Programmes

See below for the most up-to-date programme details for the 2021 TOPRA Symposium. Additional information about these sessions will be announced as they are finalised.

Human Medicines Symposium 2021

22 September

All times CEST (GMT+2)
11:10 – 12:40

HM1: A spotlight on CEE – Challenges and Opportunities in Central and Eastern European Countries
Leaders and Chairs: Eva Kopecna, Global Head of Regulatory Affairs, Medical and Pharmacovigilance, Accino, Switzerland and Tomáš Boráň, Director of the Marketing Authorisation Section, SÚKL, Czech Republic; Committee for Advanced Therapies (CAT), member for Czech Republic

This session will focus on key registration challenges and opportunities in Central and Eastern European countries. Industry experts and regulators will discuss hot topics. These include the EU procedures and choice of RMS in CEE countries or insights on the progress of GMP inspections in EAEU. The session will also provide a platform for discussion on the impact of COVID-19 on the work of drug agencies, priorities and inspections activities.

Speakers:

  • Tomáš Boráň, Director of the Marketing Authorisation Section, SÚKL, Czech Republic; Committee for Advanced Therapies (CAT), member for Czech Republic
  • Nataliya Burlakina, GMP Inspector, State Institute of Drugs and Good Practices, Russian Federation
  • Filip Vrubel, Executive Director, Czech Association of Pharmaceutical Companies, Czech Republic
12:40 – 13:10 Break
13:10 – 13:55

HM2: Fireside Chat with Emer Cooke - A Review of Current and Future Priorities
Leader: Daniela Drago, Chief Regulatory Officer, Aurion Biotech, USA

We are excited to announce that Emer Cooke, Executive Director of the European Medicines Agency (EMA), will join us for a live fireside chat on the first day of the symposium! Ms. Cooke began her mandate as Executive Director of the European Medicines Agency in November 2020. She brings to the Agency more than 30 years of experience in international regulatory affairs, 18 years of which were in leadership roles. Ms. Cooke worked for the pharmaceutical unit of the European Commission from 1998 to 2002 and at EMA between 2002 and 2016, where she held positions including Head of Inspections and Head of International Affairs. Between November 2016 and November 2020, she was responsible for all medical product-related regulatory activities at the World Health Organization in Geneva. Ms. Cooke is the fourth Executive Director of EMA and the first woman at the Agency's helm. Please join this conversation moderated by the Chair of the 2021 Human Medicines Symposium, Daniela Drago.

Speaker: Emer Cooke, Executive Director, European Medicines Agency (EMA) 

14:05 – 14:50

HM3: The evolution of the EMA-EUnetHTA collaboration - bridging of decision makers
Chair: Carlos Langezaal, Senior Director, Global Regulatory Affairs, BMS, USA

In 2010 the European Medicines Agency (EMA) and the European network for Health Technology Assessment (EUnetHTA) initiated a collaboration. During EUnetHTA Joint Action 3, the EMA-EUnetHTA collaboration continued per the work plan 2017-2021 and this has concluded at the end of May 2021. By working together, EMA and EUnetHTA were able to help medicine developers enhance clinical research and become more efficient in generating the evidence relevant for both regulatory authorities and HTA bodies (HTAs). New ways to generate evidence to address both regulatory and HTA information needs, such as the use of patient registries, have also been encouaged by the collaborating parties. The European Commission is coming with a proposal for the HTA regulation, and in the interim the EUnetHTA Heads of Agencies has been created and the group has decided to continue its function and body. It is now creating its own independent framework with the aim to prepare its members for the future EU HTA system envisioned in the HTA Regulation.

In this session, the EMA and EUnetHTA representatives will provide a short presentation on the view and experiences from both organisations on the collaboration and the future, followed by a Q&A session with practical examples.

