Programmes

See below for the most up-to-date programme details for the TOPRA Annual Symposium.

Human Medicines Symposium 2022

17 October

  All times CEST (GMT+2)
  12:40 – 14:10

HM1: A decade of innovative clinical trials: What have we learned and where are we going?

Leads: 

  • Susan Bhatti - Director EU Global Regulatory and Scientific Policy, Merck BV, the Netherlands
  • Francesca Buttigieg - Associate Director Regulatory Affairs, PTC Therapeutics, Switzerland

Adaptive protocols and trial designs that allow enrichment of the target population have been available for many years and their use has been steadily increasing. Experience gained through these innovative approaches has enabled trial designs to evolve further and benefit patients by using historical data to replace control arms, as well as using biomarkers and basket/umbrella/platform designs to investigate multiple treatments or multiple diseases in the same clinical trial. Regulatory agencies have supported the use of innovative designs by providing a number of guidelines and/or reflection papers. However, balancing the need to foster innovation with concerns by some stakeholders around potential bias and impact on trial oversight and transparency is challenging. The Accelerating Clinical Trials in the EU (ACT EU) initiative outlines further steps that the EMA, Heads of Medicines Agencies and the European Commission are proposing to foster innovation in clinical trials in Europe and reverse the trend of decreasing numbers of trials in Europe.

Speaker:

  • Mireille Muller - Regulatory Policy Executive Director, Novartis, Switzerland
  • Anja Schiel - Alternate Member, Scientific Advice Working Party at European Medicines Agency, Norwegian Medicines Agency, Norway
  • Marianne Lunzer - PV und CT Safety Assessor, Austrian Medicines and Medical Devices Agency (AGES), Austria

 

14:10 – 14:50

Networking Break
  14:50 – 16:20

HM2: Are expedited programs delivering on the promise to accelerate drug development and patient access?

Leads:

  • Laura Liebers - Director, International Regulatory Policy & Intelligence, Vertex, UK
  • Carlos Langezaal - Senior Director, Global Regulatory Affairs, BMS, USA

Expedited regulatory pathways such as FDA’s Break Through Designation (BTD), Regenerative Medicine Advanced Therapy (RMAT) designation, EMA’s PRIME priority medicines scheme and MHRA’s Innovative Licensing and Access Pathway (ILAP) are intended to facilitate and accelerate the development and approval of new promising medicines addressing areas of unmet need for the treatment of serious or life-threatening conditions.
But how have these pathways been used so far and are they delivering on their promise? In this session we will provide the audience with a global overview of these different pathways and get you the latest updates on where these different schemes are heading. Please join us for this session to hear directly from regulators and engage with an experienced panel also comprising of senior industry representatives.

Speakers:

  • Fortunato 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 (FDA), USA
  • Daniel O’Connor - Deputy Director, Innovation Accelerator and Regulatory Science, MHRA, UK
  • Francesca Cerreta - Principal Scientific Office, European Medicines Agency (EMA)

Panellist:

  • Tim Stonehouse - Vice President International Regulatory Strategy, Vertex, UK

 

16:20 – 17:00

Networking Break
  17:00 – 18:30

HM3: Lessons learned and strategic priorities

Leaders and Chairs:

  • Daniela Drago - Expert Consultant, NDA Partners, USA
  • Sabine Haubenreisser - Principal Scientific Administrator, Stakeholders and Communication, European Medicines Agency (EMA)

We are excited to announce that Emer Cooke, Executive Director of the European Medicines Agency (EMA), Christa Wirthumer-Hoche, Head of The Austrian Agency for Health and Food Safety (AGES), and Robert Califf, Commissioner of the US Food and Drug Administration (FDA) will join us for a keynote address on the first day of the symposium! In a conversational style session, these three top leaders will have a more global discussion on strategies to engage with new technology and fast evolving science and to create the right environment to foster innovation and research and enable their translation into patient access. The discussion will cover a wide range of initiatives and issues, focusing on lessons learned during the pandemic and strategic priorities. The pandemic continues to impact our global healthcare enterprise. Antimicrobial resistance and other emerging health threats continue to make headlines. Those are just some of the topics that will be addressed — all of which touch nearly every healthcare organization. This is a conversation you certainly will not want to miss!

