FDA Releases Draft Guidance on Clinical Pharmacology in Pediatric Studies

Sponsors submitting pediatric study plans for a drug have several options to determine optimal dosing for children when no standard dosing information exists, according to an FDA draft guidance on clinical pharmacology in pediatric studies.

If the disease course and treatment are similar in adults and children — but there is no consensus on pediatric dose-response data — sponsors may extrapolate from well-known adult data to children using biomarkers characteristic of adult drug responses, said the guidance released last week.

When disease courses are unique in children, sponsors should evaluate multiple doses across multiple pediatric studies, using population data from efficacy studies to determine the best doses for different age groups, the agency said.

When there is reasonable evidence that a disease progresses similarly in adults and children and that both respond similarly to treatment, sponsors should test drug concentrations in blood, urine, tissue or cerebrospinal fluid samples.

Companies may administer single or multiple doses of a product to a small number of patients with frequent collection of blood and urine samples, which are then tested for drug concentrations.

Alternatively, they can collect samples less frequently from a larger number of patients. This is ideal for children, the FDA says, because they would be subjected to fewer needle pricks. It also allows sponsors to estimate a drug’s effects both in a wider population and in more individual patient groups.

Sponsors should discuss the number of patients in each age range with the FDA, as that is often specific to each product.

The guidance, the first to deal with clinical pharmacology in pediatric studies since 1998, comes as the FDA in recent years has offered new incentives, such as added marketing exclusivity, to address a lack of pediatric testing and use information in product labeling.

Comments on General Clinical Pharmacology Considerations for Pediatric Studies for Drugs and Biological Products are due to regulations.gov, docket no. FDA-2013-D-1275, by Feb. 7.

View it at www.fdanews.com/12-08-14-pediatricguidance.pdf. — Lena Freund

Originally appeared in Drug Industry Daily, the pharmaceutical industry’s number one source for regulatory news and information. Click here for more information.