RSNN Regulatory Science session at the virtual DIA 2020 Europe Meeting

Pieter Stolk (Lygature), on behalf of the Regulatory Science Network Netherlands (RSNN), kicked off the session with an introduction to regulatory science and how this session wants to explore how regulatory science shapes policy with a number of case examples presented during this session. Two speakers, Bert Leufkens, professor of Pharmaceutical Policy and Regulatory Science at Utrecht University & chair of the RSNN, and Larry Liberti, Head of Regulatory Collaborations at the Centre for Innovation in Regulatory Science (CIRS), shared their experiences on collaborative research to inform regulation and policy-making. This was followed by an interactive panel discussion with panellists Tomas Salmonson (Consilium) and Sjaak Bot (Janssen Biologics).

Bert Leufkens started off by explaining that while regulatory science should enhance balanced decision making, it is critical to maintain a balance between ensuring patient safety and at the same time stimulating innovation. Bert used the case of advanced therapy medicinal products (ATMPs), in particular the gene- and cell-based therapies, as an example of how the science needs to be incorporated in regulatory decision-making to enable these complex therapies reaching patients in clinical practice. Other examples that were highlighted were the importance of post-approval learning, in particular how regulators can better respond to new emerging safety issues, and the gap that exists between regulators and HTA/payers.

Larry Liberti continued the session by presenting three other case study examples about the role of regulatory science. In the first case study, he explained how regulatory science is essential in providing the science-based tools to structure and document the decision-making process in order to facilitate high-quality regulatory decisions. The other cases presented by Larry contained examples about regulatory transparency, in which regulatory science can have an important role in standardising and improving regulatory communications, and the role of regulatory science in facilitated regulatory pathways (FRPs) and how can regulatory science can assist in applying the learnings from healthcare crisis such as COVID-19 and Ebola to the development of new regulatory paradigms.

The session continued with reflections from the panellists followed by an interactive discussion, during which the audience asked questions to the speakers and panellists. Sjaak Bot, started with a reflection on the presentations, pointing out the importance of regulatory collaborations, but also the recent concerns have been raised about ‘too-close’ collaborations between public and private stakeholders in the regulatory domain, which is claimed to potentially bias independent decision-making, underlining the importance to find and maintain a good balance in regulatory collaborations.

Tomas Salmonson continued with his reflection, in which he highlighted that novel therapies, with disease-modifying properties and targeting increasingly smaller patient populations, create new challenges that need to be addressed by multi-stakeholder collaborations in regulatory science to fully understand the potential of these products and how they can be applied in clinical practice. The panellists concluded by agreeing that regulatory science will increasingly become a multi-stakeholder effort and that this is critical to appropriately address new challenges posed by future medicines and maintain a consistent and transparent regulatory system that delivers for patients and public health.