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The French Medical Pharmacology is structured and positioned to play a major role in orphan drug research and development due to the required expertise concentrated into pharmacology departments, exclusively implemented within the French university hospitals, public hospitals that are linked to a medical school (and often a pharmacy school) with numerous <jats:styled-content style=\"fixed-case\">INSERM<\/jats:styled-content> or <jats:styled-content style=\"fixed-case\">CNRS<\/jats:styled-content> labelled research units.<\/jats:p><jats:p>In addition, these structures allow a close collaboration between researchers, academic institutions and biotech start\u2010up (most of them being spin\u2010off of the academic structures). Also, within university hospitals are located the clinical investigation centres, linking to the <jats:styled-content style=\"fixed-case\">F\u2010CRIN<\/jats:styled-content> network and also to Inserm and hospitals, that enable care staff and researchers to be associated and clinical research protocols to be carried out on site, in full respect with ethic and regulatory aspects.<\/jats:p><jats:p>As a consequence, this intra and multidisciplinary expertise offers all resource to elaborate a tailored approach for orphan drug development, in new entities as well as in repositioning. For preclinical development: drug screening, candidate selection (taking into account <jats:styled-content style=\"fixed-case\">PK<\/jats:styled-content>, metabolism, variability and potential toxicity) and preclinical models (<jats:styled-content style=\"fixed-case\">iPS<\/jats:styled-content>, animal models) that could allow a better translation to human research. For clinical development, we will mention here dose determination, safety evaluation and Orphan Drug Designation and Protocol Assistance preparation and submission. For post marketing evaluation and surveys, the pharmacovigilance, addictovigilance and pharmacoepidemiology expertise, combined with access to large databases allow a better approach to orphan drug use and safety.<\/jats:p><jats:p>As outlined through two success stories (Charcot Marie Tooth, vascular Ehlers\u2010Danlos syndrome)<jats:bold>,<\/jats:bold> the added value of French Medical Pharmacology structures and expertise has been evidenced in the know\u2010how, multidimensional and multidisciplinary approaches, allowing the development of numerous drugs that have been granted with Orphan Drug Designation and later Market Approval. Even if specific and possibly even more, the field of orphan drugs requires the respect of highest standards of safety and quality. French Medical Pharmacology intends to continue on this way and constantly improve his involvement in this field, committed to a single objective: answer the unmet medical need of patients with rare diseases.<\/jats:p>","DOI":"10.1111\/fcp.12314","type":"journal-article","created":{"date-parts":[[2017,8,5]],"date-time":"2017-08-05T11:12:37Z","timestamp":1501931557000},"page":"685-694","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Pharmacology and drug development in rare diseases: the attractiveness and expertise of the French medical pharmacology"],"prefix":"10.1111","volume":"31","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-7172-7835","authenticated-orcid":false,"given":"Jo\u00eblle","family":"Micallef","sequence":"first","affiliation":[{"name":"Service de Pharmacologie Clinique et Pharmacovigilance, AP\u2010HM 13385 Marseille France"},{"name":"Pharmacologie int\u00e9gr\u00e9e et interface clinique et industriel Orphandev\u2010FCRIN Institut des Neurosciences Timone \u2013 AMU\u2010CNRS 7289 Aix Marseille Universit\u00e9 13385 Marseille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pierre","family":"Boutouyrie","sequence":"additional","affiliation":[{"name":"Service de Pharmacologie HEGP Assistance Publique H\u00f4pitaux de Paris Universit\u00e9 Paris Descartes, Sorbonne Paris\u2010cit\u00e9, INSERM U970 7598 Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Olivier","family":"Blin","sequence":"additional","affiliation":[{"name":"Service de Pharmacologie Clinique et Pharmacovigilance, AP\u2010HM 13385 Marseille France"},{"name":"Pharmacologie int\u00e9gr\u00e9e et interface clinique et industriel Orphandev\u2010FCRIN Institut des Neurosciences Timone \u2013 AMU\u2010CNRS 7289 Aix Marseille Universit\u00e9 13385 Marseille France"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2017,8,24]]},"reference":[{"key":"e_1_2_7_2_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(08)60872-7"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrd3595"},{"key":"e_1_2_7_4_1","unstructured":"Agence Nationale de Recherche (ANR). 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