Improving patient access to valuable new pharmaceuticals: Working towards and evidence ecosystem in healthcare

Abstract

The focus of this thesis was the use of patient-relevant outcome measures in health technology assessment (HTA) reimbursement recommendations and the potential to improve alignment with other stakeholders in the healthcare field. Part A of this thesis showed some overlap between health outcomes used for market access, reimbursement recommendations and in healthcare quality improvement already exists. In addition these outcomes seem to be patient relevant since these are also included in International Consortium for Health Outcomes Measurement (ICHOM) standard sets. However, a greater level of alignment seems attainable, for example by using the same definitions for similar health outcomes or using the same health related quality of life (HRQoL) questionnaires. Residual discrepancies may however remain due to the different remits of each of the actors. Although information on HRQoL has become increasingly important for market authorization and reimbursement recommendations this information is often not systematically collected. Therefore part B of this thesis explored how information derived from social media could provide additional input for HTA reports that support reimbursement recommendations. Social media was shown to be a readily available source of information that can provide fast input on HRQoL from patients with both rare and common diseases. It can be used to listen to what patients discuss on the web and to distribute questionnaires. However, health data from social media is prone to selection and information bias, and validating authenticity can be difficult. Therefore, this type of data should be used complementary to traditional forms of research. Even when there is agreement in health outcomes between the different actors in the healthcare field there may still be some differences to allow each of those actors to pursue their purpose. One specific difference is the use of different comparators, for example a placebo or a treatment used in clinical practice. Part C investigated the potential of using indirect treatment comparisons for reimbursement recommendations. Indirect treatment comparisons were shown be evaluated regularly for reimbursement recommendations by the Dutch and English HTA agencies. Indirect evidence may have a substantial impact on the conclusion of the relative effectiveness assessment, however the effect on the cost effectiveness assessment and final recommendation is difficult to extract since multiple factors play a role.