Orphan designation evaluated by the EMA’s Committee for Orphan Medicinal Products to qualify as an orphan medicine for a rare disease

1. Overview
Orphan Medicinal Product Designation in the EU
Date: Thursday, September 29th, 2016, Time: 01:00 PM EDT | 10:00 AM PDT
Duration: 60 Minutes
Speaker: Adriaan Fruijtier
The European Medicines Agency plays a central role in the development and
authorisation of medicines for rare diseases. These medicines are termed ‘orphan
medicines’ in the medical world.
The EMA is responsible for reviewing applications from sponsors for the designation of
medicines for rare diseases.
To benefit from the incentives, sponsors intending to develop an orphan medicine must
submit an application to the EMA requesting 'orphan designation' for their medicine.
The application is evaluated by the EMA’s Committee for Orphan Medicinal Products
(COMP), which provides its opinion on whether or not the medicine qualifies as an
orphan medicine for the treatment, prevention or diagnosis of a rare disease. If the
COMP issues a positive opinion, the European Commission may then grant the
medicinal product orphan status.
Because rare diseases are a global issue, the EMA works closely with its international
partners on the designation and assessment of orphan medicines. The EMA works with
organizations representing patients with rare diseases through the European
Organization for Rare Diseases (EURORDIS).
Why should you attend?
Orphan medicinal products are intended for the diagnosis, prevention or treatment of life-
threatening or very serious conditions that affect no more than 5 in 10,000 people in the
European Union.
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2. Designation as an orphan medicinal product in the EU has important advantages.
Sponsors who obtain orphan designation benefit from a number of incentives, including
protocol assistance, a type of scientific advice specific for designated orphan medicines,
fee waivers or reductions for the regulatory procedures or a 10 year market exclusivity
once the medicine is on the market. Therefore, it is important for companies to know the
process for designation.
In this Webinar the process for obtaining designation is explained in detail. There are
many factors to consider when preparing an application, and taking those into account
will ensure a smooth process. The main factors to consider are:
 Definition of a medical condition
 Dealing with subsets
 How to show significant benefit
Practical experience with the process (for instance about pre-submission meetings and
the role of the Committee for Orphan Medicinal Products) is discussed and very valuable
when preparing an application.
It is also important to be aware of the activities after orphan designation.
Areas covered in the webinar
The Webinar will start with an introduction. It is estimated that today in the EU, 5-8000
distinct rare diseases affect 6-8% of the population - about 30 million people. Rare
diseases are defined as life-threatening or chronically debilitating conditions that affect no
more than 5 in 10,000 people in EU. This is equivalent to around 250,000 people or less
for each disease.
These conditions occur so infrequently that the cost of developing and bringing to the
market a medicinal product to diagnose, prevent or treat the condition would not be
recovered by the expected sales of the medicinal product.
Patients suffering from rare conditions should be entitled to the same quality of treatment
as other patients.
Since the pharmaceutical industry has little interest, under normal market conditions, in
developing and marketing medicines intended for small numbers of patients it is
necessary to stimulate the research, development and bringing to the market of
appropriate medications by the pharmaceutical industry. Incentives for the development
of orphan medicinal products have been available in the United States of America since
1983 and in Japan since 1993, and also the EU offers a range of incentives to encourage
the development of these medicines.
The EMA maintains a searchable list of opinions on applications for rare disease
(orphan) designations. Sponsors of designated orphan medicines are eligible to benefit
from the incentives offered, including:
 assistance with development of the medicine;

3.  reduced fees for marketing-authorisation applications;
 protection from market competition once the medicine is authorized
Designated orphan medicines are assessed centrally on a European level by the EMA's
Committee for Medicinal Products for Human Use (CHMP), rather than in each Member
State separately.The guidelines for Marketing Authorisation procedures and determinants
for successful Marketing Authorisations will be discussed.
Learning objective
 What designation as an orphan medicinal product means?
 What benefits the designation has?
 How to obtain designation?
Who will benefit
 Regulatory affairs officers and managers
 Clinical trial associates
 Clinical operations personnel
 Product development personnel
 Personnel in the pharmaceutical and biotech industries who need a thorough
understanding of the regulatory issues involved in the development of new
medicinal products within the European Union
Speaker profile
Adriaan Fruijtier has graduated as a pharmacist at the University of Utrecht, The
Netherlands. He is currently Director Regulatory Affairs at CATS Consultants. Until
March 2004, he has been Head of the Oncology Group within Global Regulatory Affairs
at Bayer AG, Wuppertal, Germany, and Bayer Corporation, West Haven, CT, USA.
Between 2001 and 2003, he was Director of Regulatory Affairs at Micromet AG, a biotech
company in Munich, Germany. Prior to joining Micromet he has worked for four years as
a Project Manager for Oncology Projects at the European Medicines Agency in London,
United Kingdom. He joined the European Medicines Agency from Novartis AG, Basel,
Switzerland, where he was Regulatory Affairs Project Manager in the Oncology group in
1996 and 1997. Before 1996, he was Head of Drug Regulatory Affairs for six years at
Ciba-Geigy in the Netherlands, and has worked as Manager Regulatory Affairs at Glaxo,
also in the Netherlands.
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