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.
Parallel scientific advice with the EMA and FDA may lead to an increased dialogue between the two agencies and sponsors from the beginning of the lifecycle of a new product, a deeper understanding of the bases of regulatory decisions, and the opportunity to optimize product development and avoid unnecessary testing replication or unnecessary diverse testing methodologies. Parallel scientific advice should focus primarily on important breakthrough drugs or important safety issues.
Parallel Scientific Advice procedures are conducted under the auspices of the confidentiality arrangement between the European Commission, the EMA, and FDA.
The Paediatric Regulation's main aim is to improve the health of children in Europe without subjecting children to unnecessary trials, or delaying the authorisation of medicinal products for use in adults.. Paediatric Investigation Plans are becoming increasingly important, and failure to integrate paediatric studies in the development may lead to a delay in approval. Paediatric development has now become a more integral part of the overall development of medicinal products in the EU, with the Regulation working as a major catalyst to improve the situation for young patients.
Several incentives for the development of medicines for children are available in the EU and in Member States:
Many of the products currently in development are Advanced Therapy Medicinal Products, and special considerations for the products must be considered.
This seminar is specifically designed for personnel in the pharmaceutical and biotech industries who need to understand the regulatory issues involved in the development of new medicinal products within the European Union.
Upon completion of the program, participants will be able to create a comprehensive regulatory plan. All regulatory aspects to ensure a smooth development will be discussed, including how to obtain orphan medicinal product status from the EMA, as well as when to ask for scientific advice.
Designation as an orphan medicinal product in the EU has important advantages. Sponsors who obtain orphan designation benefit from a number of incentives. 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.
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.
Choosing the right procedure for scientific advice (at the European Medicines Agency or at the National Authorities in the EU) and preparing well for the procedure is very important to assure your development program meets the requirements of the Regulatory Authorities.
This webinar will address which procedure is best depending on the circumstances, and how to choose among the National Authorities. In addition, it will cover the preparation of the meeting (including briefing document).
Additionally, the topic of how to integrate paediatric studies in the development plan will be discussed along with information regarding the special regulatory aspects of Advanced Therapy Medicinal Products.