HM1: Topic to be announced
Leader and Chair: Daniela Drago, Senior Director, Regulatory Sciences, Biogen, USA

HM2: 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.

HM3: Reliance, worksharing and recognition as 21st century regulatory tools
Leader and Chair: 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.

HM4: 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
• Tomáš Boráň, Director of the Marketing Authorisation Section, SUKL, Czech Republic; Committee for Advanced Therapies (CAT), alternate 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, insights on the progress of the new EAEU legislation, discussion on acceptance of clinical trials data from the EU to EAEU regions and vice versa, differences between the current and new requirements for managing clinical trials in EAEU in the face of forthcoming changes. 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.

TOPRA Annual Lecture

Speaker to be announced.

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.

HM5: Clinical trials of the future – patient oriented and digitally connected
Leader 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.

HM6: Regulatory progress in delivering on the promise of ATMPs
Leaders and Chairs:

• Tomáš Boráň - Director of the Marketing Authorisation Section, SUKL, Czech Republic; Committee for Advanced Therapies (CAT), alternate member for Czech Republic
• Daniela Drago, Senior Director, Regulatory Sciences, Biogen, 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.

HM7: 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.

HM8/MD4: 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

Session description to be announced.

HM9: Avoiding disastrous mistakes with an effective regulatory strategy
Leaders:

• Daniela Drago, Senior Director, Regulatory Sciences, Biogen, 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.