The EPHA member organisation Heart EU recently released a statement on the paediatric medicines regulation (COM 2004-599). Heart EU is a an-European patient alliance with a focus on risk prevention and management of inherited high cholesterol, (otherwise known as familial hypercholesterolaemia or FH.
On behalf of the European FH community, HEART EU pleads that the example of the US be urgently followed by the introduction through necessary trials for the earliest safe introduction of these drugs for FH children in Europe, as about 95% of them are presently denied access to safe and effective medication that prevents early death.
