8:19 PM
Nov 01, 2018
 |  BC Innovations  |  Targets & Mechanisms

FDA’s pediatric target picks

FDA looks beyond company pipeline horizons for pediatric oncology targets

FDA is looking ahead of industry in its guideline on targets for pediatric oncology, which includes more than 50 not yet in company pipelines in a list of over 220 the agency has its eye on. The list details molecular targets that could trigger a requirement to conduct pediatric trials, and suggests the agency sees potential in unexploited territory, in particular in gene regulation.

The list was published Oct. 16, following a series of discussions with National Cancer Institute and other stakeholders, including a February workshop hosted by Friends of Cancer Research, which identified molecular targets or groups of targets that had been linked to pediatric cancers in published papers or company development programs.

The guidelines are part of a push by FDA and regulators worldwide to accelerate pediatric drug development, which typically lags years behind development of drugs for similar adult indications (see “Pediatric Push”).

In oncology, the problem has been exacerbated by the Pediatric Research Equity Act (PREA), because it exempts Orphan diseases -- which include all pediatric cancers -- from requirements to carry out pediatric studies on drugs in development for adults.

Last year, Congress took steps to close the gap through the FDA Reauthorization Act of 2017 (FDARA). Part of the law, the Research to Accelerate Cures and Equity (RACE) for Children Act, amended PREA by excluding pediatric cancer drugs from the Orphan exemption and requiring sponsors developing molecularly targeted therapies for adult cancers to conduct trials in pediatric cancers that share the targeted molecular characteristics. The amendment also required FDA to generate a list of targets relevant to pediatric cancers.

Gregory Reaman, associate director for oncology sciences at FDA, said the list wasn’t the agency’s idea. “This was legislated solely because of efforts of PhRMA and BIO, which sought some regulatory certainty,” he told BioCentury.

“Whether there was an agent under development would certainly increase the potential relevance, but there was no requirement.”

Gregory Reaman, FDA

Reaman said maintaining an up-to-date list is impractical, given the rapid pace of breakthroughs in cancer biology. “That was not something the agency felt it...

Read the full 1728 word article

User Sign in

Trial Subscription

Get a 4-week free trial subscription to BioCentury Innovations

Article Purchase

$100 USD
More Info >