The US Food and Drug Administration has published draft guidance for clinical investigators on the agency's review process for drug trials involving children.
Under the draft guidance, institutional review boards are obliged to review clinical trials that employ children, and approval should only be given if they meet a detailed risk-benefit assessment and consent is obtained from children, as well as their parents or guardians.
However, the FDA suggests that these requirements could be set aside, provided the trial’s institutional review boards could demonstrate that the clinical trial would help ‘to understand, prevent or alleviate’ a serious childhood health disorder. The FDA Commissioner's approval would also be required in such cases, to ensure that ethical practices are followed.
Requests for FDA approval of a clinical trial involving children would be reviewed at a public meeting of the Pediatric Advisory Committee, which recommends a decision to the Commissioner. The latter's decision would be final.
The move is part of an ongoing effort by the agency to encourage appropriate studies in children, a population that has historically been under-represented in clinical testing. This has led to a situation where drugs have been used in children without the same level of evidence as has been obtained in adults.
In 2002, the USA updated existing legislation that encourages drug companies and other organisations to carry out pediatric studies on medicines by granting an additional six months’ marketing exclusivity to in-patent medicines. But this has led to an increase in the number of trials being carried out in children, and the FDA has decided that, as a result, it needs to ensure that such trials are carried out responsibly.
One high-profile case which has been highlighted as evidence that better safeguards are needed involved children in foster care in the USA who were enrolled into clinical trials of HIV/AIDS drugs from the 1980s to 2002, in some cases, it has been suggested, without proper informed consent.
The BBC aired the story in 2004, and an official investigation onto the case by the non-profit Vera Institute of Justice got underway in early 2005. This ongoing review is expected to reveal how medical decisions were made for the children, the consent process for clinical trials, and the child welfare outcomes for children and families. Last month, Vera said the investigation would take several more months to complete.