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dc.rights.licenseCC-BY-NC-ND
dc.contributor.advisorBoer, A. de
dc.contributor.authorAl-Mugoter, Nora
dc.date.accessioned2022-02-11T00:01:00Z
dc.date.available2022-02-11T00:01:00Z
dc.date.issued2022
dc.identifier.urihttps://studenttheses.uu.nl/handle/20.500.12932/490
dc.description.abstractBACKGROUND Little is known about the role Patient-Reported Outcomes (PROs) play in, the main clinical studies, the benefit-risk assessment, product labelling, and which determinants play a role herein. METHOD We evaluated the European Public Assessment Reports (EPARs) of new medicinal agents with a regulatory decision (approved or refused) by the EMA between January 1, 2018 and December 31, 2020. The primary and secondary endpoints of the main studies, benefit-risk balance, effect tables (results that have a strong influence on the assessment) and SmPC (for patient care decision-making) were evaluated for any mention of PROs. RESULTS A total of 149 products met our criteria. Of these, 84 (56.40%) mentioned PROs. PROs were used significantly more as a secondary endpoint and contributed significantly more to patient care decision-making by chronic treatment compared to acute and preventive treatment. Furthermore, the disease category played a significant role in PRO use. For example, by respiratory conditions, PROs weighed in on 100% of the effect tables and SmPC. In contrast to infectious disease, where PROs weighed in on 5%, and 0% of their effect tables and SmPC, respectively. Moreover, PROs from blinded studies contributed significantly more to patient care decision-making compared to open-label studies 3.836 (95% Cl 1.802-8.167, p < 0.01). CONCLUSION In conclusion, different product determinants play a role in the utilization and contribution of PROs to the assessment and patient care decision-making. Overall, more PRO claims are being approved by the EMA and a gradual increase are seen in their contribution to support drug licensing. They are weighing in more and more on the benefit-risk balance assessment.
dc.description.sponsorshipUtrecht University
dc.language.isoEN
dc.subjectMedicinal products submitted to and assessed by the European Commission (EMA) between January 1, 2018 and December 31, 2020 were evaluated for PRO utilization as a primary and/or secondary endpoint in their main studies and if PROs were mentioned in the benefit-risk balance, the effect table and SmPC of the EPARs.
dc.titleThe Role of Patient-Reported Outcomes in Marketing Authorization Applications and Product Labelling (2018-2020)
dc.type.contentMaster Thesis
dc.rights.accessrightsOpen Access
dc.subject.courseuuFarmacie
dc.thesis.id2234


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