Registration for a live webinar on 'Rare disease clinical trials: challenges and best practices' is now open.
See webinar detailsUpcoming Live Webinar
Rare disease clinical trials: challenges and best practices
Tuesday November 19, 2024
9:00 AM PST / 12:00 PM EST / 5:00 PM GMT / 6:00 PM CET
9:00 AM PST / 12:00 PM EST / 5:00 PM GMT / 6:00 PM CET
Rare diseases constitute a heterogeneous group of over 6,000 disorders. In the United States, a rare disease is defined by a prevalence of less than 200,000 cases. Although 6–8% of the world population has a rare disease, interventional research in these diseases is limited and sometimes of poor quality. Recruiting and retaining participants, particularly when diseases are progressive, degenerative, or life threatening and making robust inferences from studies when recruitment is constrained can be challenging.
In this webinar, Professor Remuzzi will discuss limitations, validity, and applicability of innovative designs that must be taken into consideration and may enable researchers to address questions that are unlikely to be answered with conventional designs. When research participants are limited, researchers should consider using: 1) a crossover design; 2) n-of-1 trials; or 3) one of the following adaptive designs: a) a response-adaptive randomization design, b) a ranking and selection design, c) an internal pilot design, or d) a sequential design. Bayesian analysis may be applied to conventional designs, or to any of these uncommon designs. Several of these approaches may also be used in combination. The choice between methods should be guided by factors related to the intervention, disease, anticipated recruitment duration and success, and current state of knowledge about the treatment.
Speaker
Prof. Giuseppe Remuzzi
Mario Negri Institute for Pharmacological Research IRCCS, Italy
Dr. Giuseppe Remuzzi is the Director of Mario Negri Institute for Pharmacological Research IRCCS and Chiara Fama Professor of Nephrology, University of Milan.
From 1996 until 2013, he was Director of the public-private Department of Immunology and Transplant Medicine (a collaboration between the Ospedali Riuniti of Bergamo and the Mario Negri Institute) and, from 1999 until 2018, he has also been Head of the Division of Nephrology and Dialysis. From 2011 until 2015, he was Director of the Department of Medicine of the Azienda Ospedaliera Papa Giovanni XXIII (formerly the Ospedali Riuniti) of Bergamo. In June 2015, he was nominated Chiara Fama Professor of Nephrology at the University of Milan. He was President of the International Society of Nephrology (ISN) for the biennium 2013-2015. From 2019, he is Vice President of Italian Institute for Planetary Health (IIPH). He has been a member of the Consiglio Superiore di Sanità since February 2019.
His main research interests include the causes of glomerulonephritis and the mechanisms of progression of kidney diseases. He has also conducted many studies in the field of transplant rejection. In recognition of his achievements, he received many national and international awards, among them the ISN Jean Hamburger Award in 2005 during the World Congress of Nephrology in Singapore, the John P. Peters Award (American Society of Nephrology 2007, San Francisco), the ISN AMGEN Award (World Congress of Nephrology: WCN 2011, Vancouver), the International Award "Luis Hernando" (Iñigo Alvarez de Toledo Renal Foundation FRIAT: Madrid, Spain) and the “Lennox K. Black International Prize for Excellence in Medicine” (Thomas Jefferson University, Philadelphia). Prof. Remuzzi was honoured by the Italian President of the Republic with the title of Cavaliere di Gran Croce in 2022. Prof. Remuzzi is the author of hundreds of publications in international medical journals and has written several books.