Speakers:

  • Robert Califf - Commissioner of Food and Drugs, U.S. Food and Drug Administration (FDA), USA 
  • Emer Cooke - Executive Director, European Medicines Agency (EMA)
  • Christa Wirthumer-Hoche - Head of Austrian Medicines and Medical Devices Agency (AGES), Austria

 

18 October

 

  All times CEST (GMT+2) 
  9:00 – 10:30

HM4: Assessing the value of innovative therapies: trends, challenges, and learnings

Lead:

  • Carlos Langezaal - Senior Director, Global Regulatory Affairs, BMS, USA

The new EU Regulation on Health Technology Assessment (HTA) was adopted in December 2021 entered into force in January 2022 and will be implemented as of January 2025.  In this session, the EUnetHTA representative will set the stage with the Regulation, what activities take place and challenges may lay ahead during the three year transition period. The EMA representative will then discuss joint activities at the regulatory / HTA interface in terms of evidence planning and assessment methodologies, with a view to facilitate sequential decision making under the new Regulation. To close it out, The Centre for Innovation in Regulatory Science Representative will present the evolving regulatory- HTA landscape in other regions/countries around the world, which the pharmaceutical industry should also consider for global development plans. 

Speaker:

  • Claudia Wild - CEO, Austrian Institute for Health Technology Assessment (AIHTA), Austria
  • Michael Berntgen - Head of Product Development Scientific Support Department, European Medicines Agency (EMA)
  • Tina Wang - Senior Manager, HTA programme and Strategic Partnership, Centre for Innovation in Regulatory Science (CIRS), UK

 

10:30 – 11:00

Networking Break
  11:00– 11:45

TOPRA Annual Lecture

  11:45 – 12:30

TOPRA Annual Review Meeting 

  12:30 –  13:30 Lunch Break
  14:00 – 15:30

HM5MD1: Digital health opportunities and advancements in healthcare

Lead:

  • Bjorg Hunter - Department Affairs Director, Regulatory Affairs Digital Health, Novo Nordisk, Denmark

The advancements in Digital Health are on a steep increase, further fuelled by the restrictions of COVID-19. During the lockdown period it became even more apparent how digital solutions can be a significant help for both patients and HCPs in managing disease. Digital Heath can also be many things: from simple log book apps and health monitoring devices to complex algorithms and Artificial Intelligence (AI) capable of providing insight and recommendations to care. As digital tools become more and more advanced so will the regulatory landscape around it. Of course, patient safety is always at the core and because of this we have to collectively find ways that ensure patient safety while also encouraging innovation. Organisations around the world including WHO, EMA and FDA, have therefore developed a Digital Health strategy and so will most pharmaceutical and Medical Device companies. This session will explore the complexity of regulating Digital Health in an ever evolving world.

Speaker:

  • Vikas Jaitely - Head Digital Health and Devices, Merck, Switzerland
  • Søren Smed Østergaard - Vice President Digital Health, Novo Nordisk, Denmark

Panellist:

  • Georg Neuwirther - Head of IT, Austrian Medicines and Medical Devices Agency (AGES), Austria

 

15:30 – 16:10

Networking Break
  16:10 – 17:40

HM6: Innovation in regulatory science: The path from data to evidence and the promise to streamline the regulatory lifecycle

Leads:

  • Guenter Waxenecker - Nonclinical and Biological Assessor, Austrian Agency for Health and Food Safety (AGES), Austria
  • Fortunato 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 (FDA), USA 

In an increasingly fast-paced world in which technology is continuously evolving, new opportunities to accomplish public health tasks arise for the healthcare environment and regulatory systems. For example, mobile applications, smartwatches, and other wearables have led to the collection of valuable data. While the RCT is still considered the gold standard for evidence generation of new medicines key stakeholders in the regulatory environment consider an emerging role for the transformation of this vast amount of data into real-world evidence to diagnose diseases or for the purpose of benefit risk evaluations for marketing authorization applications (MAAs) and new drug applications (NDAs). Especially in situations where the currently applied development paradigm of RCTs seems limited by feasibility constraints and data from non-pivotal clinical (and even nonclinical) studies together with (other) RWD gain critical relevance for regulatory decision-making.

More transformational initiatives are on the way. For example, a consensus is growing that there is a need to transform the submission, review, and exchange of data between company sponsors and regulators in the approval of medicinal products. A cloud-based platform (or equivalent) could house a much more dynamic and iterative exchange transforming the dialog between regulatory authorities and company sponsors by defining the future of data exchange without dropping hard-learned key requirements such as database lock, data integrity and prospective design. Accumulus-Synergy is actively working with regulatory authorities such as the US FDA, the EMA, the Pharmaceutical and Medical Devices Agency (PMDA) in Japan and others to define the path forward through practical means—for example, by establishing initial use cases that benefit both industry and regulators by tackling common pain points. This is a new approach that is not merely optimizing current document-based transactional systems but is re-imagining an entirely new direction, where authorities are increasingly involved in clinical data management and evaluation providing sufficient flexibility but also assurance on human safety and efficacy at least equivalent to that provided by current clinical development paradigms. We invite you to participate in this session, where we will cover these different aspects of innovation in regulatory science.

