Pilot I - Best Practice Transfer

The aim of Pilot I was to transfer an identified best-practice example in the EU to another EEA member state. This led to the design of an online training course in regulatory science for academic researchers.

The presentations are sorted by thematic areas. You will find a short description about the content of the presentation below each link. To access the presentations (disclaimer), please select one of the following thematic areas or download all available presentations (21 MB).

Slides of the Online Training Course

Below you will find the slides of the training 'The Winding Road from a Brilliant Idea to Drug Approval: An Online Course in Regulatory Science for Academic Researchers' which took place on 23-26 February 2021.

The curriculum covered topics on essential regulatory knowledge, case examples, insights into the regulatory system, as well as direct interaction and question & answer sessions with regulators and clinical assessors.

The slides represent a valuable source of information for academic researchers who are involved in basic research, drug development, non-clinical studies and clinical trials. The intention is to enhance the knowledge on the regulatory system in the EU and regulatory requirements as well as to learn more about regulatory tools and support offers for academia.

The overall feedback was positive and 94 % of participants would recommend such a course to other colleagues. In particular, the insights given on the regulatory system and framework and regulatory pathways were considered to be very helpful. The course evaluation revealed further input which will feed into the STARS Common Strategy.

Essential Regulatory Knowledge in Drug Development

F-I-H Sponsor – Investigator – Clinical Trial Site

Lecturer: Eva Hruskova Reinova, Clinical trial assessor at the Czech State Institute for Drug Control (SÚKL, Czech Republic)

Content: This presentation highlights all regulatory important aspects on clinical trials, the legal framework, phase I and first-in-human studies, information on essential documentations, as well as duties of the sponsors and investigators of a clinical trial.

Non-Clinical Research: Key Milestones in the Drug Developement

Lecturer: Eva Kolouchová, Nonclinical assessor for clinical trial and marketing authorisation at the Czech State Institute for Drug Control (SÚKL, Czech Republic)

Content: This presentation highlights the relevance of non-clinical studies in the drug development and gives background about the legislation, directives and guidelines. It also gives insights into special type investigational medicinal products (e.g. ATMPs), good laboratory practice (GLP) and background on national scientific advice.

Investigational Medicinal Product Dossier (IMPD) - regulatory quality

Lecturer: Barbora Ladinová, Head of the Unit of Biological Product Assessment, incl. Clinical Trials and Ivana Pravdová, Acting Head of the Chemical Products Clinical Trials Assessment at the Czech State Institute for Drug Control (SÚKL, Czech Republic)

Content: Here you will find background information the regulatory requirements for an IMPD, including legislation background, IMPs of chemical and biological/biotechnological origin, labelling, etc.

Specific Regulatory Knowledge for Advanced Therapy Medicinal Products (ATMPs)

Advanced Therapy Medicinal Products (ATMPs)

Lecturer: Tomas Boran, Director of the Marketing Authorisation Section at the Czech State Institute for Drug Control (SÚKL, Czech Republic)

Content: This presentation gives a broad overview about ATMPs, including the definition, legislation, different examples of ATMPS. It also highlights clinical trials with ATMPs and the statistics behind, gives an overview about ATMPs with marketing authorisation and further sources where to find relevant information.

Requirements on Quality (ATMP)

Lecturer: Ivana Haunerová, Quality assessor for biotech and ATMPs atthe Czech State Institute for Drug Control (SÚKL, Czech Republic)

Content: Here you will find information about good manufacturing practice (GMP) for ATMPs, information in the Common Technical Document (CTD) format of the dossier, relevant guidelines as well as information in medical devices as part of ATMP.

Case study: ATMP Scientific Advice

Lecturer: Viktoriia Starokozhko, Clinical assessor at the Dutch Medicines Evaluation Board (MEB, Netherlands)

Content: This presentation gives a case-example about a Scientific Advice for LV-based gene therapy for haemoglobinopathy and highlighted the questions, topics and scope that can be discussed in a Scientific Advice meeting.

Regulatory System and Framework

How Regulators work and Think; the basics

Lecturer: Peter Mol, Senior assessor at the Dutch Medicines Evaluation Board (MEB, Netherlands) and a vice chair of EMA’s Scientific Advice Working Party

Content: This presentation gives insights about the regulatory system in the EU, how regulators work and think and it highlights examples from regulatory daily work.

Scientific Advice (European and National)

Lecturer: Marjon Pasmooij
Head of the Science Office at the Dutch Medicines Evaluation Board (MEB, Netherlands)

Content: The presentation explains the concept of Scientific Advice, the different formats of Scientific Advice and why it is useful for academia.

Novel regulatory tools & drug development support mechanisms

Lecturer: Peter Mol, Senior assessor at the Dutch Medicines Evaluation Board (MEB, Netherlands) and a vice chair of EMA’s Scientific Advice Working Party

Content: Here you will find background information and insights about regulatory tools in the EU and an introduction into the PRIME scheme of EMA.

Case study: ATMP Scientific Advice

Lecturer: Viktoriia Starokozhko, Clinical assessor at the Dutch Medicines Evaluation Board (MEB, Netherlands)

Novel Methodologies and Real World Evidence

Lecturer: Peter Mol, Senior assessor at the Dutch Medicines Evaluation Board (MEB, Netherlands) and a vice chair of EMA’s Scientific Advice Working Party

Content: This presentation highlights novel methodologies for evidence generation, the potential and opportunities of big data and real world data and gives insights of the EMA SAWP (Scientific Advice Working Party) experience with real world data

Background

The STARS consortium performed comprehensive surveys to analyse the regulatory landscape in the EU and to identify the needs and gaps in regulatory guidance for the academic health research. As a result, targeted trainings are supposed to be one of the most efficient and common supportive tool for the academic community. In line with the working programme of STARS, the consortium organised and set up a 4-day training course in basic regulatory knowledge as well as specific regulatory requirements as a best-practice example (Pilot I) for strengthening regulatory science in academia.

The aim of Pilot I was to transfer the identified best-practice example in the EU to other EEA member states. The deeper analysis of the survey data resulted in the design of a course programme adapted to the needs of the academic community to get an overview of basic regulatory knowledge as well as insights on specific regulatory requirements. The objectives of the course were:

to cover all important steps in drug development – from quality to non-clinical, clinical,
First-in-Human studies and Clinical Trial Application preparation
to introduce national and European regulatory frameworks and to give an overview of regulatory support tools for drug developers
to give the opportunity to interact with regulators and to ask questions.

Titled 'The Winding Road from a Brilliant Idea to Drug Approval' the course took place on 23-26 February 2021 as an online seminar. The curriculum of the 4-day course covered topics on essential regulatory knowledge, case examples, insights into the regulatory system, as well as direct interaction and question & answer sessions with regulators and clinical assessors.

For further specific information on support activities on national and EU level, please have a look into the STARS Comprehensive Inventory. This inventory assists European academic drug developers in finding support on regulatory affairs. The inventory lists various support services provided by national competent authorities, public actors and private entities.

For general questions, please contact: CoordinationSTARS@bfarm.de

The STARS consortium acknowledges the organisers from OGYÈI (Hungary), AIFA (Italy), and AGES (Austria), the lecturers of the course from SÚKL (Czech Republic) and MEB (The Netherlands), all other project members and the participants, who contributed to the success of the course.

The data of the course evaluation will feed into the STARS Common Strategy, which will become the STARS road map for the planning and implementation of support activities / training programmes to strengthen regulatory sciences and improve successful outcomes from regulatory scientific advice, requested by academic health researchers.