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With all the discussion in Europe of how to move towards personalized medicine and more flexible authorization procedures, it is the rare disease community that is, in many ways, offering guidance on the route ahead.
A new project to explore methods for design and analysis of clinical studies in rare diseases is attempting to develop statistical approaches for efficient evaluation. Named IDEAL (Integrated Design and Analysis of small population group trials) and funded by the European Union to the tune of nearly $4 million, it is collating all available information that might help to optimize the search for an appropriate statistical method.
There are numerous areas of focus. They include the assessment of randomization, the extrapolation of dose-response information, the study of adaptive trial designs, and the development of optimal experimental designs in mixed models. They also include pharmacokinetic and individualized designs, simulation of clinical studies, genetic factors, decision-theoretic considerations, and the evaluation of biomarkers.
The EU rationale for backing this type of project is evident. Even though some of these conditions occur in only one or two patients, when the figures are added up across the 28 countries that belong to the EU, as many as 36 million people suffer from at least one of the 5-8,000 recognised rare diseases. And most of these diseases affect children, of whom nearly a third die before reaching their fifth birthday.
An international research team draws on academics and clinicians from Germany, Austria, France, Luxembourg, Sweden, Poland and the Netherlands, and an advisory board involves patient interests, pharmaceutical industry and delegates from regulatory authorities, who will collaborate in developing new designs and sophisticated methods of analysis.
As the EU says (or is likely to, if the strategy paper on personalized medicine that it is still working on after five years resembles the draft that has come the way of this columnist!), recent EU funding has had "a specific focus on rare disease research as a model to study personalized medicine approaches. The particularities of this area, such as small patient populations and predominantly genetic causes, make it an interesting case for investigating personalized medicine approaches." Clinical trial methodologies for small patient populations, in particular for rare diseases or personalized medicine, has also been a topic for funding in recent EU programs.
Meanwhile, the European Commission is inviting applications for appointment to the expert group on rare diseases that it is setting up (and doing so with all the subtlety of an army recruiting officer: "Are you an expert in the field of rare diseases or do you represent an organization involved in rare diseases related activities? Are you interested in making a difference in Europe? Apply now at: http://ec.europa.eu/health/rare_diseases/expert_group/call_en.htm")
So if you want to make a difference, get in touch. It's a rare opportunity.