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Drugs for rare diseases
  1. W. H. Lyle

    Abstract

    Pharmaceutical houses alone are equipped to market new medicines. The cost is very high, much of this arising from the preliminary toxicological and pharmacological work which is required in order to satisfy licensing authorities.

    The introduction of a drug, even for a rare and lethal disease without such data, even if permitted by the authorities, would leave its manufacturer exposed to adverse publicity and litigation in the event of a mishap.

    These considerations have had the effect of stifling original work in the drug treatment of rare diseases, the position having worsened in recent years.

    This trend is illustrated by the history of the introductions of penicillamine and triethylene tetramine for Wilson's disease.

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