Orphan Medicines
Vasiliki Nicolaou



•About EURORDIS
•EURORDIS is a non-governmental patient-driven alliance of patient organisations representing 826
rare diseases patient organisations in 70 countries.
•They are the voice of 30 million people affected by rare diseases throughout Europe.
•EURORDIS represent in the various activities linked to the orphan drug development process.
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•What they do
•EURORDIS' mission is to build a strong European community of patient organisations and people
living with rare diseases, to be their voice at the European level. The role played by EURORDIS is
financially independent from the pharmaceutical industry. All orphan drug activities are made
possible through the work of EURORDIS volunteers and the financial support from its members.
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•International activities
•Their vision is to unite, expand and reinforce the rare disease movement of patient organisations
and patient advocates around the world.
•

•What is Orphanet ?
•Orphanet is a unique resource, gathering and improving knowledge on rare diseases so as to improve
the diagnosis, care and treatment of patients with rare diseases.
•Orphanet aims to provide high-quality information on rare diseases, and ensure equal access to
knowledge for all stakeholders.
•Orphanet was established in France by the INSERM (French National Institute for Health and Medical
Research) in 1997.This initiative became a European endeavour from 2000, supported by grants from
the European Commission: Orphanet has gradually grown to a Consortium of 40 countries, within
Europe and across the globe.

REGULATION (EC) NO 141/2000 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL
•On 16 December 1999, the European Parliament adopted Regulation (EC) No 141/2000 (the Orphan
Regulation).
•This Regulation:
üThe main objective of this Regulation is to create a Community for medicinal products as orphan
medicinal products and to provide motivation for the research, development and placing them on the
market as orphan medicinal products.
üEstablishes the Committee for Orphan Medicinal Products (COMP).
üSponsors of orphan medicinal products designated under this Regulation should be entitled to the
full benefit of any incentives granted by the Community or by the Member States to support the
research and development of medicinal products for the diagnosis, prevention or treatment of such
conditions, including rare diseases
üThe Agency (European Agency) sends the COMP(Committee for Orphan Medicinal Products ) opinion to
the European Commission, which is responsible for granting the orphan designation.
ü

Why it was created?
üAbout 30 million people living in the European Union (EU) suffer from a rare disease.
üPatients suffering from rare conditions should be entitled to the same quality of treatment as
other patients and deserve the same quality, safety and efficacy in medicinal products. Its
necessary to stimulate the research, development and bringing to the market by the pharmaceutical
industry.
üRare diseases have a priority for the Community action in the field of public health.

Medicinal Product designated as orphan
•Orphan drug status :
•
•For  diagnosis, prevention or treatment of a life-threatening or chronically condition affecting
small number of people in the European Community (EC).

Marketing Authorization
•Authorisation to place the medicinal product on the market be granted by the Community .
•The marketing authorisation shall cover only those therapeutic indications which fulfil the
criteria set of orphan medicinal products and cover the conditions which are rare and to give the
opportunity in patients with such conditions to feel safe and to have the same quality of life as
other patients.

Market exclusivity
• The holder of the marketing authorisation for the original
•orphan medicinal product :
•
ühas given his consent to the second applicant.
üis unable to supply sufficient quantities of the medicinal product.
üthe second applicant can prove in the application that the second medicinal product, similar also
to the orphan medicinal product already authorised, is safer and  more effective.

References
•https://www.eurordis.org/orphan-drug-designations-marketing-authorisations
•https://www.orpha.net/consor/cgi-bin/OC_Exp.php?lng=EN&Expert=25017
•https://www.eurordis.org/sites/default/files/publications/Fact_sheet_OD-Eurordis.pdf
•https://www.orpha.net/consor/cgi-bin/index.php
•https://rarediseases.info.nih.gov/diseases/fda-orphan-drugs
•https://www.fda.gov/drugs/resourcesforyou/consumers/ucm143563.htm
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