EMA and FDA harmonise over development of Gaucher drugs

The EMA and the FDA have announced that they have developed a joint proposal to promote more efficient means of bringing through treatments for Gaucher disease. The proposal may serve to form a template for future drug development within rare diseases, if it were adopted and proved successful.

The two major strategies promoted by the agencies are:

The extrapolation of available clinical data, including through appropriate modelling and simulation techniques, to predict how a medicine may work in children and adolescents on the basis of studies conducted in adults or other paediatric populations

The possibility to test the safety and efficacy of medicines developed by different companies in one single trial, so-called multi-arm, multi-company clinical trials. As the same control arm is used to compare more than one medicine under evaluation, this approach facilitates the clinical testing of medicines while reducing the total number of children included in trials

The latter of the two points could lead to the saving of both money and time in the development process of the drugs for the disease. The use of multi-company trials means that the control arm could be reduced in number, meaning that fewer children with the disease would be needed and ethically it would reduce the amount of children, and their families, going through potential placebo treatment.

Gaucher disease is an ineherited disorder that affects the body’s organs and tissues, characterised by the deposition of glucocerebroside in cells of the macrophage-monocyte system.

 The collaboration between the EMA and the FDA can be recognised as a means of speeding through rare disease area treatments, which are currently a problem area. The agencies mention this being a potential method to promote development across all rare diseases.

The costs of current Gaucher disease treatments, for example, can be an average of $300,000 per year in the US. If the cost of development of new drugs is lower and speed to market is quicker then, in theory, the cost of medicines should be lower.