XI Foresight Training Course

Changes in Regulatory Sciences in the EU

How to move from a reactive to a multi-stakeholders proactive attitude

Istituti Clinici Scientifici Maugeri

Via Salvatore Maugeri, 6

Pavia, Italy

25/10/2018 - 27/10/2018

Change in Regulatory Sciences is a continuous challenging process! The challenge is to align scientific and patients’ objectives with regulatory requirements along the whole development process of health products. Moreover, all the stakeholders should be aware of the criteria by which the authorisation and access to products will be measured and decided to cover patients’ specific needs. To ensure such an alignment, it is time to moving from a reactive regulation to a proactive involvement of all the involved parties, including users as well as producers. This course is aimed at involving and networking people to properly understand the many novelties of the EU system and contributing to their implementation to anticipate good medicines and other products for human health and wellbeing on the market. The present Course will discuss the EU Pharmaceutical System novelties, with special focuses on three main topics. In addition, an initiative to set up a Regulatory Network will be presented.

MAIN TOPICS

Health data and health science
Health data represent a very crucial topic due to the increasing adoption of electronic technologies allowing FAIR data collection, interpretation, sharing and reuse in the developmental process of innovative health products (“health data science”).
This large use of health data takes extraordinary advantage by the progress of the informatic technology as applied to health. In addition, ‘health data science’ should demonstrate high capacity to deal with the implementation of a new legal and regulatory approach to patients data protection and confidentiality following the General Data Protection Regulation (GDPR). A strong impact is expected from GDPR on data quality and reliability, consent and assent issues in all research activities sharing information, including clinical trials and studies.

Medical devices
In April 2017, two new Regulations were adopted, (namely Regulation (EU) 2017/745 on medical devices, and Regulation (EU) 2017/746 on in vitro diagnostic medical devices) and entered into force on 25 May 2017. These replace the existing Directives and establish a modernised and more robust EU legislative framework to ensure better protection of public health and patient safety. The legislation now being in the form of a Regulation, rather than a Directive, means that the EU law is directly applicable at national level without requiring transposition through a specific national legislation. This should allow for greater legal certainty and prevent variation applied across EU Member States.

Rare diseases and Orphan Medicinal Product Regulation time for revision?
The Orphan Medicinal Products Regulation has obtained great results in the last 18 years. However, many rare diseases are still without any treatment and the harmonisation of the real availability of therapies for patients has not been yet achieved. For these two reasons, a revision of Regulation (EC) 141/2000 is expected soon. It will be mainly aimed to foster preclinical and clinical research of new treatments for rare diseases and to harmonise best practices and procedures for orphan medicines evaluation. What is expected is to overlap the current barriers and difficulties encountered in the new products developmental process and to reduce the inequalities derived by the geographical dispersion of patients and specialised centres. Special ethical and methodological issues will be explored.

THURSDAY 25TH OCTOBER 2018

02,00 pm

Welcome address
Adriana Ceci, Maurizia Dossena

Enrico Bosone, SIAR representative
Stefano Govoni, SIF representative
Mario Melazzini, ICSM representative
Francesco Svelto, UNIPV representative
Guido Rasi, EMA representative

03,00 pm

Opening Session
The role of the patients and healthcare professionals in the regulatory framework
Chairs: Adriana Ceci, Alan G Fraser

03,00 pm
EMA experience in supporting participation of patients and healthcare professionals in EU medicines regulation
Ivana Silva
EMA

03,30 pm
How has the role of patients got stronger and what they have learnt so far
François Houÿez
EURORDIS

04,00 pm
The evolving role of healthcare professionals in the regulatory field
Giovanni Migliaccio
Consorzio per Valutazioni Biologiche e Farmacologiche

04,30 pm
The COST programme to create networks in the scientific setting
Valentina Cardinale
Giulia Rotundo
Ministero dell’Istruzione, dell’Università e della Ricerca

05,00 pm
Presentation of COST proposal
Viviana Giannuzzi
Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus; EMA-PDCO

05,30 pm
Discussion

FRIDAY 26TH OCTOBER 2018

09,00 am

First Session
Medical Devices: the new era in Europe
Chairs: Carla Caramella, Maurizia Dossena

09,00 am
Novelties and implementation of the Regulation 745-2017
Alan G Fraser
European Society of Cardiology; Cardiff University

09,30 am
The new Regulations on medical devices and on in vitro diagnostics: a real revolution?
Franco Gattafoni
ACCREDIA

10,00 am
The challenges of the new Regulation from the Notified Bodies point of view
Luciana Gramiccioni
EUROFINS

Coffee break

11,00 am
The new provisions introduced by rule 21
Antonella Mamoli
IBSA Farmaceutici Italia

11,30 am
The manufacturing and control of medical devices in the perspective of the new regulation
Fabio Geremia
CTP System, Akka Life Sciences

12,00 am
The new regulation and the management of software and applications
Cettina Garufi
ACCREDIA

12,30 am
Conclusive remarks:
Carla Caramella, Maurizia Dossena

01,00 pm
Light lunch

02,00 pm
Second Session
Data Science supporting medicines and healthcare development
Chairs: Fedele Bonifazi, Victoria Hedley

02,00 pm
Big data & life sciences
Graziano Pesole
Consiglio Nazionale delle Ricerche, University of Bari, ELIXIR

02,30 pm
Data integration systems to support scientific research and patient enrolment in clinical studies
Riccardo Bellazzi
Istituti Clinici Scientifici Maugeri, University of Pavia

03,00 pm
When rare becomes FAIR: a discussion on how to manage sensitive, rare disease data to enable large scale analysis
Marco Roos
Leiden University Medical Centre

03,30 pm
How to protect patients data in the light of the new Regulation?
Deborah Mascalzoni
CRB Uppsala University

04,00 pm
Industry perspective and Novartis experience
Roberto Orsenigo
Novartis Farma S.p.A.

04,30 pm
Conclusive remarks:
Claudio Carta, Paola Baiardi

SATURDAY 27TH OCTOBER 2018

09,00 am

Third Session
Orphan products and perspectives on the horizon
Chairs: François Houÿez, Donato Bonifazi

09,00 am
What member states have done so far at EU and national level
Agenzia Italiana del Farmaco representative

09,30 am
Health outcomes of orphan medicines
Joseph Torrent-Farnell
Catalan Health Service

10,00 am
The demonstration of significant benefit in the EU framework
Laura Fregonese
EMA

10,30 am
Developing Advanced therapies for rare diseases in EU: opportunities and challenges in the experience of a Charity
Michela Gabaldo
Fondazione Telethon

11,00 am
The complexity of developing innovative medicines for rare diseases
Diego Ardigò
Chiesi Farmaceutici

11,30 am
Are patients satisfied from the implementation of EU policies on orphan medicines development and availability so far?
Dimitrios Athanasiou
World Duchenne Organization; EMA-PDCO

12,00 am
Researchers, networks, new funds for rare diseases
Daria Julkowska
E-Rare

12,30 am
Conclusive remarks: expectations from the Orphan Medicines Regulation revision
Viviana Giannuzzi, Enrico Bosone

Course Scientific Committee

Paola Baiardi | Istituti Clinici Scientifici Maugeri
Enrico Bosone | Società Italiana Attività Regolatorie
Maurizia Dossena | Università di Pavia
Viviana Giannuzzi | Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus