Gaucher disease is a rare genetic disorder characterized by the deposition of glucocerebroside in cells of the macrophage-monocyte system. The disorder results from the deficiency of the enzyme glucocerebrosidase. No binding strategic collaborative approach document is published by European Medicines Agency (EMA) to facilitate paediatric drug development, particularly in the field of Gaucher disease. Document initially explained the current paediatric practices for treatment of disease and discussed the unmet medical and clinical therapeutic needs. The EMA and the U.S. Food and Drug Administration (FDA) mentioned that this proposal only applies to non-neurological manifestations of Gaucher disease in children with Type I and Type III who have not received previous treatment. However, principles underlying this proposal may be extended to other areas of drug development in rare diseases.
Document illustrated that further research can be considered for the following key points:
Extrapolation of efficacy: Data extrapolation can be useful if course of disease and expected response to medicinal product is similar from one type of population to another population. This way results could be predicted from studies including adults children or from children of one age group to children of another age group. Both EMA and FDA recognise that the use of extrapolation of efficacy in paediatric Gaucher disease can avoid unnecessary studies, increase efficiency, reduce testing burden to patients, and better allocate the resources to address relevant questions.
Multi-company trials: 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, is a factor that restricts the pace of drug development. This approach will help in reducing the total number of enrolled subjects in comparison to controlled trials, because a single control arm can be used to assess the effects of more than one drug product.
Document mentions at one point that there are differences in the regulatory requirements of both Europe and the United States, particularly regarding extrapolation of efficacy from adults to children. Any approach to be followed for any upcoming trial shall be discussed with specific regulatory agency and agreed upon. Applicant can propose different approaches with proper justification. EMA has also published a draft paper on extrapolation of efficacy and safety in paediatric medicine development (Link: http://www.ema.europa.eu/docs/en_GB/document_library/Regulatory_and_procedural_guideline/2016/04/WC500204187.pdf )
In addition, EMA is planning to publish a reflection paper, which outlines a systematic approach to scientifically sound and reliable extrapolation of data to support medicine authorisation. This paper is expected to be published in the fourth quarter of 2017 to complement the approach published in this approach document. As per this news release by EMA, FDA will also publish a strategy paper in different format in next few months.