EMEA and MAA
Assessment of marketing authorisation
applications The primary activity of the
“pre-authorisation” unit, through its two
scientific committees, is the assessment of
MAAs for new medicinal products and new
active substances. In carrying out the assessment,
the scientific committee concerned
(either the CPMP or the COMP) produces
an opinion which is then transmitted to the
European Commission for conversion into
a legally binding decision (ie, approval).
In 2001, the EMEA received 110 applications,
of which 58 were for new medicinal
products and 40 for new active substances.
The remaining 12 applications were for
products to be approved under orphan drug
legislation. Under EU legislation, an MAA
submitted via the centralised procedure to
the EMEA should take a maximum of 210
days to be assessed. All the applications were
assessed in 2001 within an average of 170
days, a timescale that does not include the
time taken for the agency to ask questions
and receive responses from applicant companies.
A further average of 76 days has been
required for the conversion of the opinion
into a decision by the European Commission,
and is the one aspect of the centralised
procedure that has received greatest criticism.
Attempts are being made to minimise
the delays in the decision-making process.
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Orphan medicinal products Orphan medicinal
products are used for the diagnosis, prevention,
or treatment of life-threatening or
serious conditions that occur only rarely and
affect not more than five in 10,000 people in
the EU. Under normal circumstances, the
high costs associated with the development
of new medicinal products would preclude
the likelihood of pharmaceutical companies
generating a profit from developing medicinal
products for such conditions. The EU
has therefore followed the example set by
the US in passing orphan drug legislation,
through which incentives are offered to
companies that carry out research and
development to produce medicinal products
for such rare conditions.
The incentives that have been agreed
under Regulation (EC) 141/2000 are:
�� Market exclusivity will be given for 10
years after the granting of a marketing
authorisation (ie, during that period,
directly competitive similar products
cannot usually be placed on the market)
�� There will be provision of scientific
advice from the EMEA to facilitate the
development of the medicinal product,
and guidance on preparing an MAA that
will satisfy regulatory requirements.
This will maximise a company’s chances
of getting a medicinal product approved
�� There will be direct access to the centralised
procedure, allowing the
approved product to be marketed in all
EU member states
�� Reduced fees are offered for both scientific
advice and the submission of the
MAA, using subsidised funds from the
European Commission that have been
agreed annually by the European Parliament
�� There may be financial assistance to
companies and organisations developing
orphan medicinal products through
grants from community and member
state programmes and initiatives supporting
research and development,
including the community framework
programmes
Designation of a product as an orphan
drug and its assessment is carried out within
the EMEA by the COMP. The COMP has
one representative nominated by each
member state, three from patient organisations
and three from the EMEA.
The COMP has 90 days in which to
approve (or otherwise) a request for orphan
drug designation; a positive opinion on the
designation is then forwarded to the European
Commission, which has a further 30
days in which to confirm the opinion and
issue a decision. Once this is completed, the
medicinal product is placed on the Register
of Orphan Medicinal Products.
In the early period of the operation of
the COMP, most of the medicinal products
that were granted orphan drug status were
for the treatment of cancers, immunological
diseases and metabolic diseases often related
to enzyme deficiencies. Equally importantly,
about two-thirds of the products were for
the treatment of children, who have traditionally
been a group for whom pharmaceutical
companies have been reluctant to
devote time and resources because of the
relatively small potential market. Examples
of medicinal products that have been designated
as orphan medicinal products since
2000 are given in Table 1.
AI
