6-Hour Virtual Seminar on EU Regulatory Affairs Strategy During Development of Medicinal Products

The main document from a regulatory perspective in the development of a medicinal product is the regulatory plan. In this Seminar it is explained how to write the regulatory plan, and which aspects to consider.

The regulatory plan describes the regulatory strategy, as well as pricing and reimbursement issues in your development.
Orphan Medicinal Products will be discussed, and the advantages of having a status as an orphan medicinal product will be explained.

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.

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.

Sponsors of designated orphan medicines are eligible to benefit from the incentives offered, including:

• Assistance with development of the medicine
• Reduced fees for marketing-authorisation applications
• Protection from market competition once the medicine is authorised

Scientific advice is a vital element in the development of a medicinal product, and knowledge of the how to choose between national and EU scientific advice, as well as the preparation and procedure is vital for a successful outcome.

Scientific advice helps the company to make sure that it performs the appropriate tests and studies, so that no major objections regarding the design of the tests are likely to be raised during evaluation of the marketing-authorisation application. Such major objections can significantly delay the marketing of a product, and, in certain cases, may result in refusal of the marketing authorisation. Following advice from the Regulatory Authorities increases the probability of a positive outcome.

The Regulatory Authorities give scientific advice by answering questions posed by companies. The advice is given in the light of the current scientific knowledge, based on the documentation provided by the company. It is not the role of the Regulatory Authorities to substitute the industry's responsibility for the development of their products.

Protocol assistance is the special form of scientific advice available for companies developing designated orphan medicines for rare diseases.