Living with Lupus

Direct-To-Family (Decentralized) Clinical Trials

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Direct-to-family — also known as decentralized or remote — clinical trials could open the doors for new treatments and understanding of lupus.

One of the main hurdles to lupus drug development is getting enough people for a clinical trial. Clinical trials assess the effectiveness and safety of the drug. It can take 6-7 years to complete these trials, and it can cost billions of dollars. When it comes to autoimmune diseases such as systemic lupus erythematosus (SLE), clinical trials also come upon many obstacles.

An essential part of clinical studies is ensuring that enough people participated in the research for the statistical analysis to be adequately “powered”. A properly powered study ensures that the findings of the research will be statistically meaningful (and that the researchers can reject the null hypothesis).


Challenges with Conducting Clinical Research

There are many challenges with conducting health research. Those challenges often start in a laboratory or care delivery setting but they extend through the day-to-day trial tasks as well. One of the most difficult part of clinical research is getting enough participants for the study. In fact, 86% of clinical trials fail to hit their recruitment goals in the original timeframe.

Participant recruitment is even more difficult when it comes to rarer diseases and pediatric versions of diseases — like pediatric lupus. New technology and methods for clinical trials are helping to change that while simultaneously ensuring participant safety and data integrity.

One way that researchers are changing the nature of research has been given a large push forward due to the COVID-19 pandemic: virtual healthcare. This ability to collect research-grade and clinical-grade data via smartphone and smart device has ushered in a new era of research known as decentralized clinical studies — or direct-to-family trials. 


What are Decentralized Clinical Trials?

Decentralized trials are studies that people can find, check their eligibility, enroll, and participate in from home — sometimes without ever having to go to a clinic. These studies are not limited to surveys either. New studies can include mobile phlebotomy (to collect blood samples at the participant’s home) and even medication delivery.

These strategies make it easier to participate, improving the odds that a clinical trial will reach its conclusion. They also extend clinical trials to people who have not historically had as much opportunity to participate in research including:

  • minority populations
  • low-income families
  • people without easy access to medical facilities

Digging into an example

A 2021 paper in Lupus Science and Medicine sought to look into a pilot trial of 26 participants to see how feasible this method is. The trial used remote data collection and was looking at hydroxychloroquine’s effects and ideal dose in children. This is an understudied yet crucial area of treatment. Participants were picked from a registry and called in order to recruit them.

This trials used both a remote design and a smart pill bottle. The pill bottle had an electric sensor in the cap that recorded the date and time of bottle openings. It also reminded patients with a chime and flashing lights when they need to take their dose. The sensor was connected to an app for dose scheduling purposes. Participants could opt into receiving text or call notifications. Pill counts, off-time openings of the bottle, and pill refills were also collected electronically as a part of data collection.

The researchers concluded that remote clinical trials of this type can be conducted effectively. They have many benefits for both researchers and people with lupus. People with lupus want to better understand their disease, their options, and how to explain their needs to the people around them. In-home clinical trials can help move the clinical trial process towards this end.


Home Care, Lupus, and Clinical Trials

Direct-to-family clinical trials resemble home care in many ways. Both are patient-focused methods that bring the medical field into people’s homes. Home care by professional and qualified healthcare providers significantly reduces the likelihood of hospital visits and the length of stay in the hospital.

You can read more about why keeping out of the hospital is important for people with lupus here, but, in general, people with lupus are more vulnerable to infections and have to protect themselves from exposure to disease. Going out to a hospital increases the risks of exposure. You can read more about infections and lupus here.

Several lessons can be learned from home care practices that can be applied to direct-to-family clinical trials. The first is time and human resource management. When doctors have a lot of after-hours home visits, they add to their already high workload and often burn out. The same may hold true for researchers who have to go relatively far afield to collect data. Mental health and self-care strategies are key to preventing burnout, but technology can help as well.


What is Childhood Lupus?

Pediatric-onset lupus, also known as Childhood Lupus, is a form of systemic lupus erythematosus that occurs in childhood, usually around the age of 12 and, more rarely, before the age of 5. It has the same causes and symptoms as adult SLE, but often has more nervous system, internal organ, and kidney issues and is more likely to be at a life-threatening level.

10-20% of cases of SLE start before age 20, and may be more severe due to the still-developing organs as children. The outcomes can be serious and lifelong, like dialysis. Though with modern medicine, children with lupus can thrive and grow up to be happy, productive adults.

However, although it is more aggressive, it can also run the gamut of SLE severity, from mild to severe flares. It is also treated with the same medications as adult SLE. Though there are concerns about whether the medications may have different effects in children or adolescents. Or what the correct dosage will be in non-adult patients with lupus. This is why it is important to find ways to better recruit and retain children with lupus for clinical trials. So that we can better understand how to take care of them.


Benefits of Direct-to-Family Clinical Trials

There are benefits towards modifying what’s already there to support clinical trials and incorporating remote research. Better refinement on data collection, community engagement, and safety reviews are possible, but these are already pretty good.

Direct-to-family clinical trials help children and families better participate in lupus clinical trials without the cost in time, effort, and transportation usually associated with clinical trials. They also are resilient, allowing data to be collected in the case of illness, severe fatigue, or transportation issues.  Case studies show that technology can help monitor and identify health issues quickly. This same technology – apps, fitness monitoring devices, and social media – generates data to monitor clinical trials.

Comments (2)

2 thoughts on “Direct-To-Family (Decentralized) Clinical Trials

  1. If possible we should all try and get behind the clinical trials for lupus. It may not help us now in 2021 however, for the next generation it could mean a cure or better management of the this horrid disease. I for one would volunteer. I’m terrified I could have potentially given this disease to one of my children.

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