The drug development process is a long and arduous one, especially for rare diseases. Patient and patient representatives can and should be involved in this process from an early stage, since they... Show moreThe drug development process is a long and arduous one, especially for rare diseases. Patient and patient representatives can and should be involved in this process from an early stage, since they have the perspective of living with a disease on a daily basis and can best identify which symptoms are the largest burden and which benefits would be more important to them. In this perspective, we outline how patients can be involved optimally in drug development. We outline success factors such as finding the right partners, bilateral education, having realistic expectations, and an open and honest dialog with all stakeholders. Show less
Lin, N. van; Paliouras, G.; Vroom, E.; Hoen, P.A.C. 't; Roos, M. 2021
Background: For patients with rare diseases such as Duchenne and Becker muscular dystrophy (DMD/BMD), access to their health data is key to being able to advocate for themselves and be in control... Show moreBackground: For patients with rare diseases such as Duchenne and Becker muscular dystrophy (DMD/BMD), access to their health data is key to being able to advocate for themselves and be in control of their care. Since 2018, the DMD/BMD patient community has been committed to making DMD/BMD-related data FAIR, i.e., Findable, Accessible, Interoperable, and Reusable. On March 3, 2021, the second international meeting on FAIR data sharing for DMD/BMD was held virtually.Objective: The aim of this meeting report is to summarize the presentations and discussions of the meeting.Methods: During this meeting, the progress of FAIRification efforts since the first international meeting in 2019, new developments, stakeholder perspectives, and experiences from implementing FAIR data principles in practice were presented and discussed.Results: Over 120 attendees representing various stakeholder groups (ie, patient organizations, clinicians, clinical and academic researchers, pharmaceutical companies, regulators, and EU organizations) from 22 countries participated in the meeting. This meeting report summarizes the presentations and discussions from the meeting, provides an overview of the key lessons learned since the first meeting, and outlines the next steps.Conclusions: Patient organizations are key drivers of the FAIRification process in practice and dialogue with stakeholders is critical to success. Show less
