Patients are increasingly engaged in their own healthcare. They have access to research, community forums and patient advocacy groups. They feel more empowered to take control of their own health. This makes direct-to-patient trials an obvious choice for certain trials.
But other than meeting patients’ changing needs and wants, DTP trials can also simplify and democratize access to clinical research. Minority groups that don’t often live near major academic institutions, patients with work or caring commitments, immobile or very ill patients, or those who simply cannot travel to sites can all benefit from DTP trials.
We explore the opportunities and challenges of direct-to-patient clinical trials and which types of trials are best suited to this method.
The shift towards DTP trials is in line with the FDA’s wider call for decentralization of clinical research. Whether sponsors adopt hybrid trials, which combine virtual trials and site-based research, or incorporate real-world evidence into research, change is happening.
RWE can be collected from wearables or data submitted from patients themselves or even through electronic health records. Life sciences associate consultant Adam Kershner points out how former FDA commissioner Scott Gottlieb believes that this approach would make research more agile and accessible to more patients. There’d be less administrative requirements and costs, while data would be of a high quality.
When sites are removed from clinical trials, costs come down as does the administrative burden. Another benefit is the diversification of operational approaches, says Bert Hartog, Ph.D., senior director of clinical innovation at Janssen Pharmaceutical.
He tells Denise Myshko at PharmaVoice that DTP trials add flexibility to designs in a way that traditional trials don’t.
Data collection is another boon for DTP trials. It is less expensive and can be more efficient to collect data through RWE, EHRs and patient-gathered data. Data can also be collected more frequently. Hartog adds that siteless trials can also help to reduce “white-coat syndrome” — bias from being tested or assessed in a clinical setting.
Smartphones and wearables enable patients to connect with researchers remotely. The result is improved healthcare and health outcomes, writes Shanique Smythe, senior consultant at clinical consultancy, Halloran. In particular, these digital tools have enabled better patient-reported outcomes while also improving data quality.
Data can be transferred automatically from patients’ devices to the clinical database. Science 37 is a good example of how companies are changing clinical research. Smythe says the company has launched “clinical research in a box” with patients receiving all of the required clinical trial materials at their homes.
Remote investigators and study coordinators staff the central study site virtually to guide patients through the process.
Being able to recruit more patients also means being able to recruit a more diverse patient population. The team at Science 37 set up a DTP Phase IIb trial for trial sponsor AOBiome to treat patients with moderate acne.
Some of the advantages of the DTP approach include faster recruiting: 372 patients within seven months from a screening process of 8,000 potential participants. The Science 37 team says that’s half the time it would normally take in a similar trial.
There was also a greater diversity of patients — with 41 percent being non-Caucasian, a rarity compared with most trials. Sponsors that want a more diverse and demographically representative population would benefit from DTP trials.
This is because minority communities don’t often live near large academic centers where research takes place. Researchers are also guilty, albeit unwittingly, of implicit bias, the team says. Researchers may doubt a patient’s ability to complete the trial based on where they live, what job they have and because of perceived family commitments. DTP trials make research more democratic.
Patients’ needs have changed. The way clinical trials are designed have had to adapt to these new demands, says physician Joanne Waldstreicher, chief medical officer at Johnson & Johnson.
Indeed, the pharma giant has begun to run direct-to-patient trials, specifically to try to increase the delivery of treatment to children. By relocating treatment from trial sites to patients’ homes, the organization hopes to open up more patients to research.
More patients participating in DTP trials is good for patients. It’s good for sponsors too. The two biggest issues for sponsors are patient recruitment and retention, writes Mike Sweeney, senior director of patient centric logistics at World Courier, and DTP trials help overcome both. Indeed, DTP trials have increased patient recruitment by 60 percent and retention by 95 percent, he notes. Part of the reason why is that DTP trials enable immobile patients or those too ill to travel to participate in research. Elderly and young patients also benefit from this type of research as do those that would require frequent visits to sites or who are based in remote locations.
Sponsors also benefit from DTP trials through increased operational efficiency, reduced waste of drugs and quicker completion times.
Although siteless trials are increasingly common, they do remain better suited to certain types of trials. For instance, trials that rely heavily on patient reported outcomes work best when the devices or drugs used are neither invasive nor dangerous, according to Jimmy El Hokayeem, Ph.D., associate director of program development at contract research organization Biorasi.
Trials that don’t benefit as much from siteless trials are phase I studies, which require regular blood draws, he explains. Generally, any treatment that requires patients to be in a hospital setting won’t work as a siteless trial.
DTP trials present opportunities and efficiencies to sponsors but are not without challenges. Scalability can be an issue, says Joseph Kim, senior advisor in translational technology and innovation at Eli Lilly and host of Lilly’s podcast The Elixir Factor. If blood needs to be taken, for example, patients will need professional help. Clinical teams can be sent to patients homes but expansion remains limited. Nevertheless, technology is making siteless trials far simpler to conduct.
Another challenge is proper storage of medication, notes Glenda Womack at Applied Clinical Trials. Temperature-sensitive medication needs to be maintained to high standards at all times and in the absence of an onsite medical team, this could become an issue. Furthermore, even while patients can more easily participate in DTP trials, Womack warns that ensuring adherence to treatment regimens can be challenging.
Still, challenges can be overcome. CROs and sponsors are already using shippers with extended stability for storage at patients’ homes, for example. Plus, they are using dosing diaries to ensure patients are following the correct protocol.
The clinical research community can learn from how people use their smartphones to improve patient adherence to treatment routines. Indeed, they must become as addicted to healthy behavior as they are to their phones, says Dr. Joe Kvedar, vice president of connected health at Partners Healthcare. While not at that point yet, pharma must tap into this behavior.
Kvedar believes sensors and wrap-around products are key. A medication or injectable with a related sensor and app informs researchers when it has been taken. While not yet ubiquitous, this technology is becoming more mainstream. For example, Sanofi and Abbott created FreeStyle Libre to monitor glucose, and smart pens and insulin titration apps for accurate dosing.
Patients want to feel connected to humans even if they’re participating in DTP trials, which is why physicians consulting with patients via telemedicine has been so successful. With his colleagues at the Medical University of South Carolina, assistant professor Brandon Welch created a telemedicine platform called doxy.me, which connectors doctors, clinical researchers and trial participants via video. Increasingly, clinical researchers are using the platform to recruit and consult with patients.
They are also able to obtain consent, Welch explains. The human experience that patients want can also be fulfilled by chatbots. Welch’s doxy bot communicates with patients and asks questions to capture data.
Clinical research is changing and DTP trials is one way that this is happening. These types of trials can be valuable to a number of diverse patient needs. DTP trials can improve access to research but they are not without challenges. Careful planning, reliable technology and choosing the right type of research will help to make DTP trials succeed.
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