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<p>The Early Access to Medicines Scheme (EAMS) aims to give patients with life threatening
or seriously debilitating conditions access to medicines that do not yet have a marketing
authorisation when there is a clear unmet medical need. The Medicines and Healthcare
products Regulatory Agency (MHRA) is responsible for the scientific aspects of the
scheme and the scientific opinion will be provided after a two-step evaluation process:</p><p>
</p><p>- step I, the promising innovative medicine (PIM) designation</p><p> </p><p>-
step II, the early access to medicines scientific opinion</p><p> </p><p> </p><p> </p><p>The
PIM designation will give an indication that a product may be eligible for the EAMS
(based on early clinical data) and that the development programme is on track. The
PIM designation will be issued after an MHRA scientific meeting and could be given
several years before the product is licensed.</p><p> </p><p> </p><p> </p><p>The scheme
is voluntary and the opinion from MHRA does not replace the normal licensing procedures
for medicines.</p><p> </p><p> </p><p> </p><p>There have been no applications from
companies with products for Duchene Muscular Dystrophy.</p><p> </p><p> </p><p> </p><p>The
opinion will support the prescriber and patient to make a decision on whether to use
the medicine before its licence is approved. The EAMS scientific opinion is valid
for one year in the first instance and lapses at this time or at the time of the grant
of a marketing authorisation e.g. conditional marketing authorisation.</p><p> </p><p>
</p><p> </p><p>For certain categories of medicines going through the centralised marketing
authorisation procedure (European procedure), in order to meet unmet medical needs
of patients and in the interest of public health, it may be necessary to grant marketing
authorisations on the basis of less complete data than is normally required. In such
cases, it is possible to recommend the granting of a marketing authorisation subject
to certain specific obligations to be reviewed annually, a conditional approval. The
granting of a conditional marketing authorisation will allow medicines to reach patients
with unmet medical needs earlier than might otherwise be the case.</p><p> </p><p>
</p><p> </p><p>A conditional marketing authorisation for the first in class medicinal
product Translarna (ataluren) was granted this year. Translarna is an orphan medicinal
product that is used to treat patients aged five years and older with Duchenne muscular
dystrophy (DMD) who are able to walk. Translarna is expected to slow down the loss
of walking ability in DMD patients. As part of the conditional marketing authorisation,
the company will be required to provide comprehensive data on the efficacy of Translarna
from an ongoing confirmatory study.</p><p> </p><p> </p><p> </p><p>Rare diseases are
classified as conditions affecting no more than 5 in 10,000 people in European Union
and patients with rare conditions deserve the same quality, safety and efficacy in
medicines as other patients with more common conditions. Since the pharmaceutical
industry has little interest, under normal market conditions, in developing and marketing
medicines intended for small numbers of patients (orphan medicinal products), the
European Union offers a range of incentives to encourage the development of these
medicines in order to address the unmet clinical need (orphan drug legislation, Regulation
(EC) No 141/2000). These incentives include a period of 10 years market exclusivity,
the provision of Protocol Assistance (scientific advice specifically tailored for
orphan medicinal products) and fee reductions and waivers for regulatory procedures.
Products intended for treatment of Duchenne muscular dystrophy qualify for incentives
in the orphan drug legislation.</p><p> </p><p> </p><p> </p><p>Applications for the
designation of orphan medicines are reviewed by the European Medicines Agency through
the Committee for Orphan Medicinal Products (COMP). For orphan designation, the following
criteria must be fulfilled. The medicinal product is intended for the diagnosis, prevention
or treatment of a life-threatening or chronically debilitating condition affecting
no more than 5 in 10,000 persons in the European Union or without incentives it is
unlikely that expected sales of the medicinal product would cover the investment in
its development and no satisfactory method of diagnosis, prevention or treatment of
the condition concerned is authorised, or, if such method exists, the medicinal product
will be of significant benefit to those affected by the condition. Via the MHRA, the
United Kingdom takes an active role in the decision making process at the COMP, ensuring
applications for Orphan Drug designation of potential drug candidates for rare diseases
are appropriately recognised, encouraging companies to develop their products further.
For licensing, it is compulsory for designated orphan medicinal products to use the
centralised procedure to gain a marketing authorisation.</p><p> </p><p> </p><p> </p><p>The
UK is fully represented at the Committee on Human Medicinal Products, ensuring that
applications for a Marketing Authorisation (MA) for an Orphan Drug are thoroughly
and rapidly evaluated for quality, safety and efficacy and a MA is granted without
undue delay for the treatment of rare diseases.</p><p> </p><p> </p><p> </p><p>In some
circumstances, marketing authorisations may undergo a more rapid regulatory review
called ‘accelerated assessment’. This occurs where the Applicant can demonstrate that
the medicinal product is expected to be of major public health interest (particularly
from the point of view of therapeutic innovation). For drugs for rare diseases, marketing
authorisation applications may be granted as a conditional authorisation or an authorisation
under exceptional circumstances. The granting of a conditional marketing authorisation
allow medicines to reach patients with unmet medical needs earlier than might otherwise
be the case, and ensures that additional data on a product are generated, submitted,
assessed and acted upon. Under exceptional circumstances, the MA Applicant must demonstrate
that he is unable to provide comprehensive data on the efficacy and safety under normal
conditions of use, because, for example, the indications for which the product in
question is intended are encountered so rarely that the Applicant cannot reasonably
be expected to provide comprehensive evidence.</p><p> </p><p> </p><p> </p><p>NHS England
can confirm that a draft clinical commissioning policy for Translarna treatment for
Duchenne muscular dystrophy has been developed and is being considered as part of
the annual funding prioritisation process for 2015-16.</p><p> </p><p><strong> </strong></p><p>
</p><p><strong> </strong></p><p> </p><p><strong> </strong></p><p> </p>
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