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The role of orphan medicinalproducts and the access totreatment
Maria Puiu, Dorica Dan
ANBRaRo
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About Orphan Drugs
"Orphan drugs" are medicinal productsintended for diagnosis, prevention or
treatment of life-threatening ordebilitating rare diseases.
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Rare diseases
In the EU, a rare disease is one whichaffects fewer than 5 people per 10 000.
The number of sufferers may still behigh, however, as there are some
7000 known rare diseases.
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Rare diseases
While signs may be detected at birth or inchildhood, more than 50% of rare diseases
appear during adulthood, and are often life-threatening or chronically debilitating.
Most are caused by genetic defects, butenvironmental exposure during pregnancy orlater in life, often in combination with geneticsusceptibility, is another cause.
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The EU is promoting optimal
prevention, diagnosis andtreatmentof rare diseases bycreating networks, sharing
experience and training, anddisseminating knowledge.
Rare diseases
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Rare diseases are a serious public
health concern and a priority in theEU health and research programmes.
EU law and funding for R&D projectsaim to promote the development of
orphan drugs for patients with rarediseases.
Rare diseases
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Usually there is no effective treatment, butscreening for early diagnosis, followedby suitable care, can improve quality of
life and life expectancy.
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It became increasingly obvious that these
patients need support from European
community.
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All the national single efforts in thisfield are not sufficient and those
diseases are so rare, there we needcenters and experts in each EuropeanState.
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About Orphan Drugs
They are "orphans" because thepharmaceutical industry has little
interest under normal marketconditions in developing andmarketing drugs intended for only asmall number of patients suffering
from very rare conditions.
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To provide care for these forgottenpatients and to encourage
pharmaceutical and biotechnologycompanies to invest in treatment forrare diseases, governments havecreated various legal and financialincentives.
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In 1983 the Orphan Drug Act wasintroduced in the US and in 1999 the
Regulation on orphan medicinalproducts was approved by the
European Parliament.
This regulation grants, among other
things, a ten-year market exclusivityfor any new orphan drug.
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Orphan Europe has been developingand providing orphan drugs for thetreatment of rare diseases since1990, well before the orphan druglegislation was introduced in Europe.
We have the conviction that everypatient has the right to the bestpossible treatment, especiallypatients suffering from a raredisease.
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Scientific Committee on MedicinalProducts and Medical Devices
1997-2004
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The European Medicines Agency is adecentralised agency of the EuropeanUnion, located in London.The Agencyis responsible for the scientific
evaluation of medicines developed bypharmaceutical companies for use inthe European Union.
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http://www.ema
.europa
.eu/
A designation from the European MedicinesAgency's Committee on Orphan MedicinalProducts (COMP) permits a pharmaceutical
company to benefit from incentives fromthe European Union to develop a medicinefor a rare disease such as a geneticdisorder or a rare cancer.A large number
of these diseases affect children andnewborn babies.
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In America:
The U.S. Food and Drug Administration(FDA) website offers a list of orphandrugs that have received marketing
approval in America.www.fda.gov
In Europe:
European Union registryof designated orphan drugs
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In Romania, the growinginterest of thegovermental healthsystem is due to theRare DiseasePatients Associations
effort in the last 5years (RPWA thenRoNaRD).
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Law of 20 November 2003establishing the criteria for
designation of orphan medicinalproducts and how the marketingauthorization of its.
Published in Monitorul Oficial 874/
9 December2003 (M. Of. 874/2003)
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Health Programs
HP chronic disease in children
HP for hemophilia, thalassemia andother rare diseases - 2008
HP for rare diseases and severesepsis -2009
HP for Rare diseases - 2010
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N
ational MedicinesA
gency
47 orphan drugs - 2009
http://www.anm.ro/_/Lista%20medicamentelor%20orfane%20valide%20in%20Romania.xls
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Treatment of patients with rare diseases
through the National Plan for Rare Diseasesnot through Health Programs
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The future of orphan drugs
Together
Thank you!