Speakers: 

  • Michael Berntgen, Head of Product Development Scientific Support Department, European Medicines Agency (EMA)
  • Niklas Hedberg, Chief Pharmacist, EUnetHTA / TLV, Sweden
  • Flora Giorgio, Deputy Head of Unit B6, DG Sante, European Commission
14:50 – 15:30   Networking break 
15:30 – 17:00

HM4: Real world evidence in regulatory decision-making
Leader and Chair: Francesca Buttigieg, Associate Director Regulatory Affairs, PTC Therapeutics, Switzerland

In an increasingly fast-paced world in which technology is continuously evolving, the healthcare environment has been keeping up with the times. The use of mobile applications, smartwatches and other wearables have lead to the collection of valuable data. This has brought the importance of real world data to the forefront of the pharmaceutical industry.

The transformation of this vast amount data into real world evidence is currently being applied to the diagnosis of diseases, clinical trials, and also in marketing authorisation applications (MAAs) and new drug applications (NDAs). Within regulatory the focus on real world evidence is becoming more prominent and keeping abreast of any developments is now becoming a priority for all.

Chair: Francesca Buttigieg, Associate Director Regulatory Affairs, PTC Therapeutics, Switzerland

Speakers:

  • Fred Senatore, Medical Officer, Clinical Team Leader Division of Cardiology and Nephrology Office of New Drugs, Center of Drug Evaluation Research, U.S. Food and Drug Administration, USA
  • Rob Kalesnik-Orszulak, Director, Regulatory Innovation Lead for RWE & Data Science, Global Regulatory Strategy & Policy, BMS, USA
  • Xavier Kurz, Head of Data Analytics, European Medicines Agency (EMA)
17:00 – 17:30  Networking break
17:30 – 19:00

HM5: Reliance, worksharing and recognition as 21st century regulatory tools
Leader and Chair: Francesca Buttigieg, Associate Director Regulatory Affairs, PTC Therapeutics, Switzerland

Within the healthcare setting, the aim of all parties is to bring effective and safe medications to patients as quickly as possible. Doing so in multiple countries in as short a timeframe as possible is an even greater common goal. The simultaneous assessment by numerous health agencies of the same registration dossier is not a new concept, taking the mutual recognition procedure within the EU as an example. These types of initiatives or partnerships have extended into multiple countries worldwide and even across regions. This has most recently been observed in the COVID-19 setting and the information sharing for vaccinations and medicines between the EMA and Health Canada.

During this session we intend to delve into the intricacies of the Access Consortium, the African Medicines Regulatory Harmonisation Initiative and the Eurasian economic union.

Speakers:

  • Murray Lumpkin, Deputy Director Integrated Development (Lead for Global Regulatory Systems Initiatives), Bill & Melinda Gates Foundation
  • Martin Harvey Allchurch, Principal International Affairs Officer, European Medicines Agency
  • Heidi Wang, Vice President, Head of Oncology Global Regulatory Sciences, BMS, US

 

23 September

 

All times CEST (GMT+2) 
11:15 – 11:55

TOPRA Annual Lecture: From ‘knocking on the door’ to co-creation: 25 years of patient engagement in R&D
Virginie Hivert, Therapeutic Development Director, EURORDIS France

Patients and their families are strong advocates and active players when it comes to developing treatment for their own diseases or those of their loved ones. This session is the opportunity to share some thoughts, experiences and insights from the point of view of a patient representative on the importance of educating all the stakeholders involved, the evolution and current trends in patient engagement.

12:00 – 13:00

TOPRA Annual Review Meeting

We invite all TOPRA members to attend the Annual Review Meeting to hear about TOPRA’s financial results for the previous year, key activities and achievements for the current year and plans for the next year. The meeting is an opportunity to be introduced to new Board Directors and celebrate the various awards and honours conferred by the Board to recognise significant contributions within our valued TOPRA Community.

