Regulatory agencies in the U.S. and Europe have joined forces to develop a proposal with the aim to spur innovative approaches in the development of new Gaucher disease treatments.
The European Medicines Agency (EMA) and the U.S. Food and Drug Administration (FDA) underscored that the proposal only applies to non-neurological manifestations of Gaucher disease in children with Type I and III who have not received previous treatment.
The strategy document, however, also can be applied more broadly to rare diseases affecting children, they said.
The proposal highlights research advances in the area of data extrapolation — taking data gathered from one type of patient or conditions and applying it to another group. The two agencies argued that using appropriate modeling and simulation methods, it might be possible to predict how a medicine could work in children and adolescents by looking at studies including adults or children with other diseases.
The agencies also promoted the development of clinical trials in which several pharmaceutical companies work together to test several treatments. Recruiting enough participants for clinical trials in rare diseases, such as Gaucher's, is a factor that restricts the pace of drug development.
Since only one control group will be needed to compare the effectiveness of several drugs, this type of trial could speed up drug development. It also should ease the burden of trial participation on Gaucher-affected children and their families, while keeping drug development quality standards high, the agencies argued.
EMA and the FDA encourage drug developers to seek advice with the agencies if they plan to use these approaches in their development of new Gaucher therapies.
The two agencies also work separately to publish parts of the proposal as scientific articles. EMA focuses on a systematic approach, allowing drug developers to extrapolate data in a scientifically sound and reliable manner. This will make it easier for drugs to pass regulatory review.
The article, which EMA plans to publish by the last quarter of 2017, will complement the already presented document. The FDA will publish a strategy article of their own, in a different format, according to an EMA press release.
The strategic document was the result of collaborations involving not only the EMA and the FDA, but also patients and healthcare professionals.