Speakers:

  • Fortunato 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 (FDA), USA
  • Frank Nogueira - CEO, Accumulus Synergy, USA
  • Almath Spooner - Director Global Regulatory Policy and Intelligence, Abbvie, Ireland

Panellist:

  • Christian Gartner - Biostatistician, Senior Expert, Austrian Medicines and Medical Devices Agency (AGES), Austria

 

19 October

 

  All times CEST (GMT+2)
  9:00 – 10:30

HM7: Are patients still the missing piece in the global drug development puzzle?

Leads:

  • Susan Bhatti - Director EU Global Regulatory and Scientific Policy, Merck BV, the Netherlands
  • Sabine Ellenberger - Director, International Regulatory Affairs, Blueprint Medicines, Switzerland

This session will look at the importance of patient engagement for developing meaningful endpoints in the treatment of diseases and consider how patient input is essential to shape the approach of regulators and pharma companies when setting up clinical trials for medicines and devices. The outcome of initiatives by Regulatory Agencies to involve patients in scientific advice and use patient evidence in the risk-benefit assessment of drug applications will be discussed, as well as how lessons learned may impact requirements to bring in the patient voice in future.

Speakers:

  • Jo Dewhurst - Director, Therapeutic Expertise, ICON plc, UK
  • Maria Mavris - Patient Relations, European Medicines Agency (EMA)
  • Claas Röhl - Patient representative, NF Patients United, EUPATI, Austria

 

10:30 – 11:00

Networking Break
  11:00 – 12:30

HM8: The present and future of developing and commercializing cell and gene therapies in a global world

Lead:

  • Francesca Buttigieg - Associate Director Regulatory Affairs, PTC Therapeutics, Switzerland

The development of advanced therapy medicinal products (ATMPs) presents challenges in their development, regulatory review, and commercialization. Such therapies are extensively heterogenous and current regulations are still evolving. Additionally, ATMPs are expensive to develop and manufacture, their uniqueness and low batch volumes makes their price and reimbursement application difficult to assess, thereby making commercialization more complex than for other modalities. This has already led to some product withdrawals. Health Authorities have been keeping abreast with the advancements of ATMPs, however the field often calls for more flexibility and adaptability. Regulatory convergence of requirements and other 21st century regulatory tools would assist global development and approvals of such products.

Speakers:

  • Martina Schuessler-Lenz -Paul-Ehrlich-Institut, Germany, Chair of the Committee for Advanced Therapies (CAT), European Medicines Agency (EMA)
  • Julie Taccoen - Senior Director, Regulatory Affairs, PTC Therapeutics, France
  • Frank-Ulrich Fricke - Health Economist, University of Nurnberg, Germany

Panellist:

  • Patrick Celis - Scientific Administrator, Committee for Advanced Therapies (CAT), European Medicines Agency (EMA)
  • Silke Dorner - Assessor, Austrian Medicines and Medical Devices Agency (AGES), Austria


 

12:30 – 13:10

Lunch Break
  13:10 – 14:40

HM9: Strategy under uncertainty – Improving the odds of regulatory success

Lead:

  • Daniela Drago - Expert Consultant, NDA Partners, USA

Getting a new drug through the approval process at first attempt is worth on average more than $630 million in additional revenues. Companies hoping to speed their drug candidate to market fear the impact that a delay might have on their bottom line. These are times where a regulatory strategy can make all the difference! We invite you to come participate in an interactive session that will cover regulatory strategies under uncertainty. We will take a deep dive into appropriate steps to take to increase the probability of regulatory success. You will work with your colleagues to analyze different scenarios. Then you can compare your answers with a panel of industry leaders and FDA officials, who will provide practical advice. Hopefully, many of you in the audience will be inspired and learn about how to successfully contribute to an effective regulatory strategy.

Speakers:

  • Fortunato 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 (FDA), USA
  • Carlos Langezaal - Senior Director, Global Regulatory Affairs, BMS, USA
  • Daniela Drago - Expert Consultant, NDA Partners, USA

    End of the Human Medicines Symposium

 

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TOPRA Symposium 2022
Hilton Vienna Park, Vienna, Austria
Am Stadtpark 1, 1030

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+44 (0)207 510 2560

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