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
The regulatory plan describes the regulatory strategy, as well as pricing and reimbursement issues in your development.
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
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.
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
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
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.
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.
assistance is the special form of scientific advice available for
companies developing designated orphan medicines for rare diseases.
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.
Scientific Advice procedures are conducted under the auspices of the
confidentiality arrangement between the European Commission, the EMA,
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:
of the products currently in development are Advanced Therapy Medicinal
Products, and special considerations for the products must be
Why you should attend
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.
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
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.
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.
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).
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.Who Will Benefit
- Regulatory Affairs personnel involved in development of medicinal products in the EU, in particular from US companies