Patient Preferences – the science of tomorrow
At DIA Europe 2019, Conny Berlin, Rocco Falchetto, Ardine de Wit, and Christine Radawski presented information on the importance of patient involvement, and the rapid evolvement of the science of patient preferences. The close interactions between patients, researchers and decision makers show how it is possible to establish a new approach to support medicinal development. The presentations focused on the work of the IMI PREFER project and highlighted an example where patients views could be extremely useful in regulatory decision making.
Conny Berlin’s presentation described the 2016-2021 goal of PREFER to develop expert and evidence based recommendations to inform medical product decision making. She set the stage to why the work was essential, as the current regulatory environment has opened the door on industry’s use of patient preference data in decision-making. The FDA is authoring guidance relating to patient-focused drug development and the CHMP exploring “the feasibility of using a more explicit approach in describing value-judgments.”
Rocco Falchetto, along with Jasmin Barman-Aksozen, as presidents of the International Porphyria Patient Network presented a powerful presentation on how the decision making of medicines are influencing patients who live with porphyria. Rocco was passionate about the inclusion of patient insights and patient preferences in the medicines lifecycle gives patients a stronger voice at the decision makers' table.
Ardine de Wit, gave a succinct overview on how PREFER will achieve their goals and what it hopes to learn from the case studies. PREFER’s work began with assessing the stakeholders needs and then appraising preference methods. PREFER has compiled a summary of what questions could be answered and the decision points when patient information might be beneficial in the life cycle of a product. Then the most promising exploration and elicitation methods were summarized to be useful in gathering patient preference information. With goals of PREFER and the list of methods, the strategy for the case studies was developed. The PREFER case studies should answer methodological and operational questions, demonstrate comparative value in different methods, and guide stakeholders on when and how to elicit patient preferences.
For the last presentation for the session, Christine Radawski gave an overview of the rheumatoid arthritis (RA) case study. RA is a chronic disease with the need for lifelong treatments and because damage cannot be reversed, the need for earlier treatment is being considered. The purpose of this case study is to understand if people were at an increased risk for RA, would is their preferences in taking a medication that would delay the disease but may have particular side effects.The study is using 2 stated preference methods to gather data on patients in 2 different countries. To inform each of the various stakeholder, this case study will answer 5 out of 8 methodological research questions in PREFER.
By Christine Radawski