Orphan designation
In 2013, Prof Bobby Gaspar was granted orphan designation within the EU from the European
Medicines Agency (EMA) for one of our lead gene therapy products, ‘autologous CD34+ cells
transduced with a lentiviral vector containing the human ADA gene’, for the treatment of ADA-SCID.
This was a huge milestone for the clinical development of our product and will benefit us
enormously in terms of achieving our ultimate goal of licensing our product.
Those who are successful in being granted orphan designation benefit from a number of incentives,
including protocol assistance (a type of scientific advice specific for designated orphan medicines)
and ten years of protection from market competition with similar medicines with similar indications
once they are approved. Fee reductions when applying for protocol assistance, scientific advice and
ultimately marketing authorisation are also available. Orphan designated drugs also experience the
significant competitive advantage of being quicker to gain marketing authorisation for. Such
benefits are intended to encourage the development of medicines which might otherwise lack a
sufficient profit motive.
For these reasons, orphan designation can also be an important step in gaining the interest of the
investment community for your product.
This document aims to provide some practical guidance on how to apply for orphan designation.
How to apply
Sponsors can apply for orphan designation of a medicinal product at any stage during the
development of the medicinal product before the application for marketing authorisation is made.
Applications for orphan designation are free of charge.
To qualify for orphan designation, a medicine must meet a number of criteria:
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it must be intended for the treatment, prevention or diagnosis of a disease that is lifethreatening or chronically debilitating;
the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be
unlikely that marketing of the medicine would generate sufficient returns to justify the
investment needed for its development;
no satisfactory method of diagnosis, prevention or treatment of the condition concerned can
be authorised, or, if such a method exists, the medicine must be of significant benefit to those
affected by the condition.
1) Appoint sponsor
The sponsor of the orphan designation must be decided early, as you cannot proceed through the
application process below without this confirmed. The sponsor of an orphan designation application
is different to the sponsor of a clinical trial and does not have to be the same if you are conducting a
clinical trial with your product at the time of making your application. The sponsor of the orphan
designation application can be a legal entity or an individual person and must be established in the
EU. It is usually the lead entity/person who is guiding the medicinal product through to licensing and
who will be responsible for the orphan designation application. Incentives will be applicable to the
sponsor who received the orphan designation only.
2) Decide in which regions you wish to apply for designation
The EMA encourages parallel applications for orphan designation with regulatory authorities outside
the EU. Note there are different application forms to be used depending on where you are applying.
3) Notification of intention to submit
Sponsors should notify the EMA by email of their intention to submit an application as early as
possible, and at the latest two months prior to the planned submission date.
4) Pre-submission meeting
The EMA strongly encourages sponsors to request a pre-submission meeting with the Agency prior
to filing an application. These meetings are free of charge.
Pre-submission meetings usually take place via teleconference. Where possible, sponsors should
request a pre-submission meeting at least two months prior to filing.
Pre-submission meetings are useful since the evaluation process has a fixed duration of 90 days and
cannot be lengthened to accommodate for the lack of data or other omissions in the application. In
our experience, this was an invaluable step in the application process and one we would strongly
recommend.
5) Making your application
The Committee for Orphan Medicinal Products (COMP) review the application during meetings
which are usually held once a month. Submission deadlines therefore apply and these are available
on the EMA website.
The EMA website provides very detailed guidance on the content and format of documents to be
submitted. Once the submission package is complete, sponsors should submit the application
electronically to the EMA and appointed COMP coordinator via Eudralink.
6) Eudralink
Eudralink is the EMA’s secure email system. All documents are to be sent to the EMA via this
system. To use the system, you must contact the Eudralink Helpdesk (eudralink@ema.europa.eu) at
the EMA to open an account and you will then be sent a username and password to use the system.
7) Evaluation of applications
After submission, the two coordinators prepare a summary report on the application, which is
circulated to all COMP members and discussed at the COMP's next plenary meeting. At this stage,
the COMP will either adopt a positive opinion or raise a list of questions and invite the sponsor to an
oral explanation at the next COMP plenary meeting. The COMP should adopt an opinion by day 90
of the procedure. It forwards this to the European Commission for adoption of a decision. If the
COMP's opinion is negative, the sponsor can appeal.
8) European Commission decision
The European Commission will issue a decision on a COMP opinion within 30 days of receipt.
Following a decision:
 the Agency publishes information on the orphan designation under rare disease (orphan)
designations
 the European Commission enters the orphan designation into the Community register of
designated orphan medicinal products
For further information and useful guidance documents for preparing and submitting your
application, see:
http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000029
.jsp&mid=WC0b01ac05800240ce