Regulatory Rapporteur


December 2023  |  Volume 20  |  No.11


Held annually since 2004, the TOPRA Symposium is Europe’s premier conference for healthcare regulatory affairs with dedicated programme streams for human medicines, medical devices and IVDs, and veterinary medicines. This year’s TOPRA Symposium was hosted in Lisbon, Portugal, in partnership with Infarmed, Portugal’s national authority of medicines and health products. In addition to hearing the latest insights and developments running over multiple parallel sessions, it is also provided valuable opportunities to network and make new contacts.

We started the Human Medicines Symposium sessions with an update on EU new regulatory legislation and a view from different angles, followed by discussion on improving the odds of regulatory success. Measures to improve access to medicines will, however, remain difficult to overcome because the majority of access barriers must be resolved at national level. This issue of national versus global became a recurring theme throughout the three days. Bringing a drug to the market is a lengthy and costly process with lots of complexities and uncertainties. Developing the best regulatory strategy at all stages of the process and in consultation with relevant regulatory authorities can help avoid huge pitfalls. The bonus of being in person rather than virtual allowed for an interactive session whereby five real-life situations were presented to the audience before attendees were divided into groups to discuss and propose their answers. We then moved into the fireside chat session where the discussion spotlighted many challenges including resourcing, communication and transparency, scientific advice, patient involvement in the regulatory processes and collaboration among agencies. The human medicines sessions then moved into a more diverse agenda, covering everything from the clinical trial landscape to paediatric investigation plans, real-world evidence, health technology assessments and regulatory mechanisms for expedited development.


Francesca Buttegieg

The Medical Devices and IVDs Symposium streams kick-started with a joint human medicines session on the process for regulations and implications of different legislation. This is currently a hot topic of how to follow and decide which regulation will impact your product development. Specific sessions then followed investigation and discussion of the multiple challenges within these sectors, for example, those associated with medical device software, in the post-market setting, in a divergent global setting and on clinical investigations and performance testing.

For the Veterinary Medicines Symposium, this year’s sessions focused more on the positive opportunities available, rather than the challenges, including digital innovations like the Big Data strategy and the DECIDE project to support animal and public health, among other new approaches and initiatives.

Only one parallel session took place for the SME, academia and start-ups this year. It focused on regulatory support, including authority interactions. However, peppered within the other streams, a number of topics were either directly applicable for SMEs, or had some relevance, for example PS2 on advanced therapy medicinal product (ATMP) innovations and clinical trials with genetically modified organisms (GMOs). The key messaging was of early agency engagement to facilitate development.

Miguel Forte’s annual lecture describing the fantastic opportunities in cell and gene therapies (including CAR-T cell and gene editing) for patients and other stakeholders in the pharmaceutical industry. He described a complex business with significant investment and value to patients in oncology and rare diseases. The future looks to gene editing, engineered cell and synthetic biology, but to enable this continued development, there was a call for new financing and reimbursement models, and jointly, we have to ensure the sustainability of the field for the benefit of patients.

All sessions highlighted the dynamic nature of our industry, and although there are many challenges and uncertainties to overcome, there has certainly been rapid innovation and the regulatory landscape continues to advance at pace. To allow this advancement, regulatory professionals must also evolve and events such as the Symposium provide a wonderful platform for this continuing professional development.

Please mark your diary for the TOPRA 2024 Symposium which will be taking place in Rotterdam between 30 September and 2 October, in association with the Netherland’s regulator, the Medicines Evaluation Board. We look forward to seeing many colleagues for more interaction and communication.

Finally, on behalf of the Editorial Team, and all those at TOPRA, in the last issue of the year, we wish everyone season’s greetings and a very happy and healthy 2024!