Drug discovery today
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Drug discovery today · Jan 2011
ReviewCharacteristics of orphan drug applications that fail to achieve marketing approval in the USA.
The US Orphan Drug Act has fostered the development of drugs for patients with rare diseases by granting 'orphan designations', although several orphan drugs for which a marketing application has been submitted to the FDA have failed to obtain approval. This study identified the clinical trial design, the level of experience of the sponsor and the level of interaction with the FDA to be associated with non-approval. Sponsors, therefore, should engage in dialogue with the FDA and thoughtfully design pivotal clinical trials in accordance with FDA guidance documents.
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Drug discovery today · Jan 2011
ReviewCreativity, innovation and lean sigma: a controversial combination?
The application of lean sigma is gaining momentum in drug discovery and development but it remains controversial because of perceptions that process improvement will suppress much-needed creativity and innovation. We review the conditions required to support creativity and innovation and the principles and benefits of lean sigma in a drug discovery environment. We conclude that it is desirable to create a unified climate that encourages and enables both innovation and continuous improvement and that this is possible if three key tensions are handled carefully and with due respect to the needs of research. These three potential traps occur in the interpretation of standardization, the role of variation and the choice of how to use liberated capacity.