Optimizing the conduct and implementation of patient preference research in drug development and evaluation: a focus on oncology and COVID-19

Patient preferences and patients' views are increasingly seen as an important source of information that should be taken into account when making decisions throughout the whole drug life cycle. However, decision-making is now predominantly done by non-patient stakeholders (such as the industry, regulatory agencies and clinicians). Moreover, patients' needs and preferences are not always in line with those of the other stakeholders. As a consequence, decisions may be of reduced quality and medicines with less relevant characteristics for patients may enter the market. Research on patient preferences through patient preferences studies (PPS), is therefore considered by all stakeholders in healthcare (such as pharmaceutical industry, healthcare providers, regulatory agencies and health technology assessment (HTA) bodies) as valuable information in making their decisions. However, currently a validated PPS workflow including a robust standardized methodology for selecting attributes and appropriate methods is lacking and there is insufficient clarity on how to embed the outcomes of PPS in the decision-making of different stakeholders. This doctoral project, will investigate in how to optimize, validate and implement PPS in healthcare decision-making along the drug life cycle. The disease areas of this PhD research are Oncology and COVID-19; disease areas of particular interest given the rapid development of several new treatments with varying benefits, risks and uncertainties, and only limited empirical information on what matters most to patients. This PhD will investigate how 1) the development of PPS attributes and selection of an appropriate method in PPS can be optimized; 2) PPS' outcomes (such as unmet patients’ needs and relevant patient endpoints) can be systematically included to guide decisions by industry, regulators, HTA bodies and payers; 3) develop consensus-based guidance for regulators and HTA bodies on how to include, systematically assess and report patient preference results in their decision-making.