IDeAl researchers disseminate latest findings at ISCB 2015

Several researchers of the IDeAl project are meeting up at ISCB 2015 in Utrecht in order to disseminate their latest findings on methodolgy for small population group trials. Ralf-Dieter Hilgers together with Armin Koch from the Asterix project gave a pre-conference workshop to their fellow researchers introducing them to Stratification and Randomisation in clinical trials. The topics for the contributed talks of the IDeAl researchers range from “Strategies for dealing with missing data in randomization tests” (N. Heussen),  over “Understanding variation in n-of-1 trials” (A. Araujo),  “Baselines as possible controls in trials in small populations” (S. Senn), “The effect of selection bias on the test decision of the randomization test in small population group trials” (D. Uschner), “Desirability of restricted randomization procedures in small population group trials in case of selection and chronological bias” (D. Schindler), “Efficient tests for the similarity of dose response curves using a bootstrap approach in regard to small sample sizes” (K. Möllenhoff) and “Evaluation of the fisher information matrix without linearization in nonlinear mixed effect models for discrete and continuous outcomes” (M.K. Riviere). In addition, Ralf-Dieter Hilgers has been invited to talk about “New developments in integrated design and analysis of small population group trials”. Last but not least, ISCB 2015 is a great opportunity for IDeAl to stay in touch with stakeholders of the rare disease community. Even more, one meeting is dedicated to the joint work with the Asterix and Inspire projects.

Gerald Hlavin receives Arthur Linder Award

The International Biometrical Association honours Gerald Hlavin for his work “Evidence, Eminence and Extrapolation” with the renowned Arthur Linder Award. The research addresses the crucial question under which circumstances evidence from one subgroup can be transferred to another subgroup. This question arises especially in the context of clinical trials for children. The work is embedded in the research of Adaptive Designs and marks a cornerstone in the IDeAl project. The press article is available on the website of MUW [in German].

INI meeting in Cambridge was a big success

Several members of the IDeAl consortium got together at Isaac Newton Institute in Cambridge for one week (July 6th – 10th, 2015) to discuss mathematical implications for the design and analysis of experiments in healthcare. The meeting comprised two main parts: The first part was devoted to the fruitful exchange of the IDeAl consortium and EAB members, and the scientific community. F. Mentré, M.K. Riviere, S. Ueckert (INSERM), R.-D. Hilgers (UKA), and H. Dette (RUB) stimulated the discussions with talks from their cutting-edge research, and the EAB members C. Jennison, R. A. Bailey, S. Julious, and W. F. Rosenberger added presentations replete with experience form their areas of expertise. The second part, inofficially baptised “industry day”, was dedicated to the dialogue with stakeholders from industry and regulators from EMA. The second part of the meeting highlighted state-of-the-art design of experiments methodologies and how these can impact the wider societal health objectives. The broad audience was captivated by R.-D. Hilgers talk on the recent advances of the IDeAl project. F. König added an in-depth presentation about the newly developed approach for tests in dose-response studies. The meeting is expected to boost the advances in the field of Design and Analysis of Experiments in Healthcare, as well as the cooperation with regulatory sector. As a special feature, all talks given throughout the workshop can be downloaded from the INI website, spreading the range of influence even further.
INI-Meeting-2015

Austrian magazine “Profil” issues extensive article on Pediatrics

The Austrian magazine “Profil” released an extensive article on Paediatrics. Quoting Christoph Male (MUW), the article calls attention to the lack of drugs licensed for the tharapy of diseases in children. The smaller the child, the bigger are the differences of the effectiveness and safety of the drug. One reason for the lack of therapies for children is the low number of affected people, making it both unprofitable and difficult for the drug companies to investigate therapies for children. On the other hand, the fundamental agreement of society to leave children out of clinical research needs to be overcome. Both issues are being tackled by new regulations of the European Medical Agency and its Paediatric Committee. The drug companies are now required to consult the Paediatric Committee in the early stage of the development of a new drug for the therapy of any disease that is prevalent in children. Christoph Male is the Austrian representative in the Paediatric Committee. The new regulations also benefit the drug companies by longer exclusive licenses and easier licensing processes, and are expected to have a positive effect on the number of available therapies already in a few years.

IDeAl mentioned in April newsletter of Japanese agency PMDA

The Japanese Pharmaceutical and Medical Devices Agency (PMDA) mentiones IDeAl explicitly in their latest newsletter. Peter Bauer, Martin Posch and Franz König explained the IDeAL project for PDMDA staff members and gave a special training on adaptive designs with the title “Trends in adaptive design and statistical approaches to small population group trials in EU”, including an outline of adaptive designs as well as regulatory issues.