Aligning clinical endpoints with patient relevant endpoints

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.