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    Orphan medicinal products

    What is an orphan medicinal product?

    The orphan medicinal products can be defined as drugs not produced by the pharmaceutical industry for economic reasons, but to meet public health needs in the treatment of rare diseases.

    According to the European Medicines Agency (EMA), in order to recognize a medicinal product as an orphan, it must meet the following criteria:

    • it must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating;
    • the incidence of the disease in the European Union must not exceed 5 per 10,000 or it is unlikely that marketing the medicine would generate sufficient returns to justify the investment needed for its development;
    • there is no satisfactory method of diagnosing, preventing, or treating the condition in question or, if such a method exists, the medicine must be of significant benefit to those affected by the condition

    If the drug meets the above criteria, the sponsor may apply for orphan designation.

    Obtaining orphan designation guarantees you will receive a number of benefits from the EMA, such as protocol assistance, scientific advice, 10-year market exclusivity for the registered indication(s) and significantly lower registration fees (which translates into greater reimbursement for research)

    How to apply for orphan drug designation?

    Before applying  for orphan designation, a pre-submission meeting with the EMA is possible. This meeting  most often takes place via teleconference, but it is also possible to hold such a meeting in person at the Agency. If a sponsor feels they could benefit from a preliminary discussion before the submission of an orphan drug application, they can request a pre-submission meeting/teleconference at least two months prior to their planned submission date. This should allow enough time for any amendment of the application as recommended by EMA.

    An application for orphan designation must be submitted via the IRIS online platform. The document can be sent without notice, but it is good practice, as requested by EMA, to be sent a few days before any of the published application deadlines available on the EMA website. This will provide more time for the validation process and the possibility to intervene in case of technical problems.

    EMA will validate the application and if the application is considered  invalid or incomplete, the Agency sends a letter explaining to the sponsor. SciencePharma provides comprehensive support to make the entire procedure go smoothly. Once the validation is completed, the Agency will set a timetable for the evaluation procedure

    In the next stage, the application is assessed by two coordinators:

    • one member of the Committee for Orphan Medicinal Products (COMP);
    • one scientific administrator from the EMA secretariat.

    They prepare a summary report which is sent to all COMP members and then discussed in the plenary meeting. At this stage, COMP issues a positive opinion or presents a list of questions and invites the sponsor to the next COMP plenary meeting to provide oral explanations. COMP should adopt an opinion by day 90 of the procedure, to be eventually forwarded to the European Commission for adoption of a decision.

    If the COMP’s opinion is negative, the sponsor can appeal.

    The European Commission will issue a decision on the COMP opinion within 30 days of receipt, and following the decision, EMA publishes information on the orphan designation. The European Commission enters the orphan designation into the Community register of orphan medicinal products.

    What scientific data should be presented in orphan designation application?

    The sponsor should submit details of the proposed orphan condition for which designation is sought, including a review of the relevant scientific literature. A clear description should be given of the disease or condition, with details of its causes and symptoms, as well as a detailed rationale for the use of the medicinal product in the proposed orphan condition (medical plausibility). When describing the condition, it is important to discuss its prevalence, whether there are other means for its diagnosis, prevention or treatment and how effective they are, and justify that the proposed new product brings significant benefit to patients. To support the rationale for developing the product in the proposed condition, non-clinical or preliminary clinical data are generally required. In particular, sponsors are expected to present studies with the substance in a relevant model(s) of the condition and, if possible, preliminary clinical data in patients with the condition.

    Why SciencePharma?

    Thanks to our experienced experts and medical writers, we can offer full support in preparing and submitting documentation for orphan drug designation, as well as supervising the procedure and taking part in the meeting with the EMA. We know how important it is to take a comprehensive approach to each application to avoid unnecessary delays, translating into financial losses and lowering the credibility of the company. Companies from all over the world benefit from our support, and constant cooperation is the best indicator of the high quality of services provided by SciencePharma.

    Keeping abreast of the latest Competent Authorities requirements is much easier with the support of an experienced and well-informed team. We offer preparation and submission of an application for orphan drug designation and advise on doubts related to the selection of the most favorable solutions. SciencePharma support Project Coordinators in planning the registration schedule based on a customized registration strategy. We also provide comprehensive registration/post-registration services for medicinal products – depending on the individual needs of the Marketing Authorization Holder.

    If you are interested in our services, please do not hesitate to contact us. Our Experts will answer all your questions and doubts.

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