On today’s Rare Disease Day 2016, awareness is raised for rare diseases and the impact they have on patients lives. Several events take place around the globe, all coordinated by EURORDIS. The Rare Disease Day is commemorated each year on the last day of february. For further information, please visit http://www.rarediseaseday.org.
The 11th Workshop on Population Optimum Design of Experiments, PODE 2016, will take place on Monday, 20 June 2016, in Uppsala, Sweden. As it has always been at PODE, there is no registration fee. Abstract submission is open until the end of February 2016.
Please see http://www.maths.qmul.ac.uk/~bb/PODE/PODE.html for more information about the PODE meeting.
Prof Alan Agresti visited the IDEAL group for one week in February 2016 funded by an ERS grant of RWTH-Aachen University and became Theodore von Kármán Fellow. Professor Agresti is distinguished Professor Emeritus of Statistics at the University of Florida (1972-2010). He has published more than 100 articles and six books including the book “Categorial Data Analysis” from which we all learned about categorical data analysis. He is fellow at the American Statistical Association since 1990 and the Institute of Mathematical Statistics (2008) and was visiting professor at the statistics department of Harvard University from 2008 – 2014. We enjoyed the talks he give during his stay and discussed with him the evaluation of categorical data after covariate adaptive randomization for small samples.
The IDeAl Annual Meeting 2015 in Leuven, Belgium, was a great success. The senior researchers and PhD students of the IDeAl project coordinated by Ralf-Dieter Hilgers met with the EAB members for a two-day meeting in order to discuss their recent advances. In their talks, the EAB members Segolène Aymé and Ralf Herold addressed the projects impact on the rare disease community, particularly its influence on the regulators perspective. The subsequent discussion stimulated further close cooperation of IDeAl with EMA and IRDiRC.
The Pre-Meeting workshop was packed with two half-day courses from very distinguished researchers: Professor Chris Jennison of University of Bath (EAB) offered valuable insight on group sequential trials, followed by Professor Molenberghs of Universiteit Hasselt (WP 10) who shared his expertise on Mixed Models and Missing Data.
During the Young Scientist Meeting, the PhD students had the opportunity to learn from the EAB members about a future career in research.
Even the coffee breaks were used by the researchers to have fruitful discussions about their recent work, resulting in numerous proposition to further intensifying their collaboration.
The IDeAl project is strongly represented to discuss multiplicity issues related to rare diseases at the 9th International Conference on Multiple Comparison Procedures MCP2015, September 2nd – 5th, in Hyderabad, India (http://www.mcp-conference.org/). This conference promotes research and applications of Multiple Comparison Procedures and provides a forum for interactions among industry practitioners, regulators, research scientists from subject matter areas and statisticians.
The IDeAl project co-organized an invited panel discussion on “Frontiers of confirmatory inference in small populations” chaired by F. König and M. Posch.
Generating clinical evidence for small populations is extremely challenging, e.g., in the development of orphan drugs, personalized medicines or drug development for children. The ability of conventional statistical methods to evaluate new therapeutic approaches for any given rare diseases is limited due to the small number of patients concerned. This means that established statistical approaches to demonstrate the efficacy and safety of therapies may fail in this situation.
The number of rare diseases is also increasing simply due to the fact that what was considered as a single indication in the past is nowadays understood as multiple different diseases that may require different treatments. Furthermore, when conducting clinical trials in smaller and smaller study populations, we land in multiplicity territory in ways that traditional development programs and trials in “common” diseases do not. For example some diseases may have numerous symptoms. A treatment that benefited any of the symptoms might be considered as useful. Which level of evidence is needed? Should a drug be disregarded because the primary endpoint chosen turned out not to be the one the drug impacted on? What if the treatment effect was diluted due to a too broad study population? Single arm versus parallel group designs?
In addition, IDeAl presentations by RD Hilgers, N. Heusen, CF Burman and F König are included in the regular program with several presentations.
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