Description

This is an introductory course to European Drug Regulation and Advanced Therapies, targeted to PhD students in Medicines and Pharmaceutical Innovation (i3DU Programme), but we welcome other students or professionals from the pharmaceutical, medical, and general health sciences field. The course will include theoretical sessions, discussions, and networking opportunities

Course Program

14h00-14h15  - Opening Session



Jorge Ascenção Oliveira

Prof. Pharmacology & Neuroscience
Faculty of Pharmacy, U.Porto


Course Coordinator

Domingos Ferreira

Director Dep. Drug Sciences
Faculty of Pharmacy, U.Porto

 José Sousa Lobo

Director of the
Faculty of Pharmacy, U.Porto



14h15-15h15 - Overview of EMA and the centralized procedure for Advanced Therapies


Ana Hidalgo-Simon, Head of Specialized Scientific Disciplines, EUROPEAN MEDICINES AGENCY (EMA)


Dr Hidalgo-Simon has a degree in Medicine and Surgery from the University of the Basque Country, Spain, and a PhD from the University of London, UK. Additional qualifications include Health Economics (University of York) and Computing (University of Westminster). She currently heads the Specialised Scientific Disciplines Department, in the Human Medicines Research and Development of the European Medicines Agency (EMA). She is also the Agency’s lead for ATMPs and Biosimilars. The Department provides scientific support throughout the product lifecycle. It supports in particular the assessment work relating to the pharmaceutical quality of chemical and biological medicinal products for human use, nonclinical, clinical pharmacology and biostatistics. Previously, Dr Hidalgo-Simon worked in the Pharmacovigilance and Risk Management Sector also at EMA, as Head of Section for Risk Management and before that as Head of the Signal Detection and Data Analysis Section. Before joining EMA she held industry and academic jobs with a focus on medical communications.

Lecture Summary


Complex biological therapies offer enormous promise for the treatment and cure of currently untreatable diseases. But with that promise come serious risks. The assessment, authorisation and monitoring of these therapies is therefore both complex and key to Public Health. In the EU, these advances therapies (ATMPs) encompass genetic therapies, somatic cell therapies and engineered tissues. All ATMPs are authorised centrally via the European Medicines Agency (EMA). They benefit from a single evaluation and authorisation procedure, but this process is adapted to the specific complexity of these medicines. The Agency also gives scientific support to developers to help them design pharmacovigilance and risk management systems used to monitor the safety of these medicines. This lecture will present an overview of how the centralized procedure works and how it is applied to ATMPs, how they are classified and what is the certification procedure. Finally, it will look at approaches to mitigate their risks from early development and establish good pharmacovigilance practices.
Learning objectives
Participants will be provided with: (1) A high level summary of the centralized procedure in the EU and the classification of ATMPS (Advanced Therapy medicinal products); (2) An overview (and where to get more information) of relevant guidelines and information on incentives for the development of ATMPs in the EU; and (3)  A high level synopsis of pharmacovigilance practices for ATMPs  and how they fit in the wider context of drug safety activities.


15h15-16h00 -
Challenges & Trends in Advanced Therapies: Paving the way for innovation in Oncology

 

Fátima Ventura, Head of Scientific Evaluation Unit / Medicines Evaluation Department, INFARMED


Dr. Ventura holds a PharmD in Pharmaceutical Sciences and a PhD in Pharmacy (Biochemistry) from the Faculty of Pharmacy, U.Lisbon (FFUL). She was a PhD student at the Lab. Genetic Metabolic Disorders of the Academical Medical Center, U.Amsterdam. She is an Assistant Professor at the Dep. Biochemistry and Human Biology and a member of iMed.ULisboa, where she develops her research in the Metabolism and Genetics Group in the area of Inborn Errors of Metabolism. She has expertise in expression and characterization of recombinant human proteins. She is currently on secondment at INFARMED - National Authority of Medicines and Health Products, I.P. where she is the Head of the Scientific Evaluation Unit of the Dep. Medicines Evaluation. She is a quality assessor of chemical and biological medicinal products including advanced therapies. She is a member of the Medicines Evaluation Commission (CAM) from INFARMED. She is also the alternate Portuguese member of the Committee of Human Medicinal Products (CHMP) and the CHMP representative at the Healthcare Professionals' Working Party at the EMA.

