Orphan Drugs Bucuresti 25 Martie 2011

8/6/2019 Orphan Drugs Bucuresti 25 Martie 2011

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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!

Orphan Drugs Bucuresti 25 Martie 2011

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