Meeting Highlight - RSNN workshop 2022

The chair of the workshop was Peter Mol and various stakeholders shared their different perspectives, experiences, challenges, and opportunities in the elicitation and inclusion of PP across the different phases of drug development, with a specific emphasis on regulatory decision-making. 
 

The workshop started with the perspective of a patients’ association (Cristina Guerrero Paez), followed by the input of a regulator (Francesco Pignatti), a basic scientist (Stan van de Graaf), and industry (Cathelijne de Gram). An overview of the largest initiative on PP, the IMI PREFER project, was presented  by Isabelle Huys, Rosanne Janssens, and Liese Barbier, who showed the role and impact on the field of PP of obtaining the European Medicines Agency (EMA) qualification of IMI PREFER, a framework elaborating on why, when, and how to use and assess PP studies in dug development. The meeting continued with a panel discussion with input from the audience, which responded to several questions about patient centricity, heterogeneity in shared-decision making, and the inclusion of minorities in PP studies, among others. Finally, the audience was challenged with an interactive PP exercise conducted by Douwe Postmus, Hans Hillege, and Sonia Roldan. During this exercise participants imagined to be living with early Alzheimer’s disease, and were asked to set their maximum acceptable risk in exchange to delaying their disease progression. The exercise gave concrete insights about the design, methodology and applicability of these studies.

PP studies for more transparency and robustness

All presenters agreed on the relevance and added value of PP studies, and the willingness of patients to collaborate in the studies in different manners was highlighted. PP studies were recognised to increase the transparency and robustness of regulatory decisions, since they can elicit, qualitatively or quantitatively, the importance of various drug effects, the acceptable risks, and the heterogeneity in preferences across different subgroups of the population, contributing to a more objective benefit-risk evaluation.

Challenges

The different stakeholders also discussed some of the challenges to overcome. For example, the importance of using patient-friendly language and patient-relevant outcomes, which may not always be in line with the outcomes of clinical studies. Bridging this information in the most accurate manner is, however, essential to incorporate PP studies in regulatory decisions. Furthermore, industry and regulators highlighted the difficulties to include PP studies in the drug development, since the lack of guidelines about their role in the decisions and the limited information for their execution in a regulatory acceptable manner makes it unstructured and challenging.

The workshop conveyed the importance of the different stakeholders cooperating together from the earliest phase, basic science research. This will facilitate the setting of initiatives, the design and conduct of adequate studies, and the development of appropriate guidelines. All stakeholders agreed on the  growing importance of PP studies in drug development and assessment, and aimed for a better implementation of these studies in the near future through further collaboration.