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      Aligning clinical endpoints with patient relevant endpoints

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      conference-abstract
        1 ,
      RExPO24 Conference
      REPO4EU
      RExPO24
      3-5 July 2024
      Patient engagement, patient involvement, patient centricity, regulatory science, real world evidence
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            Abstract

            Generating clinical evidence based on mechanistic disease models, is a corner stone of drug discovery, new drug product development and approval. Such clinical studies are performed in randomized small patient populations focusing on the clinical parameter and the occurrence of harmful adverse reaction. By involving patients, these mistakes could have been avoided in the early development could have been avoided. While the clinical study outcomes suggest patient benefit, often the results in the “real world” patient population cannot be reproduced post approval [1]. One important reason for this is the increasingly active role of patients in therapy decisions and especially implementation of complex treatment schedules, which is reflected in adherence, among other things. Patient centric drug product design and development has emerged as a concept to address the non-adherence and poor acceptability issue [2–4].

            To take this into account, patients are increasingly involved in many different areas at the EMA and influence development and approval procedures [[5]. The potential to bring clinical endpoints into line with patient-relevant endpoints is far greater than previously assumed. In retrospect many new therapies that have been discontinued due to undesirable side effects (Accomplia™, Relenza™, Fanapt™). inappropriate product design (Exubera™, or potentially could have been prevented if patients had been included in the target product profiling and development process. Furthermore, we underestimate the therapeutic burden to which multimorbid patients are exposed. It is often only small details that cause patients to fail because the complexity of the products and therapy ex8ceeds the patients' ability to deal with them [6,7].

            Patients know the opportunities that we as scientists might not see.

            Content

            Author and article information

            Conference
            RExPO24 Conference
            REPO4EU
            9 May 2024
            Affiliations
            [1 ] Leibniz JointLab First in Translation; Leibnitz-Institut für Interaktive Materialien, Aachen, Germany ( https://ror.org/0186h8060)
            Author notes
            Author information
            https://orcid.org/0000-0003-4360-1714
            Article
            10.58647/REXPO.24000060.v1
            6fe9500b-2bc2-44e4-a5e9-bd7a0f09d6de

            This work has been published open access under Creative Commons Attribution License CC BY 4.0 , which permits unrestricted use, distribution, and reproduction in any medium, provided the original work is properly cited. Conditions, terms of use and publishing policy can be found at www.scienceopen.com .

            RExPO24
            3
            Munich, Germany
            3-5 July 2024
            History
            : 9 May 2024
            Product

            REPO4EU

            Categories

            Data sharing not applicable to this article as no datasets were generated or analysed during the current study.
            Medicine
            Patient engagement, patient involvement, patient centricity, regulatory science, real world evidence

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