RSNN workshop 2017 ‘Beyond the current clinical endpoints’

06 July 2017
Utrecht, The Netherlands
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While the majority of current clinical endpoints used in clinical trials can quantitatively inform us about the efficacy and clinical relevance of a new drug compared to a placebo or comparator in a randomized clinical trial, there are also endpoints that are difficult to translate into a clinically meaningful benefit. For example, the six minute walk test (6MWT) is widely used as an endpoint to estimate effectiveness of interventions in numerous diseases such as osteoarthritis, and fibromyalgia, heart failure, chronic obstructive pulmonary disease (COPD), pulmonary fibrosis, pulmonary arterial hypertension (PAH), Duchenne Muscular Dystrophy, Fabry’s Disease, etc., and reasons for use vary across diseases: as surrogate for the clinical outcome, as accepted standard or “the best we have”. 

Also for other diseases, such as Amyotrophic Lateral Sclerosis, endpoints are used that can be challenging to provide insight in clinically relevant outcomes. In the end, the choice of the endpoint in the most relevant patient population is paramount in the benefit/risk discussion in the context of licensing and reimbursement of drugs.

This workshop aims to gather experts from patient communities, academia, industry, health technology authorities and regulatory authorities to discuss how we can improve the selection and use of such challenging endpoints or how we can move past them by exploring new concepts such. These novel ways of thinking have the aim to reinvigorate the ‘old’ quantitative endpoint in order to capture the qualitative aspects of a quantitative endpoint.

This should allow setting up key criteria for endpoint definition and selection that are also better aligned to the clinical relevance of primary endpoints in RCTs from the perspectives of the needs of all relevant stakeholders in clinical drug research. In this respect, the workshop continues on its topic presented at the FIGON Dutch Medicines Days and the annual RSNN Workshop 2016.

For more information and the program, click here.