Orphan Medicinal Product Designation in the EU

Overview:

The European Medicines Agency plays a central role in the development and authorization of medicines for rare diseases. These medicines are termed ‘orphan medicines’ in the medical world.

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 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 medicine orphan status.

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;
• Reduced fees for marketing-authorization 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.

Because rare diseases are a global issue, the EMA works closely with its international partners on the designation and assessment of orphan medicines:
It works with the United States Food and Drug Administration (FDA), sharing information on orphan medicines under their confidentiality arrangement. The two authorities have also developed common procedures for applying for orphan designation and for submitting annual reports on the status of development of designated orphan medicines. It collaborates with the Ministry for Health, Labor and Welfare (MHLW) in Japan on issues related to 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.

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

Areas Covered in the Session:

• Legal background
• The COMP
• Criteria for designation
• How to describe the medical condition
• How to deal with subsets
• Significant benefit
• Timing of designation
• Procedure for obtaining designation
• Guidelines for Marketing Authorization procedures
• Determinants for successful Marketing Authorizations

Who Will Benefit:

• Regulatory Affairs personnel
• Senior management
• Project Managers
• Medical writers
• CRAs and CRCs
• QA / Compliance personnel
• Investigators
• Clinical Research Scientists
• QA / QC Auditors and Staff
• Consultants