Around 50% of the drugs approved for adults in the US are being used in children, even though their safety and effectiveness had not been established for this use. Last week, FDA announced further measures to help increase the number of paediatric trials in the US to help address this.
There are already two US laws specifically aimed at encouraging paediatric drug trials - The Best Pharmaceuticals for Children Act (BPCA) and The Pediatric Research Equity Act (PREA). Before these laws came into effect, as many as 80% of adult drugs were being used in children without their effects being tested in paediatric trials.
The BPCA grants an extra six month marketing exclusivity, whereas the PREA requires drug companies to study their products in children under certain circumstances.
Paediatric studies have their own highly defined rules within good clinical practice guidelines – especially in terms of informed consent. The following is taken from the US version of our ICH GCP online course:
“These subjects may have consent given for them by their legal representative, however, the subject should be given information to the fullest extent of their understanding. If possible they should sign and date the consent.
Consent forms should be tailored to the age of the patient even if this requires one trial to have several different forms. Each form should be approved by the local IRB/IEC. Minors approaching adulthood may review and sign the consent forms themselves but for younger patients, their parent or guardian should review and sign the forms and the child should review and indicate their assent (in writing where possible) to the trial.”
In some cases, FDA has allowed sponsors to defer paediatric studies, depending on the circumstances. However, deadlines for deferred studies have often been missed.
If a sponsor does not have a deferral extension, has missed the deadline for submission of deferred paediatric studies, or has not requested approval for a required paediatric formulation, FDA can now send a non-compliance letter and publish the letters on the web.
Last week the FDA published the first letters together with sponsor responses.
A recent study linking use of antipsychotics in children with type 2 diabetes mellitus shows how crucial this issue is. The article, published in JAMA Psychiatry, indicated a three-fold increase in incidence of diabetes amongst children prescribed the drugs.
This was particularly alarming not only because of the availability of other treatments for ADHD, but also because of the disproportionate use of the drugs to treat the poorest patients through Medicaid.
With adequate paediatric trials, the risks could have been better understood and prescribing limited to only those cases where the benefits outweighed potential harm.
Let’s hope that the FDA’s measures achieve their aim of protecting the most vulnerable patients in the US.
I'd love to know what you think.
Blog Author: Dr. G.C. Practice
