Healthy Skepticism Library item: 18690

Warning: This library includes all items relevant to health product marketing that we are aware of regardless of quality. Often we do not agree with all or part of the contents.

Publication type: news

Hassner Sharav
Globalization of Pediatric Drug Trials--For Whose Benefit?
AHRP 2010 Aug 26
http://www.ahrp.org/cms/content/view/721/9/

Notes:

Globalization of Pediatric Research: Analysis of Clinical Trials Completed for Pediatric Exclusivity
http://pediatrics.aappublications.org/cgi/content/abstract/126/3/e687

Full text:

There is no evidence to indicate that children have been the beneficiaries of the Pediatric Exclusivity Provision of FDAMA (enacted in 1998): there is evidence that pharmaceutical companies have increased their profits from the legislation by at least $14 billion.

A Duke University study published in the journal Pediatrics (abstract below) examined the effects of the Pediatric Exclusivity Provision of FDAMA (enacted in 1998). FDAMA provides six months of patent exclusivity to pharmaceutical companies to conduct safety and efficacy studies of drugs in children.

The findings confirm that Big Pharma is conducting most pediatric drug trials offshore—including developing or transitioning countries in Eastern Europe, Asia, Africa, and South and Central America—where ethical standards, oversight, and legal protections against children’s exploitation are least reliable (if they exist at all).

Of the 174 published trials that indicated the study location, 65% were conducted offshore, 38% were conducted in a developing country, and 11% did not include any sites in the US.

The authors note that “It’s much cheaper, easier, and less time consuming to conduct research outside the U.S.”
Indeed, they found that the Pediatric Exclusivity Provision has led to an estimated $14 billion in profits to pharmaceutical companies.

The authors raise concerns about the validity of 150 pediatric label changes that (presumably) resulted from these offshore trials:
“The original objective of the program was to encourage research that enables the FDA to label drugs for appropriate use in children in the U.S.,” says Dr. Sara Pasquali. “Whether it’s valid to extrapolate the results from trials conducted in other countries is not known. The efficacy of a medication may depend on genetic background and access to health care resources, among other factors, which may differ across countries.”

And they note the ethical issue of exploitation of poor children in trials testing products they will not have access to.

The authors put forth several recommendations for reforming the conduct of pediatric trials conducted outside the United States. These include:

Requiring pharmaceutical companies to describe how the proposed study population matches the intended market for the drug being tested
Requiring all studies conducted under the program be published and include specific data regarding the setting and location of the trial
Improving mechanisms of global regulatory oversight with input from all stakeholders

We would add an additional requirement: that FDA not accept data from pediatric trials conducted at sites not inspected by the agency during the course of the trial.