Central to success31 January 2020
There is increasing emphasis on the importance of patient-centricity within clinical trials, including encouragement from the FDA to incorporate the patient voice through the drug development process. Diana Villalobos, vice-president, clinical, at Dermavant Sciences, speaks to Emma Green about how to implement this approach to maximise recruitment and retention.
While drug prices seem to be continually rising, with notable recent attempts in both the US and UK to curtail these costs, health plans are shifting more of the burden of cost to patients, transforming them into ‘consumers’. This demands a new paradigm within clinical trials, termed ‘patient-centricity’, which in many ways mirrors the consumer research models adopted by other industries. This approach is gaining traction throughout pharma, not least within clinical trials.
The biggest issue within clinical trials is recruitment. According to the National Institutes of Health (NIH), over 80% of studies fail to enrol sufficient patients within allocated time frames. Diana Villalobos, vice-president, clinical, at Dermavent Sciences, has over 20 years’ experience in all phases of clinical trials, including work within the CMO sector in the US, Canada, Germany and the UK. She is passionate about ensuring that patient experiences and perspectives are incorporated into clinical trials, starting with the recruitment stage.
One of the central barriers in enrolling patients into studies is their beliefs. “There is still a perception that it is not something that they can do as part of standard care,” says Villalbos. “There is also a mistrust of being a guinea pig; I think is it not always that appealing to patients to be in an experiment.”
Awareness and willingness varies significantly between different patient groups. “Those with aggressive cancer are willing to do anything, and they are already doing their homework and research,” explains Villalobos. “But something that is more standard and not threatening – a chronic illness, perhaps, that a patient can live with – might not be as motivating to go and participate in a study.”
The perceived commitment of participating can also be problematic. “There are a lot of patient databases that provide evidence of the idea that ‘I don’t want to participate in your clinical trial because it means I am going to have to come into the office a lot, I am going to have to fill out a bunch of forms and I don’t want to be bothered with all of that’,” says Villalobos.
Site issues can also be prohibitive to recruitment, despite their best intentions. “We have sites that will commit to providing patients because they’re altruistic,” says Villalobos. “They want to be able to provide new, novel therapies to their patient population, but they don’t have access to the patients within their catchment area or they have difficulty reaching the patients. Sometimes the sites will overcommit, they are very busy and may have several studies going and don’t realise that they don’t have the bandwidth to manage a study.”
In terms of solving the issue of enrolment into studies, communication is key, as Villalobos explains. “This is used very effectively, and has been for a very long time; even having a radio advertisement, or now using social media, information gets shared so quickly but there is a lot of success with advertising,” he says. “Of course all of that needs to be vetted through ethics committees to ensure that patient safety is kept top-of-mind.”
Social media, in particular, needs to be managed mindfully. “Social media has to be done in the right way, with the intention to respect the care and not act like a used car salesman,” says Villalobos. “It has to be done in a way that is respectful and in alignment with patient needs, rather than throwing something out there that might be uncomfortable. Social media in general does not have a great reputation for a number of reasons.”
Despite the potential of social media, with certain health conditions, other channels may be preferable. “Oftentimes with rare diseases, there are advocacy groups or patient registries that collect and help disseminate information,” says Villalobos. “That is another avenue for information-sharing for patients and sponsors.”
A new recruitment model
Incorporating patient-centricity into recruitment can help to overcome these barriers and can be implemented in a relatively simple way.
“You can pull together a patient workshop, and you get your 30 people that have a disease into a room and you talk about what would motivate them or help them to participate in a study,” says Villalobos. “We can ask them to tell us about their daily activities, how they walk through their day, whether they have a caregiver, whether they have someone to help them get around to places. All those types of questions can elicit a very different perspective to inform the trial design.”
Although seemingly straightforward, such practices represent a significant departure from conventional methods. “It is very different to tradition methods, where a bunch of scientists or physicians or clinicians would put together a design,” explains Villalobos. “From a patient perspective, that just doesn’t marry up. You can’t deliver on a protocol that doesn’t take into account the patient’s daily living experience.”
