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Mission Critical: How Translation-Focused Disease Foundations May Save Medical Research

Patients waiting expectantly for medical research to produce important new cures are finding bad news almost everywhere they turn.

Pharmaceutical companies are suffering from a much-discussed innovation crisis, as old drugs lose patent protection without new drugs to replace them; meanwhile, the small biotechs that could potentially bail big pharma out struggle to raise capital .

University scientists, for their part, are beset by an unseemly credibility crisis, as the intrinsic fragility of medical research is now vividly apparent from the soaring number of high-profile retractions, and the well-documented difficulty of reproducing many published findings outside the originator’s lab.

At the heart of this crisis is the misalignment of two very different cultures.

Academic scientists tend to focus on publishing papers, and usually assume that the results will eventually be useful. They place a high value on novelty, and relatively less value on whether the data are robust, easily reproducible by others, or truly relevant to human disease. Captivating data from putative laboratory models of disease generate publications, even if the model is not very predictive of human disease – and unfortunately, most models aren’t.

Conversely, big companies traditionally focus on generating efficiencies through scale, and on developing reproducible processes. This works very well for manufacturing, reasonably well for large late-stage clinical trials, and essentially not at all for early-stage (discovery) research.

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