Pediatric Rare Disease Clinical Trials – Challenges and Solutions

Clinical trials (CT) are the long and complex processes for bringing novel medical products to patients through the regulatory process of evaluating the safety and efficacy of treating specific medical conditions in humans. In addition to the challenges associated with adult clinical trials, there are unique challenges in conducting pediatric clinical trials. The challenges facing pediatric CTs are particularly severe in the case of rare diseases that affect only a small subset of the population.

Pediatric CTs are perhaps the most challenging area of treatment development as the risk is higher. Children have a lower risk tolerance to drugs, and a clinical trial can affect their lifespan. There are complex logistical problems in coordinating children and their guardians’ schedules for travel and trial site visits. Children are growing during a clinical trial and can start forming their own opinion. For instance, there can be a significant difference in a one-year-old and the same child at age six.

When considering pediatric clinical trials in rare diseases, the challenges are manifold. A disease is considered rare in the US if it affects fewer than 200,000 Americans. According to the American Journal of Managed Care, there are more than 7,000 distinct rare diseases in the world today, affecting about five percent of the overall population—30 million Americans, 400 million people worldwide. Over ninety-three percent of these rare diseases do not have any FDA-approved treatment yet. Half of those affected by rare diseases are children. Three out of ten of these children will not live to see their fifth birthday. Meanwhile, the time it takes for a patient to get an accurate rare disease diagnosis is about seven years.

In addition to regulatory considerations and protocol design, pediatric rare disease clinical trials have added complexities that the target patient population is extremely small and sparsely distributed globally. Since over 93 percent of rare diseases have zero approved treatments, participating in a clinical trial, if an option is available, is often found to be the best option for informed patients. Patient’s family members, often mothers, take a very active role in care management, choices, and time off (often from their paid work) if the child must participate in a clinical trial. Many developmental rare diseases can be debilitating and severe conditions making travel many times harder compared to some of the common conditions.

One logistical challenge in pediatric CTs is that children are not free agents. Informed consent requires that the participants’ parents or guardians understand what is at stake. There is also a whole subset of logistical issues, given that children are dependent on their parents’ transportation and work schedules.

It is unfair to expect patients and their caregivers to visit a hospital or clinic when it is really not necessary, especially when the necessary tasks can be effectively and safely completed from the comfort of where they live.

  • The best means to sustain and scale of pediatric clinical trials.
  • How utilizing human-centric SaaS not only improves the efficacy of clinical trials and its impact on pharmaceutical efforts.

The best way to deal with these issues is to use human-centric technology to resolve these objections. For both trial regulators and participants, the basic concerns are free information exchange and verification—both parties understanding what has been told—and a way to resolve side issues.

With digital communication, prospective clinical trial participants can ask questions, discuss the trial with the investigator team, to complete a proper informed consent remotely. Trial sponsors can translate background material, including educational videos, into as many languages as needed. Enabling participants to join a trial from any browser enabled device that they are already comfortable with—smartphone, tablet, or laptop—can also create better ties with the community.

Both sides need options. Clinical trial participants need to be able to get information from the comfort and safety of their own homes. Trial sponsors need to make the field of participants as diverse as possible, and at the same time they need to make sure they’re getting informed consent. Digital technology—particularly Bring Your Own Device (BYOD)—is the way to satisfy all stakeholder requirements.

A cloud-based Software as a Service (SaaS) solution to collect patient reported outcomes data or to capture clinical outcomes assessments, conduct remote telehealth or video visits in lieu of in-person visits can enable both children and their parents to participate in the study from home. A well-designed SaaS solution that can combine all these digital tools into a single login can save significant time and costs of IT infrastructure and hardware. All these are bound to bring significant speed and cost efficiencies to clinical operations.

Dr. Rajasimha is the founder and CEO of the decentralized clinical trials software company, Jeeva Informatics Solutions, based in Virginia. He is a precision medicine data scientist-turned social entrepreneur on a mission to accelerate human-centric clinical research through technology innovation and global advocacy. He is the founder and chairman of the humanitarian non-profit Indo US Organization for Rare Diseases. Earlier, he served as cofounder and co-chair of the Organization for Rare Diseases in India from 2013 – 2019. He is academically affiliated as a faculty in the School of Systems Biology at George Mason University in Fairfax, VA since 2012.

Dr. Rajasimha has more than a decade of experience working on various interdisciplinary projects involving genomics and big data as a consultant for clients including National Cancer Institute, National Eye Institute, Georgetown University, and Genome International Corporation. His research has focused on the genomics and systems biology of diseases including cancer, infectious diseases, neuro-muscular diseases, and retinal degenerative diseases. He completed his M.S. in Computer Science and Ph.D. in Genetics, Bioinformatics and Computational Biology at Virginia Tech, where he developed and applied reusable simulation models of mitochondrial DNA heteroplasmy dynamics to study various diseases.

About Jeeva Informatics

The personal experience of losing a child born with a rare disease and a brother with a chronic disease became the springboard for Dr. Harsha Rajasimha to apply his years of postdoctoral training at NIH and FDA to accelerating therapies for rare and common conditions. He knew that technology in itself is not the limiting factor and that patient-centered design guided by stakeholder needs and regulatory requirements would guide their continuous learning digital platform. By digitizing and automating manual repetitive tasks and reducing the logistical burdens on patients and study teams by over 70%, Jeeva accelerates the process of bringing new medicines or vaccines to patients who need them by over 3x faster. The Virginia-based company’s modular software-as-a-service platform is fully scalable and facilitates patient enrollment, engagement, and evidence generation in clinical trials on any browser-enabled mobile device. Visit https://jeevatrials.com/

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