EMA in push for more paediatric drug trials
pharmafile | July 24, 2015 | News story | Medical Communications, Research and Development | EMA, European Medicines Agency, clinical trials, drug development, paediatrics
The European Medicines Agency has relaxed some of its rules around the development of drugs in children, in order to “stimulate the exploration of the use of these novel products in children.”
Under new rules to come into force in 2018, manufacturers of certain classes of drugs will need to submit development plans in order to study whether these classes of drugs are effective in paediatric groups. The European Regulator hopes the ‘lengthy and comprehensive review’ that led to the changes will prompt companies to revisit eligible classes of drugs’ potential use in children.
Since the Paediatric Regulation came into force on 26 January 2007, all companies that are making applications for marketing authorisation for new medicines have had to submit a paediatric investigation plan (PIP) early in the development of the medicine, describing how the medicine should be studied in children.
This requirement also applies when a company wants to add a new indication, pharmaceutical form or route of administration for a medicine that is already authorised and patented.
As it is not always appropriate to develop certain medicines in children, the EMA can waive the requirement to develop a class of medicines in children, called a class waiver, meaning the developer is not required to submit a PIP.
But some critics argue that this is one of the reasons for the low number of clinical studies involving children. For example in oncology, between 2012 and 2014, only 14 of the 26 new anticancer medicines authorised for use in adults had a development plan for use in children.
And due to fears at the EMA that the class waiver may be stifling the possible use of treatments in children, European regulators have reviewed the list of drugs that are eligible for class waivers and made ‘extensive revisions’. The Agency hopes this “will encourage companies to develop more new medicines for use in children.”
The EMA’s Paediatric Committee (PDCO) has reviewed the drugs that are eligible for a class waiver. It concluded that “the current class waiver list results in insufficient opportunities for the PCDO to consider the potential benefits of some new medicines for children”, the report states.
Following the review, the PCDO has revoked eight classes, updated a further 15, and retained nine in the current class waiver list. Classes of drugs have been revoked “on the grounds that the concerned condition does occur in the paediatric population, and clinical studies may fulfil a therapeutic need of the paediatric population.”
Dr Jordi Llinares, head of product development scientific support at EMA, says the changes “will allow new promising medicines to be assessed for use in children.”
“We have to keep our obligations in balance”, he says. “We want to encourage dialogue between pharma companies and regulators about the potential use of some classes of drugs in children in future, but without unnecessarily exposing children needlessly to clinical trials.”
Dr Koenraad Norga, vice-chair of the EMA’s paediatric committee, adds: “If we are revoking or revising waivers then we are telling clinical trial sponsors that they have to come to the PDOC and discuss the future development line for that product in children.
“Our hope is that it will trigger more development and a broader discussion about drug development. We are responding to the science around new molecular targets – the more broadly we can talk, the more options there are for discussion about ongoing development.”
The updated class waiver list will come into effect in 2018, but Dr Norga says the EMA is keen for companies to begin their development conversations with the regulator as soon as possible. “Any changes in our work will take a few years to become apparent to the outside world. But we hope it will feed into companies’ plans for new paediatric products.”
Lilian Anekwe
The eight classes for which class waivers no longer apply are:
- Medicines for treatment of liver and intrahepatic bile duct carcinoma
- Kidney and renal pelvis carcinoma
- Coronary atherosclerosis
- Peripheral atherosclerosis
- Vascular dementia and vascular cognitive disorder / impairment
- Non-juvenile Parkinson disease
- Huntington chorea
- Amyotrophic lateral sclerosis.
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