13:00 – 13:45 Networking Break
13:45 – 15:15

HM6: Clinical trials of the future – patient oriented and digitally connected
Leaders and Chairs:

  • Susan Bhatti, Director EU Global Regulatory and Scientific Policy, Merck BV, the Netherlands
  • Carlos Langezaal, Senior Director, Global Regulatory Affairs, BMS, USA

Traditionally, clinical trials require patient enrolment, care and monitoring to be done on-site and in-person, which often limits access to participants who are located close to approved sites. This approach impacts both trial recruitment and retention and leads to a high burden for patients, who have to repeatedly visit the site, coupled with high study costs. The COVID-19 pandemic forced a re-think in the conduct of trials to a more decentralised model, which could transform clinical research by using digital technologies to enrol and monitor patients, resulting in more inclusive, flexible, connected and patient-centred studies.

In this session, the opportunities and limitations of decentralised clinical trials will be discussed by regulators, including lessons learned from regulatory flexibilities introduced in response to the COVID pandemic. The IMI project “Trials@Home” will be presented by an industry representative from the consortium, describing in particular the development of an innovative online platform designed to connect with patients and the general public.

Speakers:

  • Tanja Keiper, Director Clinical Trial Execution, Clinical Delivery Unit, Merck Healthcare, Germany
  • Isaac Rodriguez-Chavez, Senior Vice President, Scientific & Clinical Affairs Head, Icon, US
  • Fergus Sweeney, Head, Clinical Studies and Manufacturing Taskforce, European Medicines Agency (EMA) 
Panellist:
  • Ann-Marie Janson Lang, CTFG co-chair, MPA, Sweden
15:15 – 16:00 Networking Break
16:00 – 17:30

HM7: Regulatory progress in delivering on the promise of ATMPs
Leaders and Chairs:

  • Tomáš Boráň - Director of the Marketing Authorisation Section, SÚKL, Czech Republic; Committee for Advanced Therapies (CAT), member for Czech Republic
  • Daniela Drago, Chief Regulatory Officer, Aurion Biotech, USA

There is mounting pressure to improve performance and facilitate timely access to safe, effective, and quality cell and gene medicinal products on regulators, industry officials, and other stakeholders. The task has become even more challenging due to globalization, increasingly complex technologies, negotiations with healthcare payers and growing public expectation. This session brings together different perspectives to discuss the ability of regulatory frameworks to deliver advanced therapies to patients globally and to discuss what are the main hurdles of entry of ATMPs to the market. It will consider whether existing tools, such as facilitated regulatory pathways and early dialogue, are living up to their aspirations and meet the demands of stakeholders to address unmet medical need in this rapidly evolving sector. Join us for a discussion on challenges and opportunities of fostering systems that facilitate innovation, ensure patient safety, and promote public health.

Speakers:

  • Wilson W. Bryan - Director of the Office of Tissues and Advanced Therapies at the Center for Biologics Evaluation and Research (CBER), USA
  • Ana Hidalgo-Simon - Head of Advanced Therapies, European Medicines Agency (EMA)
  • Angelo Loris Brunetta, Board member of Thalassaemia International Federation and former member of the Committee for Orphan Medicinal Products (COMP), Italy

Panellists:

  • Tomáš Boráň, Director of the Marketing Authorisation Section, SÚKL, Czech Republic; Committee for Advanced Therapies (CAT), member for Czech Republic
  • Michael Ladd, VP Regulatory Science, Orchard Therapeutics, USA

 

24 September

 

All times CEST (GMT+2)
  Welcome to Day 3
12:15 – 13:45

HM8: Enhancing dialogue to foster innovation
Leader and Chair: Susan Bhatti, Director EU Global Regulatory and Scientific Policy, Merck BV, the Netherlands

In the rapidly evolving and complex landscape of medicine development, an iterative, responsive and holistic regulatory dialogue – with flexibility in the delivery of advice and alignment across stakeholders – is needed to reflect the changing pace and process of science and innovation along the development continuum.

The recent example of the COVID-19 pandemic challenges emphasises the importance of proactive measures and time-efficient interactions and offers useful learning tools for a future system.

