need to hear the individual’s voices about what they really need in clinical trials. That can come from meeting patients and that is critically important to having clinical trials be effective.”
While it is important at an early stage to engage, keeping patients engaged throughout the study is also important, primarily by removing patient burden. Technology has been helping to improve patient-centricity and remove patient burden in trials by reducing site visits, Alp worries that some sponsors go too far when using decentralised models, which ends up adding burden again on patients.
“When it’s possible to capture data from patients remotely, there is a temptation to just add more and more assessments remotely. While you think you’re making life easier for the patient, actually, you’re loading them up with assessments, and you’re increasing the burden. Even in the hybrid and decentralised model, it’s important that we follow what I think is like the first principle of clinical trial design – keep it simple and just collect the necessary data.”
Not only are the patient’s needs important, however, adds Daniels, who believes that sponsors and stakeholders also need to consider caregivers more to make trials more patient-centric.
“There’s a whole support army behind every person participating in a trial, but these people can be unsung heroes behind the scenes supporting them. To round out that patient-centric approach, you must recognise the impact, influence, and participation that the caregivers have,” Daniels explains. “Caregivers don’t sign the consent form and may not be getting compensated for their time, but they are often just as invested as the patients, and it’s such an important role and something we also need to highlight as part of that patient-centric approach.”
Harris adds that sponsors also need to support sites and patients throughout the clinical trial because if sites are busy with administrative tasks, they have less time to focus on patient care. Daniels agrees that sites should also be at the centre if a sponsor wants to make a trial more patient-centric.
Daniels says: “If the sites themselves aren’t fully supported, they end up spending time figuring out the technology and less time with the patient. When we talk about patient centricity, we’re doing a disservice unless we also talk about supporting sites.”
While patient centricity is important, it needs to be balanced by conducting an effective trial. “A sponsor cannot embark on a protocol if it impacts efficacy and safety so if there is some pushback to that from a patient advocacy organisation, they would say no,” Demolle concludes.
