WHAT MAKES AN ORPHAN DRUG?HAWKES, Nigel; COHEN INVESTIGATE, Deborah.BMJ (Overseas and retired doctors ed.). 2010, Vol 341, Num 7782, pp 1076-1078, issn 1759-2151, 3 p.Article

Médicaments orphelins : L'Europe comble son retard = Orphan drugs : Europe makes its delayLa Revue Prescrire. 1998, Vol 18, Num 190, pp 868-870, issn 0247-7750Article

Les médicaments orphelins en Europe = Orphan medicinal products in EuropeHERON, Emmanuel.MS. Médecine sciences. 2005, Vol 21, pp 66-68, issn 0767-0974, 3 p., NSArticle

Orphan products-pain relief for clinical development headaches: As the cost of clinical development rises, and products take longer to reach the market, the biotechnology industry may be wise to take a closer look at orphan drug indicationsMILNE, Christopher-Paul.Nature biotechnology. 2002, Vol 20, Num 8, pp 780-784, issn 1087-0156, 5 p.Article

Market uptake of orphan drugs: a European analysisPICAVET, E; ANNEMANS, L; CLEEMPUT, I et al.Journal of clinical pharmacy and therapeutics (Print). 2012, Vol 37, Num 6, pp 664-667, issn 0269-4727, 4 p.Article

Orphan Drug development : International program and study design issues : Optimizing pharmaceutical development : The global experienceHAFFNER, M. E.Drug information journal. 1998, Vol 32, Num 1, pp 93-99, issn 0092-8615Conference Paper

Les médicaments orphelins auront bientôt droit de cité en Europe = Orphan drugMOILLE, F; WOLF, A.Technologies internationales (Strasbourg). 1997, Num 36, pp 3-6, issn 1165-8568Article

Orphan drugs and the NHS : should we value rarity?MCCABE, Christopher; CLAXTON, Karl; TSUCHIYA, Aki et al.BMJ. British medical journal (International ed.). 2005, Vol 331, Num 7523, pp 1016-1019, issn 0959-8146, 4 p.Article

Exploring emerging technologies using metaphors : A study of orphan drugs and pharmacogenomicsBOON, Wouter; MOORS, Ellen.Social science & medicine (1982). 2008, Vol 66, Num 9, pp 1915-1927, issn 0277-9536, 13 p.Article

Performance indicators for the EMEA in 2003 = Indicateurs de performance de l'EMEA en 20032004, 13 p.Book

Unlicensed and off-label drug use in children: Implications for safetyCHOONARA, Imti; CONROY, Sharon.Drug safety. 2002, Vol 25, Num 1, pp 1-5, issn 0114-5916Article

The need for worldwide policy and action plans for rare diseasesFORMAN, John; TARUSCIO, Domenica; TAMBUYZER, Erik et al.Acta paediatrica (Oslo). 2012, Vol 101, Num 8, pp 805-807, issn 0803-5253, 3 p.Article

The Impact of the Orphan Drug Act on the Development and Advancement of Neurological Products for Rare Diseases: A Descriptive ReviewBURKE, K. A; FREEMAN, S. N; IMOISILI, M. A et al.Clinical pharmacology and therapeutics. 2010, Vol 88, Num 4, pp 449-453, issn 0009-9236, 5 p.Article

Dossier. Les médicaments orphelins enfin reconnusBANGA, Bernard; GUIGNOT, Caroline.DECISION SANTE. 2008, Num 250, pp 8-13, issn 1157-6197, 6 p.Article

Orphan Drugs for Rare Diseases: Is it Time to Revisit Their Special Market Access Status?SIMOENS, Steven; CASSIMAN, David; DOOMS, Marc et al.Drugs (Basel). 2012, Vol 72, Num 11, pp 1437-1443, issn 0012-6667, 7 p.Article

Characteristics of Clinical Trials to Support Approval of Orphan vs Nonorphan Drugs for CancerKESSELHEIM, Aaron S; MYERS, Jessica A; AVORN, Jerry et al.JAMA, the journal of the American Medical Association. 2011, Vol 305, Num 22, pp 2320-2326, issn 0098-7484, 7 p.Article

Orphan drug development across Europe : bottlenecks and opportunitiesHEEMSTRA, Harald E; DE VRUEH, Remco L. A; VAN WEELY, Sonja et al.Drug discovery today. 2008, Vol 13, Num 15-16, pp 670-676, issn 1359-6446, 7 p.Article

Maladies rares : Enrichir sa panoplie thérapeutique : bilan de l'année 2007 = Orphan diseases : 2007 updateLa Revue Prescrire. 2008, Vol 28, Num 291, pp 46-47, issn 0247-7750, 2 p.Article

Développer des traitements pour les maladies rares : ERDITI, un partenariat public-privé pour la recherche thérapeutique = ERDITI, the European Rare Diseases Therapeutic Initiative : A public-private partnership is promoting research on new treatments for rare diseasesGILGENKRANTZ, Simone; BORENSZTEIN, Pascale.MS. Médecine sciences. 2006, Vol 22, Num 1, pp 84-86, issn 0767-0974, 3 p.Article

L'exemple des maladies rares : un partenariat entre recherche publique et industrie pharmaceutique : Chimiogénomique : Quelles ressources? Quelles perspectives? = The example of rare diseases. A partnership between public research and pharmaceutical industryROUSSEL, Claire; BORENSZTEIN, Pascale.Biofutur (Puteaux). 2005, Num 260, pp 32-35, issn 0294-3506, 4 p.Article

Growing pains for biopharmaceuticalsASHTON, Gabby.Nature biotechnology. 2001, Vol 19, Num 4, pp 307-311, issn 1087-0156Article

Modulating the profit motive to meet needs of the less-developed world. CommentarySCHIEPPATI, Arrigo; REMUZZI, Giuseppe; GARATTINI, Silvio et al.Lancet (British edition). 2001, Vol 358, Num 9293, issn 0140-6736, 1570,1638-1641 [5 p.]Article

What the Orphan Drug Act Has Done Lately for Children With Rare Diseases: A 10-Year AnalysisTHORAT, Chandana; KUI XU; FREEMAN, Scott N et al.Pediatrics (Evanston). 2012, Vol 129, Num 3, pp 516-521, issn 0031-4005, 6 p.Article

L'accès aux médicaments orphelins en France : entre besoins des patients, soutien à la recherche et politique de prixDE SAUNIERE, Anne.2012, 54 p.Thesis

Characteristics of orphan drug applicationsthat fail to achieve marketing approval in the USAHEEMSTRA, Harald E; LEUFKENS, Hubert G. M; CHANNING RODGERS, R. P et al.Drug discovery today. 2011, Vol 16, Num 1-2, pp 73-80, issn 1359-6446, 8 p.Article