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Quantifying emerging drugs for very rare conditions

By K. A. Miles, C. Packer and A. Stevens


Background: EU legislation is encouraging pharma-ceutical companies to develop drugs for rare con-ditions, but their often high cost, and potential for long-term administration has led to debate about their affordability and cost-effectiveness. Aim: To investigate how many drugs are in develop-ment for very rare conditions. Methods:We defined very rare conditions as having a prevalence of <1:50 000, and identified pharma-ceuticals in phase II, phase III trials or pre-registration for these conditions using commercial databases. Results: We identified 42 very rare conditions with at least one drug in late-stage clinical development, with a total of 113 drugs in development (17 for at least two indications). Sixteen drugs were pre-registration, 29 were in phase III development, 65 were in phase II development, one drug was both pre-registration and phase II for different indications and two drugs were in both phase II and phase III trials for different indications. Discussion: Not all the drugs in development will reach the market, but it is likely that a signif-icant number will do so. Affordability and methods to assess cost-effectiveness will need debate and clear national policy for decision-makers to follow

Year: 2007
DOI identifier: 10.1093/qjmed/hcm021
OAI identifier: oai:CiteSeerX.psu:
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