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Management of Paediatric Clinical Trials Calls for Urgent Technological Innovation

Changes in legislation, increased interest in pediatric studies by the pharmaceutical industry, and a renewed interest in rare diseases have increased the demand for clinical trials (CT) of investigational medicines for children. Nonetheless, pediatric clinical trials continue to have a very high failure rate. Many of the problems affecting pediatric clinical trials, says Dr. Harsha Rajasimha, founder and CEO of Jeeva Informatics and Founder Chairman of the Indo US Organization for Rare Diseases (IndoUSrare), have to do with the way trial participants are recruited and managed. “You cannot conduct a pediatric CT the same way you would with adults.” says Dr. Rajasimha. “Fortunately, improvements in both understanding and technology are enabling a better process—which, in time, should lead to better results.”

One persistent challenge in pediatric CTs, notes Dr. Rajasimha, 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. Perhaps the most satisfactory solution, Dr. Rajasimha says, is telehealth: enabling both children and their parents to participate in the study from home, via today’s now ubiquitous digital platforms.

“It is really unfair to expect patients and their support-givers to visit a hospital or clinic when it is really not necessary, especially when these tasks can be effectively and safely completed from the comfort of where they live,” says Dr. Marshall Summar, Director of Clinical Research at the Rare Disease Institute of the Children’s National Medical Center and Chairman of the National Organization for Rare Disorders (NORD).

In addition to minimizing the travel requirement, digital engagement with patients and caregivers and management by clinical researchers, says Dr. Rajasimha, offers a continuous level of record-keeping and reporting over the course of the CT. This is particularly important in pediatric CTs, he says, because in some cases they can take years, during which time the growing participants will undergo physiological changes, as well as possible changes in their reaction to the treatment being tested.

Pediatric rare diseases

The challenges facing pediatric CTs, notes Dr. Rajasimha, are particularly severe in the case of rare diseases that affect only a small subset of the population. There are over 7,000 distinct rare diseases in the world today, affecting about five percent of the overall population—30 million Americans, 400 million people worldwide. Ninety-three percent of these rare diseases do not have any FDA-approved treatment yet. Of those affected by rare diseases, fifty percent are children, three out of ten of who will not live to see their fifth birthday. Meanwhile, the time it takes for a patient to get an accurate rare disease diagnosis is six to eight years.

On the last day of February, the world observes Rare Disease Day, which was established in 2008 to raise awareness of rare diseases and improve access to treatments for them. This is, says Dr. Rajasimha, a date important to him on several levels. On the professional side, Jeeva Informatics has developed an eClinical cloud platform for CT management that facilitates remote patient screening, education, enrollment, electronic informed consent, and other CT functions, all accessible from any browser-enabled mobile device.

On a personal level, a decade ago Dr. Rajasimha and his wife lost their daughter to a rare genetic disease. At that point he decided to take his post-doctoral work at the National Institutes of Health, and his experience with the FDA, and invest them in a quest to accelerate the development of therapies for rare and common disorders. It has been, and remains, his goal and that of his company to save lives globally by simplifying the clinical trial operations.

“A consultative and collaborative human-centric approach is critical in accelerating clinical research. Digital access to CTs represents the next generation of clinical research and patient engagement solutions that were long overdue, especially in pediatric clinical trials,” adds Dr. Summar.

Pediatric CTs, Dr. Rajasimha points out, are perhaps the most challenging area of treatment development. For one, the risk is higher. Children have a lower risk tolerance to drugs, and the trial can affect their lifespan. For another, as noted, there are complex logistical problems in simply coordinating children and their guardians’ schedules. Finally, the length of the trial is subject to a child’s growth. For instance, there can be a significant difference in a one-year-old and the same child at age six.

“While individually these diseases are rare,” says Dr. Rajasimha, “the experience of being struck by them—or having a family member so affected—is all too common. I personally, and we as a company, believe that to combat these diseases—all diseases, actually—there is an urgent need for remote eConsent, screening, management of the active period of clinical trials, and safety follow-ups, and we are dedicated to providing the best and most effective human-centric digital solutions to it.”

Connect with Dr. Rajasimha and other stakeholders of rare diseases at the virtual Rare Disease Day event hosted by IndoUSrare on the 28th of February 9am to 11am EST to listen to experts from both the USA and India.

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