Most people and organizations are wary of change. New processes and opportunities create risks that could outweigh the benefits of deviating from the norm. But then a global pandemic grinds industry to a halt and requires innovations at speeds that were previously unheard of. This is what has occurred with direct-to-patient (DTP) clinical trials.
Even as the pandemic slows and cases get under control, trial developers continue to lean on direct-to-patient clinical studies. It’s likely that this design style will only grow in usage. Here are some of the challenges — and one of the big opportunities — researchers face as they look to invest further in direct-to-patient clinical trials.
Travel is one of the biggest hurdles facing patient recruitment. Researchers studying rare diseases will recruit patients across the country but then may require them to travel frequently for testing and medication administration.
In theory, direct-to-patient trials can help solve this enduring problem in clinical research. However, removing one hurdle can merely create new ones.
For example, vulnerabilities in patient privacy emerge. When researchers have to ship medicines and devices to patients’ homes, how can they be sure that the points of contact — e.g. signing for a package from the courier — remain compliant with HIPAA or GDPR regulations?
Anonymizing the sender and the recipient is one solution, notes Arnold Bernard at logistics company AmerisourceBergen. But that still creates complications for the delivery service provider, which must ensure safe and accurate delivery between two anonymous parties.
For direct-to-patient models of clinical research to truly thrive, the infrastructure must first be in place to ensure the same level of patient experience that participants would receive when they travel to a research facility.
Here is another facet of the patient experience question.
As we have written before, patients want to feel connected to the people they are working with in clinical trials. Telemedicine helps in this regard, but video calls only go so far. This is a great way to check in on patients and to ensure informed consent, for example, but in certain trials there are times when person-to-person interaction is the optimal — maybe only — way to conduct the research.
Hybrid models of working have evolved from this. For example, researchers in some such trials have been administering initial doses of a treatment to patients “in a hospital setting or other healthcare setting, so that there's support in case of [an] adverse event,” says Alex Guite, vice president of strategy and services at World Courier.
From there, the patient can administer subsequent doses at home. Having that initial in-person connection, though, gives the patient confidence that they won’t have any issues with the medication. They can also learn to administer the treatment properly by learning in an in-person setting.
Beyond patient confidentiality, shipping supplies and therapies directly to a patient’s home creates a variety of logistical problems.
For one, some drugs “require special preparation like reconstitution of a lyophilized powder, thawing of an ultra-frozen drug product, or preparation of other concentrated drug product for weight ... based dosing,” writes Ryan Ridge, senior director of IRT operations at YPrime.
The COVID-19 pandemic gave the entire world a lesson in medical logistics. The Moderna and Pfizer vaccines have to be stored at such cold temperatures that healthcare professionals had to be trained on how to store and handle them.
In direct-to-patient clinical trials where a drug’s transport and storage must be executed with, well, clinical precision, there needs to be at least significant patient education and training. Alternatively, an off-site professional could go to that patient’s home to assist with last-mile delivery, storage and handling of the drug.
Further, some patients need trial-provided tools or specific equipment to complete trial staff directions. This means that every item that is sent out as part of a trial needs to be tracked, monitored and maintained.
“For DTP studies, kits will get to patients via a central pharmacy or visiting nurses, and so having reliable shipping systems in place is essential, as equipment needs to be tracked and accounted for at all times,” writes the team at Woodley Equipment Company.
This is a conversation that goes both ways, too. When patients have samples for researchers to collect, for example, the supply chain infrastructure needs to be able to collect and ship that sample with the same ease and precision.
As such, researchers will have to build and fortify relationships with their logistics partners to ensure delivery and collection of all materials is reliably smooth throughout the duration of a trial.
Despite the challenges, DTP trials do offer the promise of new methods of real-world evidence (RWE) collection.
“While these concepts are fairly new, they can provide invaluable data in terms of patient feedback and real-life contextual understanding of treatment efficacy,” according to CSOFT Health Sciences.
“Unlike randomized clinical trials, where the environment is controlled and where participants are typically not representative of entire populations, RWE gives broader insight into how the medical product may work in real life context, therefore allowing medical professionals to improve care with a new deeper understanding of their patients on an individual, case-by-case level.”
This is one of the big rewards for clinical researchers when direct-to-patient models achieve stability and maturity. Evidence collection inside of a patient’s own home could reveal so much about a given treatment’s therapeutic value or the hurdles to its adherence.
The COVID-19 pandemic has opened doors for researchers to experiment with direct-to-patient trials years before they otherwise might have. As more clinical research organizations have success with this model, it will likely become the go-to option for different types of treatment and disease.
"The Direct-to-Patient model is particularly advantageous for trials involving rare diseases or pediatric treatments and patients across many therapeutic areas that cannot easily travel to sites, whether due to illness or other factors," says Mike Sweeney, senior director of patient centric logistics at World Courier.
Researchers might have had to scramble to create direct-to-patient trials during the pandemic, but now is the time to invest time and effort into this option. While they require more logistical resources, DTP trials have the power to provide better data sets and increase patient recruitment.