It is all parties that stand to benefit from these strategies. “Having your protocol designed from a patient perspective, while still maintaining all of the quality in the end points and risks, will make for a much better experience on the site-side,” says Villalobos. “So there is an alignment with needs of the patients and the needs of the study, which creates a nice, symbiotic approach to the patients’ next steps, saying to them, ‘here is an option, what do you think?’”
Patient-centricity doesn’t just boost study numbers. “The other piece of this is that if you are able to create a clear understanding on how patients seek out treatment, that can inform your selection of sites,” explains Villalobos. “You might need a physician and a referral site, you might need a pulmonologist to be part of the research or you might need to have both centres for every patient. Maybe you are even going to have double the sites.”
Like other aspects of clinical trials, technology can also play a role in recruitment, as Villalobos explains. “The obvious thing is that you can make things easier for the patient,” she says. “You say, instead of having to fill out 14 patient-reported outcome documents to say how things are going for your disease state, you could even use a smartwatch that has notifications on it, with different timings for medications and stuff. I know there are companies that are doing that.”
Use technology strategically
Despite the exciting capabilities of technology, it shouldn’t be implemented unilaterally. “I struggle with it a little bit because I think that technology is great for those who are willing and able to embrace it, for younger patients, those who are tech-savvy, who will be given something and will just go off with it and be fine,” says Villalobos. “I think there are some who are technologically challenged and will always be that way.”
Being flexible is key. “We have to be nimble enough in the industry to accommodate both,” says Villalobos. “We want to push, leverage and use technology, streamline data collection, make the patient experience easier and allow them to give us their data in the way that is most comfortable for them. Sometimes that does mean ‘old school’ paper methods.”
Patient-centricity has to be built into the whole of the study design. “With rare diseases and patients who are dispersed across a wide area, you can also consider a concierge service,” says Villalobos. “You can put a system in place where you call them, tell them they have an appointment, and go and pick them up to take them there.”
This can be highly challenging to orchestrate but makes a huge difference to patients. “You might be transporting patients across countries,” says Villalobos. “You need to build into your study the fact that you are going to fly them to a speciality centre and put them up for a period of time, depending on what the protocol requires.” These efforts do pay off, however. “It takes a commitment to do that, logistically and financially, but these patients are pretty motivated,” says Villalobos. “For paediatric patients, for example, the parents are going to want to take their children to get them an innovative therapy.”
Villalobos says that incorporating caregivers into the trial design is also important. “They become crucial to recruitment,” she explains. “You want to engage them, make them feel comfortable and ensure they feel that they have a say.”
Similarly to incorporating patient experiences and perspectives, this can be implemented relatively simply. “You would want to have workshops with them where they can tell you what they do every day,” says Villalobos. “Ask them how they get the patient to the doctor, how they navigate the medical system, how many times they have to do it. Those questions need to be answered.”
The future is patient-centric
In looking to the future, it is clear that patientcentricity is going to continue to be discussed and implemented much more widely throughout the pharma industry.
“It is definitely something we need to put more emphasis on and there is momentum,” says Villalobos. “I think there is a place for patient-centricity in earlyphase studies and making sure your design is wellaligned with the patient-lived experience.”
Embedding patient-centricity within clinical trial design isn’t confined to patients, sponsors and sites. “The bigger, more strategic approach is education,” explains Villalobos. “You have to begin incorporating the benefits and demands at the earliest stages of healthcare professionals’ training.”
This doesn’t just mean doctors. “All of those disciplines that are working with patients, they should have this as part of their curriculum,” says Villalobos. “It needs to be ingrained so that when they are facing patients, they are thinking about study opportunities for them.”
Greater effort from healthcare professionals will be hugely influential. “It will have a nice umbrella effect on the industry because now you have communication starting at the ground level and commitment to that dissemination of information to the patient,” explains Villalobos. “That has a nice ripple effect on patients. You can start to change the perception among them about being a guinea pig – it becomes something that patients seek out.”