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Getting Approval For Drugs And Devices In Pediatrics – Why Is It So Challenging?

Published: 7/20/14
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By Geoffrey Martello

Twitter summary: Why is it so difficult to approve new pediatric drugs & devices? The importance of clinical trials, and how study design affects products …

Short summary: Have you ever wondered why there are fewer options in drugs and devices for children with diabetes than there are for adults? Pediatric clinical trials face several unique challenges, and solving them often requires some creative strategies. Dr. Rick Turner proposed a few at ADA this year.

Have you ever wondered why there are fewer options in drugs and devices for children with diabetes than there are for adults? For that matter, why do treatment options take significantly longer to come to market than their counterparts for adults? The answer lies in the difficulties of designing and completing clinical trials for children – a subject discussed by Dr. Rick Turner at the ADA conference in June.

The biggest hurdle to approving new drugs and devices is often the length and cost of conducting trials. Before submitting a new therapy to the FDA, companies have to conduct clinical trials to assess the action, effectiveness, and safety of the drug. These studies can involve tracking up to thousands of patients, often spanning many years and costing many millions of dollars. As such, studies for adults are huge undertakings, so companies often won’t allocate the resources for trials for children.  To learn more about the drug development and regulatory process, please read our learning curve on this topic.

A pediatric trial cannot simply be a repeat of an adult trial with children and adolescents as the subjects; designing trials for younger patients faces unique challenges. For example, infants, children, and adolescents are all classified together as pediatric patients, even though each group has unique physical, cognitive, and psychological characteristics that have to be taken into account. Biological changes during puberty can also make it especially difficult to study pediatric populations.

Recruiting children and teens to participate in trials can be challenging; oftentimes parents or guardians don’t know how to have their children participate in clinical trials or do not know about trial opportunities. For parents and children looking to get involved, JDRF has a valuable website, Clinical Trials Connection, to find out about trials for type 1 diabetes, and TrialNet is also an excellent resource for type 1 diabetes. Unfortunately, the United States’ official website, ClinicalTrials.gov, is notoriously hard to use.  That’s why we publicize these opportunities every month through our  trial watch.

Dr. Turner said that clinical trials should have broader inclusion criteria to encourage more participation. Relatively minor changes, such as widening the A1c window and the age limit, can allow many more people to participate.

Clinical trials, in short, are the key to developing new drugs and devices, and we must reconsider how we use them for children. 

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