development of an Orphan Drug policy is part of the 1996 work program

the policy is likely to include:

• designation based on prevalence of disease in the population of less than 0.05% (about 180 000 patients) AND no expectation of profitability AND subject to review and withdrawal if criteria no longer apply

• shared cost program to support research in addition to the BIOMED program already in place

• monitored release program in addition to the current provision which permits marketing authorization for some drugs based on a limited dossier

• development of a telematic network to facilitate clinical trials and research

legislation is already in place providing market exclusivity and provisions for fee exemptions under cost recovery

many member states already have incentives in place for R&D related to orphan diseases

individual member states control access to drugs through their own programs (see France, U.K.)

no Orphan Drug policy but leading initiatives in the E.U. (see E.U.)

temporary approval (ATU) for "orphan" drugs may be granted based on available data for a time period of 3 months to 1 year

approval may apply to a cohort of patients or may require release on an individual patient basis

no Orphan Drug policy (see E.U.)

historically, some government research funds have been made available for the development of drugs for rare diseases

practitioners can procure unapproved drugs for individual patients based on clinical judgement

an Application Under Exceptional Circumstances can be made when insufficient information on the safety, quality and/or efficacy of a product exists or when it may be unethical to collect such information. Manufacturers must detail PMS studies to be undertaken.

provision for abating fees for NDA for small market drugs under cost recovery