This session will discuss a future strategy for enhanced holistic and multi-stakeholder dialogue and advice considering global drug development approaches.

Chair: Susan Bhatti, Director EU Global Regulatory and Scientific Policy, Merck BV, the Netherlands 

Speakers:

  • Carlos Langezaal, Senior Director, Global Regulatory Affairs, BMS, USA
  • Iordanis Gravanis, Head of Scientific Advice, European Medicines Agency (EMA)
  • Claudia Hey, Senior Director Global Regulatory and Scientific Policy Europe, Merck Healthcare, Germany
13:45 – 14:30 Networking Break
14:30 – 16:00

HM9MD5: Innovation in drug-device combinations – how to navigate a complex regulatory environment?
Leader and Chair: Bjorg K. Hunter, Department Manager, Regulatory Affairs NextGen Drug-device, Novo Nordisk, Denmark

Innovation in drug delivery has been on the rise in recent years. This has been a result both of a more patient centric focus, new technology development and a wish for more and better at home treatment. Further to this the trend has been to integrate the medicine and the delivery device into one product to reduce user steps and for easier access. The introduction of the Final Rule of Combination products in the United States as well as the update to the MDR and introduction of Article 117 in Europe are examples of regulatory pathways also changing to meet the challenges in regulating these new innovative solutions. However, as medicines are still primarily regulated though the medicinal offices in the Health Authorities it becomes very complex to navigate regulatory interactions for innovative drug-device combination products. This session will focus on discussions between regulators and industry to share best practises and ideas on how it is possible to have meaning interactions on early innovation.

Speakers:

  • Christelle Bouygues, Regulatory Affairs Officer, European Medicines Agency (EMA)
  • John (Barr) Weiner, Associate Director for Policy and Product Classification Officer, US FDA
  • Manfred Maeder, Global Head Device Development & Commercialization, Novartis, Switzerland
Panellist:
  • Nada Alkhayat, Policy Officer – Medical Devices, Unit B6, DG Sante, European Commission
16:00 – 16:45 Networking break
16:45 – 18:15

HM10: Avoiding disastrous mistakes with an effective regulatory strategy
Leader:

  • Daniela Drago, Chief Regulatory Officer, Aurion Biotech, USA, Carlos Langezaal, Senior Director, Global Regulatory Affairs, BMS, USA
  • Fred Senatore, Medical Officer, Clinical Team Leader Division of Cardiology and Nephrology Office of New Drugs, Center of Drug Evaluation Research, U.S. Food and Drug Administration, USA

Obtaining a new product approval quickly is one of the most critical factors shaping the pharmaceutical industry’s performance. Getting a new drug through the approval process on the first attempt is likely to be worth millions of dollars in additional revenue. Companies hoping to speed their drug candidate to market fear the impact that a delay would have on their bottom line. This is where a robust regulatory strategy can make all the difference! We invite you to participate in an interactive session that covers how to develop and successfully implement a regulatory strategy. We will take a deep dive into appropriate steps to take to avoid disastrous mistakes. You will analyse different scenarios and will have an opportunity to compare your answers with panellists, who will provide practical advice on how to avoid potential pitfalls.

Speakers

  • Fred Senatore - Medical Officer, Clinical Team Leader Division of Cardiology and Nephrology Office of New Drugs, Center of Drug Evaluation Research, U.S. Food and Drug Administration, USA
  • Carlos Langezaal - Senior Director, Global Regulatory Affairs, BMS, USA
  • Daniela Drago - Chief Regulatory Officer, Aurion Biotech, USA

*There will not be a recording or slides available for this session.

  End of the Human Medicines Symposium

 

No content found

No content found

No content found


TOPRA Symposium 2021

Contact us
meetings@topra.org
+44 (0)207 510 2560

#TOPRASym21

Exhibition / Sponsorship
Call +44 (0)2076 510 2573
or email erik@topra.org