 

Lecture Summary 


Advanced Therapies are finally becoming a reality in the armamentarium of medicinal products for treating genetic, neurologic, and oncological pathologies, among others. The development of advanced therapy medicinal products (ATMPs) surpass the typical long road of bringing medicines to patients and very often faces constraints while applying the current regulatory guidance. It is of no surprise that ATMPs are increasing among the requests for scientific advice and constitute 40% of the products eligible for PRIME (PRIority MEDicines), one of European Medicines Agency's early access routes supporting patient access to innovative medicines. This lecture will focus on the complex risk-based approaches taken in the development and assessment of cell-based and gene therapy ATMPs that are in the frontline of a fast-evolving science demanding a rapid evolution of the regulatory framework to ensure the quality and long-term safety and efficacy of these medicinal products. ATMPs are by excellence the basis of many personalised medicines where Chimeric antigen receptor (CAR) T-cell therapy and gene editing technologies will play major roles in a very near future in the diagnosis and treatment of many disorders with significant unmet medical need. Special focus will be given to the most recent advances and progress against cancer.
Learning objectives
Participants will be provided with: (1) A high level summary of the steps taken for the regulatory assessment of ATMPs under the scope of clinical trials and marketing authorisation applications; (2) An overview of approved or to be approved ATMPs and the novelties in the area with focus on CAR-T cells and gene editing technologies; and (3) An outline of the recent innovations in the field oncology.


16h00-16h30 - Coffee Break and Networking



16h30-17h00 - Gene Therapy: Dawn of a new era







Luís Pereira de AlmeidaCenter for Neuroscience and Cell Biology and Faculty of Pharmacy, U.Coimbra


Luís Pereira de Almeida holds a PharmD, a MSc and a PhD in Pharmacy (Pharmaceutical Technology) and is a tenured professor at the Faculty of Pharmacy, University of Coimbra, where he coordinated the Masters in Pharmaceutical Biotechnology. His research activity is developed at the Center for Neuroscience and Cell Biology of the University of Coimbra (CNC), Portugal where he is Principal Investigator and Vice-President. Luis did his PhD in the Gene Therapy Center of Lausanne, CHUV, Switzerland and spent short sabbatical leaves at CEA, Saclay, France (2005), at the Massachussetts Institute of Technology (MIT - 2010) and has been vice-president of the Portuguese Society for Stem Cells and Cell Therapy (2013-2015). Luis leads a talented research group focused on gene therapy approaches for brain disorders, particularly Machado-Joseph disease/spinocerebellar ataxia type 3. In 2017 one of the therapeutic strategies developed was given the Orphan Drug designation by the European Medicines Agency. Luis has been responsible for over 20 research projects and presently coordinates the National Research Infrastructure for production and experimentation with viral vectors – ViraVector, and the transnational projects SynSpread and ModelPolyQ within the European Joint Programme for Neurodegenerative Diseases (JPND).


Lecture Summary


Gene therapy aims to cure or modify the progression of acquired or hereditary diseases by adding, silencing, replacing or repairing missing, mutated or pathogenic genes. In the last decades, different viral and non-viral delivery systems - vectors for gene therapy, as well as administration methodologies have been developed. Unfortunately, issues related to the efficacy and safety of vectors as well as insufficient knowledge from mechanism of disease prevented gene therapy from rapidly reaching results in line with initial expectations. However, recent ground-breaking successes of this scientific area have have allowed the initiation of the implementation of gene therapy in clinical practice for blood disorders, such as immunodeficiencies, leukemia and haemophilia, as well as for central nervous system and eye disorders, such as spinal muscular atrophy and Leber amaurosis. Pre-clinical results suggest that an acceleration in the discovery of effective therapies for many other disorders with unmet medical needs is on-going announcing the dawn of a new era in the pharmaceutical field.
Learning objectives
In this talk participants will be provided with an overview of the main strategies of gene therapy, while highlighting previous issues, recent successes and opportunities. Examples from our own investigation involving gene therapy for the rare Machado-Joseph disease and others will also be presented.



