36 new orphan drugs approved by FDA in 2014

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According to the website RDR Rare Disease Report, “a remarkable 36 orphan drugs were approved for new indications by the U.S. Food and Drug Administration (FDA) in 2014.” The indications addressed by the new therapies include (among others) rare cancers as well as gaucher disease and idiopathic pulmonary fibrosis. This development shows that rare diseases are gaining importance, also for the pharmaceutical industry. The need of innovative statistical methods for these trials is thus immanent.

IDeAl explicitly mentioned in recent methodology paper

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The IDEAL project has been especially mentioned in a recent article of Smith et al.  on “Methododolgy of clinical trials for rare diseases” as an example how important the development of novel methodology is to improve the efficiency of rare disease trials.” The paper recognizes that the ongoing research of the IDeAl project is making a significant impact on the development of innovative methods for rare diseases by expanding the collection of available trial designs.

Franz König is invited panelist at FDA & SCT/ QSPI workshop

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Franz König is one of the invited panelists at FDA & SCT/QSPI workshop on innovations in the science and practice of clinical trials in Washington today. The panel discussion is part of the session on Rare Diseases, Small Clinical Trials and Rare events. For further information see the sct website. The program of the workshop is provided here.

R package for dimension reduction of genetic information now available online

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Piotr Sobczyk now released the R package varclust for dimension reduction via variables clustering. varclust is highly relevant for the identification of genetic pathways. In particular, it achieves the aim of finding the the major regulatory process and identifying the genes that are the triggers behind the whole group. The ease of use is ensured by an online interface for the package and a detailled package vignette. Furthermore, varclust is freely available for download from github.

Statistics for Rare Diseases at ISCB 2015

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Ralf-Dieter Hilgers has been invited to present the IDeAl project at ISCB 2015. He is Invited Speaker in the session on Statistics for rare diseases. The coordinators of the InSpiRe and Asterix projects will present their projects in the same session. More information will be available on the ISCB website.