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Avant-propos - Synthèse - 1. Objectifs stratégiques et population cible - 2. Le contexte ... Venir en aide aux plus vulnérables - 3. Mesures proposées par domaine d'action - 4. Timing de la mise en œuvre, du suivi et de l'évaluation - 5. Analyse de l'impact budgétaire - 6. Annexes

Ziekten en aandoeningen (niet-HRM) : Zeldzame aandoeningen --- Maladies et affections (non GRH) : Affections rares

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This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar levels of investment are needed from a pharmaceutical company to bring a drug to the market for both

Orphan Drug Production -- periodicals. --- Orphan drugs --- Drug approval --- Drug and Narcotic Control --- Evaluation Studies as Topic --- Drug Industry --- Industry --- Legislation, Drug --- Investigative Techniques --- Analytical, Diagnostic and Therapeutic Techniques and Equipment --- Social Control, Formal --- Technology, Industry, and Agriculture --- Sociology --- Technology, Industry, Agriculture --- Social Sciences --- Anthropology, Education, Sociology and Social Phenomena --- Orphan Drug Production --- Drug Approval --- Health & Biological Sciences --- Business & Economics --- Industries --- Pharmacy, Therapeutics, & Pharmacology --- Law and legislation --- Orphan drugs. --- Drugs of limited commercial value --- Nonprofitable drugs --- Drugs --- Ziekten en aandoeningen (niet-HRM) : Zeldzame aandoeningen --- Maladies et affections (non GRH) : Affections rares