The New Clinical Trials Regulation and Regulatory Affairs Aspects of Medicinal Product Development in the EU
Course "The New Clinical Trials Regulation and Regulatory Affairs Aspects of Medicinal Product Development in the EU" has been pre-approved by RAPS as eligible for up to 12 credits towards a participant΄s RAC recertification upon full completion.
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.
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.
Paediatric Investigation Plans are becoming increasingly important, and failure to integrate paediatric studies in the development may lead to a delay in approval.
Many of the products currently in development are Advanced Therapy Medicinal Products and medicinal products used in oncology, and special considerations for the products must be considered.
An overview of the current process for clinical trial applications is included, but the Seminar will also look ahead and describe the effects of the Clinical Trials Regulation.
The Clinical Trials Regulation aims to create an environment that is favourable for conducting clinical trials, with the highest standards of patient safety, for all EU Member States. Intrinsic to this is the simplification of current rules, for example:
A streamlined application procedure via a single entry point - an EU portal and database, for all clinical trials conducted in Europe. Registration via the portal will be a prerequisite for the assessment of any application;
A single authorisation procedure for all clinical trials, allowing a faster and thorough assessment of an application by all Member States concerned, and ensuring one single assessment outcome and authorisation per Member State;
The extension of the tacit agreement principle to the whole authorisation process which will give sponsors and researchers, in particular SMEs and academics, more legal certainty;
Strengthened transparency for clinical trials data.
Ethics committees will be involved in the assessment of clinical trials application. However, as with the current situation, their responsibilities and detailed composition will be determined independently by each EU country. In this way the different traditions in the various Member States are respected.
The Regulation, while continuing to uphold patient safety, takes better account of the actual risk to which subjects will be exposed during the clinical trial and adapts the regulatory burden in relation to the risk posed. It introduces the concept of a ΄low-intervention clinical trial΄ - an example being clinical trials comparing already authorised medicines. In such cases, the regulatory requirements will be lighter. Transparency on the conduct and results of clinical trials has several benefits, and the Regulation strengthens the rules accordingly. Transparency avoids redundancy and duplication. It ensures that even clinical trials with
unfavorable results are made public, thereby avoiding ΄publication bias΄. Finally, transparency gives patients the possibility to find out about on-going clinical trials in which they may wish to participate.
Why should you attend?
This seminar is specifically designed for personnel in the pharmaceutical and biotech industries who need to understand the regulatory issues involved in applications for clinical trial approval and clinical 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.
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 and Oncology Medicinal Products.
It is important to know the new procedures for approval of clinical trials to avoid delays and minimize the time for approval. The current system for clinical trials applications in the EU will be reviewed, as well as the changes under the new Clinical Trial Regulation.
In the current situation, a common protocol is proposed in all EU Member States that participate in a clinical trial but requests for amendments may result in country-specific changes that require subsequent amendments in other countries to maintain a common protocol. In the Seminar it will be explained how this problem can be avoided.
The new Regulation will change the way pharmaceutical companies handle the clinical trial approval process, and companies need to start preparing for these changes now. The timelines for implementation will be discussed in this Seminar to allow optimal preparation.
Areas Covered in the Session:
Developing a regulatory strategy
Pricing and reimbursement issues in your development
Orphan medicinal Products
Scientific advice and how to choose between centralised and national scientific advice
Paediatric Investigation Plans
Advanced Therapy Medicinal Products
Oncology medicinal products
Day 1 Schedule
Elements of development regulatory affairs
The regulatory plan
Overview of the European Medicines
Regulatory strategy and regulatory plans (including target SmPC)
Structure of the regulatory plan
Sources for the regulatory plan
Pricing and reimbursement
What is Health Technology Assessment (HTA)?
Who decides on pricing and reimbursement: the HTA bodies
Orphan medicinal products
Rare diseases: orphan medicinal products?
Applying for orphan medicinal product designation
Why and when is scientific advice needed and useful?
Topics for scientific advice
Briefing document, timelines and planning: from submission to final scientific advice by CHMP
National versus EMA
Joint scientific advice CHMP + HTA bodies: benefits and issues to consider
Research and development programme for medicines in children: Paediatric Investigation Plans
Cases in which studies in children are not needed or will be done later: Waivers/deferrals
What is a paediatric use marketing authorisation (PUMA)?
Advantages of micro-, small- and medium-sized-enterprise (SME) status
How to apply for SME status
Day 2 Schedule
Advanced Therapy Medicinal Products (ATMP)
Early scientific evaluation of quality and non-clinical data: Certification procedure
Practical exercise: Determine for various products if they fall under the ATMP definition
Why is development of oncology products different?
Preclinical considerations: Which animal studies are needed for oncology products?
The new clinical oncology guideline
Practical session: regulatory plan
Case study: New oncology product
Does it qualify for orphan drug designation?
How to propose the optimal orphan indication
Paediatric studies necessary?
Scientific advice: Topics, selection of authorities
How to apply for a clinical trial authorisation in the EU
The clinical trial directive
Outline of the procedure
Content of the Investigational Medicinal Product Dossier and important guidance documents
Voluntary Harmonisation Procedure (VHP)
Harmonisation of assessment of clinical trial applications in several EU countries through the VHP:
Overview of the process
Advantages and disadvantages
Future changes to the clinical trial authorisation process in the EU
The Clinical Trial Regulation
Impact on the pharmaceutical and biotech Industry
Interactive discussion: what are the advantages and disadvantages of the new Regulation?
Regulatory Affairs Consultant, CATS Consultants GmbH
Adriaan Fruijtier has graduated as a pharmacist at the University of Utrecht, The Netherlands.