Making decisions with precision
Making decisions with precision
In the PhD thesis MAKING DECISIONS WITH PRECISION: Stimulating Alignment of Precision Medicines’ Development Trajectories with Regulatory Decision-Making Needs, Lysbeth Bakker investigates how the development of precision medicines (treatments tailored to specific patient groups) can be better aligned with the regulatory approval of medicines in Europe. The ultimate goal is to make more suitable medicines available to patients. The research focuses on the role of biomarkers (measurable characteristics that provide insights about the disease or possible treatment response), real world data, and patient reported outcomes in regulatory decision making, using case studies from, among others, kidney diseases and neurodegenerative disorders.
This topic is highly relevant because many medicines do not work equally well for all patients, and chronic diseases often show large differences in treatment response between individuals. Precision medicine promises more effective and safer treatments by matching the right patient with the right therapy.
The main findings show that there is broad support for precision medicine among patients, healthcare professionals, industry, and regulators, but that its practical implementation lags behind. Only a limited number of biomarkers have been formally approved for regulatory use. Although biomarkers are frequently used in clinical studies, they are still relatively rarely explicitly included in official medicine indications. In addition, real world data and patient reported quality of life measures appear to provide valuable complementary information, but so far contribute only to a limited extent to formal regulatory decisions on medicine approval.
The thesis concludes that closer collaboration between all stakeholders, improved validation of biomarkers, clearer documentation of assessors’ reasoning, and greater attention to patients’ experiences are needed to gain better insight into the evidence requirements for precision medicines. These steps can help ensure that precision medicine is applied more effectively and that innovative, patient centred treatments become available more quickly and carefully for those who benefit most.