17h00-17h30 - Orphan Medicinal Products in Europe

 

Bruno Sepodes, Chairperson of the Committee for Orphan Medicinal Products (EMA), Prof. at FFUL


Dr Sepodes holds a PharmD, a MSc in Regulatory Science, a PhD in Pharmacy (Pharmacology), and habilitation (‘Agregação’) in Pharmacology and Pharmacotherapy from the University of Lisbon. He is currently a student of the Master in Public Health of the Bloomberg School of Public Health - Johns Hopkins University. He is an Assistant Professor at the School of Pharmacy of the University of Lisbon (FFUL), where he develops his research in Pharmacology, Translational Medicine and Regulatory Science, being author of several scientific publications in international journals. Presently he is the Chair of the Committee for Orphan Medicinal Products (COMP), member of the Committee of Human Medicinal Products (CHMP) and of the Committee of Advanced Therapies (CAT) at the EMA. As Chair of the COMP, Bruno is also a member of the Scientific Coordination Board of the EMA. Nationally, Bruno Sepodes is an expert for INFARMED and the Food and Veterinary General Directorate (DGAV) and develops several works within the Board of Pharmacists.

Lecture Summary


It is estimated that 5 000 to 8 000 distinct rare diseases exist in the European Union. Although their prevalence is low, rare diseases affect 27 to 36 million people in the EU (6-8 % of the population). The development of orphan medicinal products (OMPs) is therefore an important consideration for public health policymakers seeking to address patients’ needs. The Orphan Regulation was adopted to encourage the development and market authorisation of medicinal products for rare diseases, with the main objective of the Orphan Regulation of ensuring that patients suffering from rare conditions have the same quality of treatment as any other patient in the EU. The key measures in the Regulation are: i) the establishing of the Committee for Orphan Medicinal Products (COMP) within the European Medicines Agency; ii) EMA protocol assistance for sponsors of medicinal products on the conduct of the tests and trials necessary to demonstrate their quality, safety and efficacy, or regulatory assistance; iii) 10 years of market exclusivity (in which other industry operators are prevented from entering the market with a similar product for the same therapeutic indication); iv) access to a centralised procedure allowing immediate marketing authorisation in all Member States; v) a system of reduced fees for regulatory procedures (as payable under Community rules), and; vi) a repository of all designated and authorised OMPs. Contrary to what may be believed, the orphan status of a product in Europe is not easy to obtain, is not easy to maintain, and is not meant to last forever.
Learning objectives
Participants will be provided with: (1) A high level summary of the orphan designation and orphan status maintenance procedures at the time of marketing authorisation in the EU; (2) An overview (and where to get more information) of relevant guidelines and information on incentives for the development of orphan drugs in the EU; and (3) A high level synopsis of previous and current challenges for drug development in rare diseases under the existing regulatory framework.

17h30-18h00 - Round Table Discussion




Jorge Gonçalves

President of the Scientific Council
Professor of Pharmacology
Faculty of Pharmacy, U.Porto



Domingos Ferreira

Director Dep. Drug Sciences
Faculty of Pharmacy, U.Porto

Jorge Ascenção Oliveira

Prof. Pharmacology & Neuroscience
Faculty of Pharmacy, U.Porto



18h00-18h30 - Closing Session and Porto de Honra






ORGANIZATION

Course Coordinator:
Jorge Ascenção Oliveira

Scientific Committee:
Jorge Ascenção Oliveira, Domingos Carvalho Ferreira, Jorge Moreira Gonçalves, Alberto Nova Araújo, Brígida Ribeiro Pinho

Organizing Committee & Support Staff:
MitoNeuro team: Jorge Ascenção Oliveira, Brígida Pinho, Liliana Almeida, Regina Vilela
nowScience team:
Adriano Costa, Inês Lobo, Maria João Lima, Maria José Marques



Faculty of Pharmacy, University of Porto, PT