Finding drugs to treat some of the world’s rarest diseases has many challenges and research is often driven by dedicated patient groups, as Big Pharma is less incentivised to develop drugs for such a small population set.

Another challenge is that the patient population is often spread out all over the world. Some patients may be more able to tolerate travel to a study while some are not able to travel at all. To achieve a high-quality trial it is important to gather data across the whole spectrum of symptoms and ensure that the participants are diverse.

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Sometimes studies cannot afford to reimburse travel costs for patients meaning that poorer patients may be excluded from the trial, which again has implications for participant diversity.

For patients living with rare diseases, the burden on them and their family is often very high, from issues around travelling long distances for treatment to the prospect of invasive procedures.

One of the silver linings to come out of the Covid-19 pandemic is that the uptake of decentralised clinical trials (DCTs) has been accelerated, bringing trials closer to patients or even into their homes.

The burden on the patient can be greatly reduced by bringing research closer to them and data gathering and patient engagement notably improved.

Decentralised clinical trials
IQVIA senior vice president and chief digital officer Nagaraja ‘Sri’ Srivatsan. Credit: IQVIA

“The decentralized model works very well for a smaller patient population that might be spread across the world and cannot come to the sites, which is often the case for rare disease patients,” says IQVIA senior vice president and chief digital officer Nagaraja ‘Sri’ Srivatsan.

“Decentralised trials really give you the platform infrastructure to do that remote engagement in a better way. It also really helps with the data acquisition component, which includes diaries, biometric data and connected devices. These are very important in rare disease situations as there are a lot of things that must be managed and monitored and you can start to take a lot of that diagnostic data from home, which enables you to see trends and patterns.”

In her virtual presentation for the OCT West Coast Conference, Precision for Medicine associate director Haley Arellano broke down some of the decentralisation strategies that are supporting rare disease studies.

Digital technologies

Harnessing digital technologies such as Electronic Clinical Outcome Assessments (eCOA), eDiaries and wearable technologies enables the acquisition of real-time and real-world data that would not be able to be recovered in a traditional trial set-up.

The decentralised model allows for trial providers to act quickly on the data they receive.

Through the use of telemedicine or apps on the patient’s phone, a healthcare practitioner can reach out to inquire about changes in a patient’s biomarkers, or to discuss what they are eating or how they are feeling and get fast feedback without the patient needing to leave their home.

“Typically, when we think of home health we think blood draws and vital signs,” Arellano said. “But home healthcare providers can actually do a lot more specialised testing in patient homes including spirometry, ECG, EEG, telemedicine-guided physical exams and in-home infusions.”

Rare disease research
Precision for Medicine associate director Haley Arellano. Credit: Precision for Medicine

eDiaries mean a patient can avoid having to travel to fill out a simple questionnaire and also reduces the burden on sites by eliminating the need for a physician to assess them in person.

Direct patient shipments mean that patients can have their study treatment sent directly to their home for administration with or without the help of a nurse or HCP.

Sensors and wearable tech can also make access to research a lot easier for rare disease patients through the use of watches, adhesive patches and other types of sensors that can be used to capture ongoing data about the patient’s daily activity – their sleep patterns, vital signs, posture and more. This reduces the need for them to come to the clinic to have these tests done by their physician.

Better data

DCTs can also improve the quality of data as data collection can be continuous and captured as often as needed, 24 hours a day using wearables and sensors.

“Decentralisation gives a better insight into the patient experience by collecting data in their real-life environment – when they are running errands, taking care of children or exercising,” said Arellano. “You are capturing data that is actually applicable to their day-to-day life.”

Additionally by bringing research into the patient’s home, trial groups have more control over variables. Safety monitoring can be improved because information being captured by a wearable or sensor can be transmitted to sponsors in real-time, allowing them to keep a better eye on how the patient is doing at home.

“All in all, accessible research means a more comprehensive study population which means better data,” said Arellano.

Increasing protocol compliance

One of the biggest concerns in clinical trials is missing, lost, damaged or incomplete data. In rare diseases this risk factor is increased; with the patient group often very small, every data point is incredibly important.

With DCTs these risks can be mitigated in a number of ways as data is collected on a patient’s phone or device and can be backed up digitally.

In-app reminders can generate notifications to a patient to input data and sponsors can easily see any gaps in data, allowing them to follow up with the patient if steps are being missed. At-home data collection also mitigates the risk of missed visits to trial sites, another significant issue in clinical data collection.

Access to electronic patient records allows time-stamped data to ensure it was collected within the window needed for the trial.

Improved patient retention

“Providing decentralised options introduces patient choice which empowers them and offers flexibility,” Arellano said. “Making home health an option for patients makes things much more conducive to their day-to-day lives and causes less disruption to the patient and their caregiver.”

DCTs can also help build a better relationship between the patient and trial providers.

“In the case of having a home health nurse where typically we would see that it is the same nurse going to the patient’s home — our patients tend to create relationships with those nurses and it really fosters a connection to the research,” Arellano added.

The real-time feedback from wearables and sensors can act as an incentive for patients. For example, a watch displaying updated vital signs can help incentivise a patient to stay on a study to better understand their health.

“Anticipating the future,” says Srivatsan, “once you put the infrastructure to engage the patient, there are lots of mechanisms you can put into the patients’ homes to start getting more and more rich data and information to engage them better and detect changes earlier.”

“DCTs are the future of all research,” said Arellano. “Specifically, rare disease research and [it] is something all companies should consider embracing and working on building into their trials.”