Please login to the form below

Not currently logged in
Email:
Password:

EMA and FDA calls for innovative approaches for Gaucher disease medicines

The regulatory agencies also urge a similar method for all paediatric diseases

FDA EMARegulators in the US and Europe have outlined a series of proposals, including multi-arm clinical trials, in a bid to improve research into new, innovative treatments for the rare genetic disorder Gaucher disease. 

The joint proposal from the European Medicines Agency (EMA) and the US Food and Drug Administration (FDA) also urges the use of innovative methods in developing medicines for diseases in children in general, and encourages medicine developers to extend its clinical data through modelling and simulation techniques.

The agencies are also encouraging the use of multi-arm clinical trials to test the safety and efficacy of medicines developed by different companies in one single trial.

The proposal aims to 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,” the EMA said in a statement.

In addition, the EMA is finalising a reflection paper - expected to be published in the fourth quarter of 2017 - that outlines an efficient approach to allow reliable extrapolation of data to support medicine authorisation.

Article by
Gemma Jones

4th July 2017

From: Regulatory

Share

Tags

COVID-19 Updates and Daily News

Featured jobs

PMHub

Add my company
fox&cat

up to your ears in it? some pressure is helpful but too much of the wrong sort quickly takes its...

Latest intelligence

Sharing patient stories for World Pulmonary Hypertension Day
For World Pulmonary Hypertension Day and we’re here to help raise awareness of pulmonary hypertension (PH) - a frequently under and misdiagnosed condition. Created in collaboration with the PH patient...
How nature can help you manage the pressures of agency life
Paul Hutchings, founder of fox&cat, outlines how nature can help you can boost staff wellbeing in your agency this Mental Health Awareness Week...
How can we strike the right balance between familiarity and innovation when it comes to data presentation?
Following our webinar in March, Getting MedComms right: navigating the age of the amateur expert, we’re taking the time to respond to questions we were unable to answer during the...