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So the European Medicines Agency (EMA) organized a meeting of two days
inviting all stakeholders for the 10th anniversay of the Orphan Drug
Regulation
and the first day was dedicated to a sort of overview of the achievements
obtained thanks to the Orphan Drug Regulation
and so every stakeholder gave his
point of view and described the impact of the regulation
in his own field
and the second day was more dedicated to
analyse what remains to be done, what are still the burden to be
overcome in the future and have to be addressed in the future
at different levels but not only at the level of the regulation in Europe
but also outside of the scope of the Regulation.
I think the general feeling was that really that of general satisfaction
because that I think we reach the goals that were
imagined when the regulation was adopted
in 2000
but in addition of the satisfaction there was also general agreement on what are
the challenges for the future so I think
that the stakeholders are really agreed on what remains to be done
and there are already some plans in terms of actions on both national and Europe level
because
as an outcome of the meeting we can say that the
regulation is successful
but this is just one piece
of the whole frame of an orphan drug development
still the big bottlenecks are remaining today
and that is what I expressed during my intervention at the panel of the meeting.
The big ones are
before the regulation
so in the phases
where the research has been
asked to be conducted that on the diseases to understand the disease and to imagine
new therapies
so there we still need support,
funding,
both at European & National level
and the second big bottleneck is after the regulation is intervening so
when the orphan drugs are authorized
at the European level
but then there are some difficult in being available for patients at national
level
so these are the two major areas
where we should focus our own
actions in the future
I think that the regulation has really fulfilled the scope
In general I think that all the stakeholders are agreed that probably
we don't need to re-evaluate the Regulation,
re-open the discussion for a revision but
the problems are out outside of the Regulation.
In addition the Regulation had a positive impact on other pieces of legislation in
Europe
as it gave really a very unique example of how
different stakeholders can work together and how important is the role of patients
in the regulatory framework.
So it was the first time when patient groups really pushed for a new piece of legislation
in Europe
and the first time that patient groups are involved in the regulatory processes
at the agency level
so you know that the Committee for Orphan Medicinal Products (COMP) is the first committee
that has members coming from patient groups
and then the patients are also involved after the Marketing Authorization
really monitoring if these drugs
are available or not and what are the reasons for when they are not available
and these examples are then taken for the paediatric regulation and
recently for the Advanced Therapy one
so the new committees at the EMA all have patient groups
among their members.