Tougher rules likely as pharma is rapped by European Parliament for pediatric trials record


The European Parliament is calling for a shake-up in legislation to make it harder for pharma companies to duck obligations on testing medicines in children.

European lawmakers voted through a resolution asking the European Commission to draw up—as a matter of urgency—to revise the Pediatric Medicines Regulation (PMR) to make it harder for drugmakers to sidestep the 10-year-old requirement to test most new medicines in children.

As it stands, pharma companies have a legal obligation to test all new medicines in children as well as for adults through a pediatric investigation plan (PIP), unless they obtain an exemption or waiver. In return they can claim additional market exclusivity for the new drug.

The new resolution is asking for additional incentives for companies that carry out trials of new drugs in children—at the moment those are unspecified but could take the form of tax breaks—but potentially also penalties for companies that sidestep their obligations. The MEPs say there is a particularly pressing need to boost testing of new medicines for childhood cancers and for newborns.

The European Society for Pediatric Oncology (SIOPE) is among the charities pushing the European Parliament to introduce measures to accelerate the development of safe and effective anticancer therapies for children and adolescents.

SIOPE and other charities including Cancer Research UK and Unite2ure are unhappy that drug companies can ask for a waiver from the obligation to investigate the potential benefit of a drug in a child if the adult cancer for which the drug was originally developed does not exist in children.

They maintain such drugs may still be used to treat other childhood cancer types, and welcome the new resolution as it seeks specifically to plug that loophole. The number of waivers being awarded is rising year-in-year, according to the MEPs, and there is nothing to stop a company pulling a trial in children if it doesn't work in adults.

"There is still not enough research, not enough drugs and not enough options," said patient advocacy group Unite2Cure in a statement.

The MEPs agree, with the resolution noting that "fewer than 10% of children with a non-curable life-threatening relapse have access to new, experimental drugs in clinical trials from which they could benefit," and only two targeted anticancer drugs have been approved based on a paediatric investigation plan (PIP) since the PMR came into force.

"We call on the European Commission to revise the 2007 Regulation on Paediatric Medicines and introduce measures facilitating the testing and developing of new drugs that could potentially be beneficial for children and young people," said the charities.

The MEP resolution asks the Commission to deliver a report on the PMR, including an analysis of the economic impact of the rewards and incentives, and consider revising the legislation.