paediatric medicinal products
A significant proportion of the medicinal products prescribed to
children have not yet been (adequately) researched in children. The
efficacy and risks of their use in children therefore require more
thorough documentation.
The MEB attaches great importance to obtaining appropriate data on
medicinal products for children. The development of new knowledge,
as well as additional information about the use of medicinal
products in children, will contribute to improved treatment.
The MEB now has a good working relationship with prominent
paediatricians/pharmacists in the Netherlands in the field of
pharmacotherapy in children, including the Dutch Knowledge Centre
for Paediatric Pharmacotherapy NKFK and various research
groups.
Information about paediatric medicinal products:
Paediatric Committee
On 26 January 2007, the Regulation on medicinal products for
paediatric use was published and an EMA
committee was established, the Paediatric Committee (PDCO). This committee plays a key role
in monitoring authorisation studies for paediatric medicinal
products.
Currently, the
marketing authorisation application for a new medicinal product
must also include the outcomes of research performed on the basis
of a so-called PIP (Paediatric Investigation Plan). This PIP must
be approved by the PDCO. The PDCO can also grant deferment for
research in children or decide that such research is not needed
(e.g., because the medicinal product is intended for diseases that
do not occur in children). As an incentive for paediatric medicines
research, patents for medicinal products (in effect the
Supplementary Protection Certificate = SPC or additional patent
protection certificate) is extended by 6 months.
The option of marketing a special paediatric medicinal product
(Paediatric Use Marketing Authorisation - PUMA) is created for
existing medicinal products. A special protective mechanism applies
for these products: even if the paediatric medicinal product is a
line extension of an existing medicinal product, a new protective
period for the dossier is nonetheless created.
Paediatric
Worksharing Procedures
Articles 45 and 46 of the Regulation on medicinal products for
paediatric use outline two types of worksharing procedures. Article
45 of the Regulation stipulates that marketing authorisation
holders must submit the outcomes of all trials with their medicines
in children to the authorities before 26 January 2008. All
marketing authorisation holders have now complied, even if no
paediatric indication is authorised for their product. The
objective of this regulation article is to have all data which had
not been submitted previously, available and evaluate them in a
worksharing procedure. Following the worksharing procedure, a
public assessment report is published and paediatric information in
SmPCs and package leaflets of products is harmonised where
possible. The CMDh coordinates
this worksharing procedure. Harmonisation often proves difficult as
the products did not undergo prior harmonisation.
Regulation article 46
stipulates that marketing authorisation holders must submit all
paediatric medicine trials to the authorities within 6 months
following completion of the trial. The objective of this regulation
article is to evaluate all new studies in a worksharing procedure
also, enabling direct harmonised inclusion of this new information
in the SmPCs and package leaflets of all relevant products in the
EU. This worksharing procedure is also coordinated by de
CMDh.
Paediatric
Medicine Experts
Both the European Commission and the MEB attach great importance
to:
- the development of new medicinal products for children
- continued research into the use of existing medicinal products
in children
- the development of new pharmaceutical forms of existing
medicinal products for children
- remaining focused on medical (research) practice while
realising its tasks
With the adoption of the Regulation on medicinal products for
paediatric use, and in that connection the establishment of the
Paediatric Committee in Europe, the MEB has expanded and
consolidated its expertise in the field of paediatric medicine. To
this end, collaboration has been sought with a number of prominent
paediatricians/pharmacists and research groups in the Netherlands
in the field of pharmacotherapy in children.
History
The paediatric medicines expert group was established on
23 March 2005 at the initiative of the then chairman Frits
Lekkerkerker. The MEB was eager to establish such a sounding board
group because significant regulatory and scientific developments
were anticipated in the field of paediatric medicines. This was
obvious from many initiatives, such as the establishment of the
Medicines for Children Research Network MCRN, the Dutch Knowledge
Centre for Paediatric Pharmacotherapy NKFK, and the implementation
of the paediatric regulation. It seemed wise to bring this
expertise together. There was a need to have a group that
critically monitored MEB activities in the field of paediatric
medicines and conveyed signals from the field to the MEB. A group
that would take joint initiatives and would meet regularly – a
sounding board group for the MEB. Additionally, members were asked
on an ad hoc basis to provide input on specific issues or problems,
based on their experience and knowledge. They were also asked to
screen reports written by MEB staff specifically for paediatric
medicines or applications.
The paediatrician group clearly expressed two wishes that
aligned with the objective of the paediatric medicine knowledge
centre: encouraging and expanding paediatric medicine research in
the Netherlands, and having a database available on the efficacy
and safety of paediatric medicines that doctors have in their
office, so as to improve prescription. They wanted the MEB to take
a proactive approach to the field so as to make information
available rapidly and transparently. There was an obvious need for
adequate pathways to accelerate the availability of paediatric
medicinal research outcomes.
The expert group has played a very significant role for the MEB
and developments in the field of paediatric medicine evaluation and
linking the assessment of these medicinal products to medical
practice.
Current
collaboration
Although the expert group contributed significantly on an ad hoc
basis to MEB activities in recent years, it was concluded that the
expert group in its current form where meetings are planned at
regular intervals, no longer meets our mutual needs. Additionally,
it has become clear that there are now many ways to share knowledge
and experience outside of the meeting setting, that the MEB and
experts in the field can work together effectively, and that an
efficient network has evolved. Following an in-depth evaluation of
the past 6 years of collaboration using the sounding board group
concept, it was decided to redesign this collaboration effort with
paediatric medicine experts in a way that will likely better meet
practical needs. The MEB now has a good working relationship with
the Dutch Knowledge Centre for Paediatric Pharmacotherapy NKFK and
various research groups.
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