In its first full year of operation, the work of the new pediatric medicines committee at the European Medicines Agency (EMEA)
in London has led to the formal approval of only 15 pediatric investigation plans (PIP), according to data recently released
by the European Union.
Peter O'Donnell
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As many readers of this column are aware, since the start of 2007 there has been a new European rule requiring all European
marketing authorization applications to be accompanied by an outline of how the product is to be tested for use in children
or by a compelling argument for a waiver of the obligation. Of the 233 applications the committee received in the last 12
months, 49 were requests for a full waiver for all conditions and all subsets of the pediatric population, covering some 420
indications. The committee adopted 71 opinions: one was negative, 39 were approvals of PIPs, and 31 were approvals of full
waivers.
The committee's opinions are then given formal status as decisions of the EMEA in London—a process that takes additional time.
So by a coincidence, in formal terms, at present there has been approval of 15 PIPs and 15 waivers.
On July 28th the first inventory was published of the results so far of the EU scheme to boost research for medicines for
children. It also lists the rewards being offered at the EU and national level, over and above the six-month extension to
patent protection. The specially favorable conditions for orphan medicines (which include two additional years of market exclusivity)
attracted around 40 applications, and three-quarters of these provided a full PIP with no request for any waiver. Forty-plus schemes
The inventory reveals that by July of this year there were more than 40 different schemes in operation in Europe to promote
research into medicines for children. At the EU level, they include—in addition to the operation of the pediatric medicine
regulation—a range of EU-funded research programs. Among the most advanced are a project for selecting and validating drug
targets from the human kinome for high-risk pediatric cancers, the development of a European embryonal tumor pipeline, work
on chimeral T cells for the treatment of pediatric cancers, and a task force for drug development for the young population.
Funding has also been granted to a project entitled Relating Expectations and needs to the Participation and Empowerment of
Children in Clinical Trials. The EU is also finalizing a proposal to promote childhood immunization, which will urge member
countries to step up efforts to achieve high childhood vaccination coverage for priority vaccine-preventable diseases.
Breaking it Down: PIPs and Waivers
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There are also numerous national schemes in the member states to promote research into children's medicines. Austria offers
financial grants and scholarships and, along with Bulgaria, tax breaks. In the Czech Republic, the ministry of health is preparing
a program for 2009–2011 that includes pharmacological and pediatric research, focused on improvement of health care for young
people, new therapeutic procedures, and other contributions in the field of pediatric health care provision.
In France, experts from the National Medicines Agency provide scientific advice free of charge for the development of potential
pediatric medicines, and the National Health Agency is creating networks of pediatric investigators. The Agency has also introduced
controlled systems for access to medicines without market authorization in France. Preauthorization access can be arranged
for patients with serious or rare diseases (a quarter of which are children, says the Agency).
Steps are also underway to allow access to medicines authorized elsewhere in the EU but not in France. And if a suitable proprietary
medicinal product is not available, hospital pharmacies are authorized to produce their own preparations, which are then declared
to the Agency—and nearly half of all medicinal preparations are for pediatric use.
Italy has established a pediatric working group to coordinate all issues related to pediatric drugs. Spain has made a priority
of pediatric research in its latest support plan for biomedical research. And in the UK, a research network for medicines
for children has been set up to improve the coordination, speed, and quality of randomized controlled trials and other well-designed
studies of medicines for children and adolescents.
