European and U.S. regulators have released a white paper on ways to develop innovative therapies for Gaucher disease that could also apply to Batten disease and other rare children’s disorders.
One recommendation is that scientists use clinical-trial data on how a drug works in adults to help them predict how it will work in children and adolescents.
Another recommendation is that drug developers use multi-arm clinical trials to compare the safety and effectiveness of more than one drug at a time.
These recommendations will “reduce the number of patients needed for clinical trials, meaning overall less burden on children and their families, while maintaining high quality standards for medicine development,” according to the European Medicines Agency webpage.
The regulators drew up the drug-development principles to address Gaucher disease, a genetic disorder whose severest forms include neurological symptoms. But the recommendations can apply to other children’s diseases, too, they said.
The paper encourages those developing treatments to ask the European Medicines Agency, the FDA, or both, for scientific advice as they carry out their work.
“Due to differences in the regulatory requirements of both Europe and the United States, particularly regarding extrapolation of efficacy from adults to children, additional trials may be required to support an application for approval,” the document said.
The European Medicines Agency will publish a paper that compliments the white paper later this year. It will explain how researchers can develop “scientifically sound and reliable extrapolation of data” to support requests for regulatory approval of therapies. The FDA will publish the paper in a different format in the next few months as well.
Batten disease is a fatal hereditary disorder affecting the nervous system that typically begins in childhood. The first symptoms include vision problems or seizures. As it progresses, children develop cognitive impairment, worse seizures, and progressive loss of sight and movement.