Friday, January 2, 2015

Uncertain Health in an Insecure World – 17

“Children Are Not Small Adults”

Dr. Peter Moore so titled his 1998 Lancet article about the under-regulation of prescription drugs for children. Children are classified as “vulnerable individuals” in many countries.

But until quite recently, the actual scope of that vulnerability within developed country health care systems has been under-appreciated.

What exactly has been threatening the world’s most vulnerable patient cohort – our children?

Over the past decade, clear evidence has emerged about the risks related to the paucity and poor process of pediatric clinical drug trials.

A 2008 Lancet review of 7-years of pediatric clinical trials data showed that only 2% (13/740) of research trials involving children had an independent data safety & monitoring committee.

A 2008 PLOS Medicine article reported that over half of the drugs used in hospitalized children are either used off-label (“compassionate” use) or are unlicensed for use in children. Drug dosing & pharmacological effectiveness in children is often incorrectly extrapolated from adult drug trials.

In 2010, Dr. John Ioannidis, Stanford University disease prevention advisor to countries around the world, was named a ‘brave thinker’ by The Atlantic magazine, after calling for better reporting of the ‘harms’ to children participating in pediatric drug trials.  

A 2012 Pediatrics study showed that despite the high burden of childhood diseases (59.9%), only 12% (292/2440) of all clinical trials involve pediatric populations. Existing pediatric trials are usually restricted to cancer drugs and vaccine development, and are most frequently funded by governments or non-for-profits. Lucrative adult drug trials are more often corporately funded by ‘Big Pharma’. 

In November 2014, Dr. Martin Offringa of Toronto Sick Kids Hospital acknowledged that these "failures are now on the table, rather than under the table..."

To those of us more familiar with adult drug and medical intervention trials, all of this information is frankly shocking!

The overdue global response to these mounting concerns has been impressive.

International experts are actively mobilizing effective pediatric clinical research networks:
•        StaR Child Health Network (est. 2012) – U.S., Canadian, Australian, European and Asian experts aid in the development of standards for the design, conduct and reporting of pediatric trials.

•        Eunice Kennedy Shriver National Institute of Child Health & Human Development Pediatrics Trials Network (est. 2012) – now operational in the U.S., and actively seeking global partners.

•        Global Research in Pediatrics (GRIP, est. 2011) – a European Union (EU)-funded pediatrics clinical trial resource for on-line tools and expertise in pharmacology, trial sample size calculations, data safety & monitoring committee creation linking >1,000 GRIP institutions worldwide.

•        National Institute for Health Research medicines for children research network (MCRN, est. 2006) – guides U.K. researchers in designing better pediatric clinical trial information leaflets; MCRN now actively involves children and young people in this process.

So - has this child-unfriendly clinical drug trials riptide turned?

No - not completely.

However - greater global medical community awareness of the need to reduce the risks & harms to vulnerable children from poorly designed clinical trials is now properly front & center.

And with these small steps, sick children may soon run more safely in The Square.

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