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    Integration of Patient Reported Outcomes in Pharmaceutical Drug Development For Prostate Cancer – Focus on the Patient

    Holstrum, Carl (2019) Integration of Patient Reported Outcomes in Pharmaceutical Drug Development For Prostate Cancer – Focus on the Patient. Doctoral thesis (PhD), Manchester Metropolitan University.


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    Understanding, and adequately articulating how and what the patient feels and experiences during the progression of a disease and during drug treatment is an important aspect in a patient’s life. Yet this articulation does not always get appropriate attention and thus the patient experience may not be fully understood by others. Nor is it fully explored in drug development programs due to the lack of focus on patient outcomes. Drug development has in the past primarily focused on meeting the regulatory approval of the new drug. To gain regulatory approval a company has to provide evidence of the drug’s safety, and efficacy, combined with a favourable benefit-risk ratio. The patient benefits or patient concerns, especially in the case of cancer, is thus often masked within the clinical endpoints, such as overall survival or progression free survival, or if patient outcomes are measured, they may not be appropriately articulated in dialog between stakeholders, or in publications. Recent years have seen an upswing in pharmaceutical companies introducing a so-called patient-centric research and focus is shifting towards patient relevant endpoints. However, pharmaceutical companies do not always fully understand how such patient-centric research is to be conducted, and if data is collected, how to interpret the data and how to present the data to stakeholders. The two most important pharmaceutical drug regulators and approval bodies, at least in terms of millions of lives they potentially affect, the European Medicines Agency (EMA) and the Food and Drug Administration (FDA) have both in the recent years embraced the concept of patient-centricity, and patient experience in their guidance to the industry. There is an increased acceptance of this type of evidence in regulatory submissions, thus, opening opportunities for pharma companies to promote research towards patient relevant endpoints and evidence. It is with this background that this thesis is written and compiled, with the aim of discussing how Patient Reported Outcomes (PRO) research can, and should be integrated into pharmaceutical drug development, and how such results can be presented, while keeping in mind the many different stakeholders that need this information, ranging from regulatory agencies, to payers, physicians and patients. The thesis makes use of eight recent patientcentred papers published in the field of prostate cancer. The papers and the research make a number of important contributions by providing examples of different approaches on how the PRO analyses are conducted, exploring different way of reporting results and by linking the results of PRO evidence to clinical outcomes. Through the iterative learning process, which came as a result of the research I conducted over time and by exploring different analyses methods, ways of presenting results and presenting results to different journals, I learnt how to conduct this type of research, all the way from conceptualization, through data collection, analyses and reporting of results. Thus by combining this learning, which was gained from this research, with the papers selected for this thesis, this has provided me with the structure and learning and legacy that I can bring forward from this research. It provides the basis for the construct of the framework presented in this thesis for how such Outcomes Research can be implemented in drug development. This blueprint and framework can be adapted to any disease area and can enhance the impact of the research, enhance new drug treatments, and help patient’s get the best and most suited treatment options. As an overall mantra of clinical drug development, we must embrace that the ultimate raison d’etre of any medicine or intervention, must be to the benefit of the patients and to improve their health.

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