Development of a Roadmap and Evaluation of Quality Decision Making Practices During Medicines Development, Regulatory Review and Health Technology Assessment
Bujar, Magdalena Gabriela
The science of decision making has been established, but research is limited regarding the quality and transparency of the decision-making processes through which medicines become available. Indeed, it is not always clear what explicit processes pharmaceutical companies, regulatory authorities and health technology assessment (HTA) agencies are using to arrive at their decisions. Previous research resulted in the development of the ten Quality Decision-Making Practices (QDMPs), which are considered as the best practices in decision making in the lifecycle of medicines, as well the Quality of Decision-Making Orientation Scheme (QoDoS), which is a technique that can be used to measure the incorporation of these QDMPs in companies and agencies. The aim of this research, therefore, was to develop a roadmap for improving the quality of decision-making processes for key strategic decisions made by pharmaceutical companies, regulatory authorities and HTA agencies, by firstly evaluating current processes in place; secondly by assessing the ten QDMPs and thirdly by developing approaches for incorporating the ten QDMPs into organisational processes. In formalising the study design, various methodologies were appraised and it was rationalised that in order to fulfil the aim of the study, it would be appropriate to adopt a mixed method approach i.e. combination of self-administered questionnaires, focus groups as well as case studies. In addition, a systematic review of literature was conducted to identify the most robust and psychometrically sound technique for assessing the QDMPs. Results from four questionnaires developed for the purpose of this research demonstrated that companies and agencies do not always utilise frameworks for key decision-making processes, namely regulatory submission processes, regulatory approvals, HTA submissions and HTA appraisals. Moreover, the majority of organisations with established frameworks did not incorporate all ten QDMPs into their processes, although all these practices were considered relevant. The study also identified challenges in decision making as well as biases, where all the participants believed there was room for improvement and suggested a number of solutions, which included training and the use of structured processes. The results also uncovered that organisations do not generally have formal assessments in place to measure the quality of decision making. However, a systematic literature review of techniques for evaluating the QDMPs during the lifecycle of medicines revealed a paucity of available techniques, which could explain why such assessments are not taking place. The QoDoS was considered as the most promising technique out of the 13 identified in the review. A subsequent case study with QoDoS demonstrated its reliability and relevance in target audiences. Three additional QoDoS case studies, with a company, a regulatory authority and an HTA agency, demonstrated its practicality in assessing the consistency of favourable and unfavourable practices in organisations. Finally, three focus groups with individuals from companies, agencies and academia resulted in the development of practical approaches for embedding quality into strategic decision-making processes. These recommendations, as well as the outcomes from the other studies, were used to develop a checklist for incorporating the ten QDMPs into a project matrix structure including the steps and variables to be documented at the time of decision making. The methods and approaches developed and validated during this programme of research, namely the questionnaires, the QoDoS and the checklist, led to the development of a roadmap for improving the quality of decision-making processes within companies and agencies for key strategic decisions. The goals are to raise the awareness of the importance of quality decision making during the development, regulatory review and HTA of medicines, secondly to improve the probability of good outcomes and finally to increase public trust as a result of more transparent and consistent decisions.