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

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

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

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 drugs in JapanUCHIDA, K.Drug information journal. 1996, Vol 30, Num 1, pp 171-175, issn 0092-8615Conference Paper

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

A framework for applying unfamiliar trial designs in studies of rare diseasesGUPTA, Samir; FAUGHNAN, Marie E; TOMLINSON, George A et al.Journal of clinical epidemiology. 2011, Vol 64, Num 10, pp 1085-1094, issn 0895-4356, 10 p.Article

Database identifies FDA-approved drugs with potential to be repurposed for treatment of orphan diseasesKUI XU; COTE, Timothy R.Briefings in bioinformatics. 2011, Vol 12, Num 4, pp 341-345, issn 1467-5463, 5 p.Article

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

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

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

How did uncommon disorders become 'rare diseases'? History of a boundary objectHUYARD, Caroline.Sociology of health & illness (Print). 2009, Vol 31, Num 4, pp 463-477, issn 0141-9889, 15 p.Article

Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EUJOPPI, Roberta; BERTELE, Vittorio; GARATTINI, Silvio et al.European journal of clinical pharmacology. 2013, Vol 69, Num 4, pp 1009-1024, issn 0031-6970, 16 p.Article

Orphan drug development is progressing too slowlyJOPPI, Roberta; BERTELE, Vittorio; GARATTINI, Silvio et al.British journal of clinical pharmacology. 2006, Vol 61, Num 3, pp 355-360, issn 0306-5251, 6 p.Article

Zulassung und Erstattung von Orphan Drugs im internationalen Vergleich = Authorization and Reimbursement of Orphan Drugs in an International ComparisonROLL, K; STARGARDT, T; SCHREYÖGG, J et al.Das Gesundheitswesen (Stuttgart. Thieme). 2011, Vol 73, Num 8-9, pp 504-514, issn 0941-3790, 11 p.Article

Toward a Functional Definition of a Rare Disease for Regulatory Authorities and Funding AgenciesCLARKE, Joe T. R; COYLE, Doug; EVANS, Gerald et al.Value in health. 2014, Vol 17, Num 8, pp 757-761, issn 1098-3015, 5 p.Article

A Pilot Study of Multicriteria Decision Analysis for Valuing Orphan MedicinesSUSSEX, Jon; ROLLET, Pierrick; GARAU, Martina et al.Value in health. 2013, Vol 16, Num 8, pp 1163-1169, issn 1098-3015, 7 p.Article

Predictors of orphan drug approval in the European UnionHEEMSTRA, Harald E; DE VRUEH, Remco L; VAN WEELY, Sonja et al.European journal of clinical pharmacology. 2008, Vol 64, Num 5, pp 545-552, issn 0031-6970, 8 p.Article

Orphan drugs assessment in the centralised procedureNISTICO, Giuseppe.Annali dell' Istituto superiore di sanità. 2011, Vol 47, Num 1, pp 98-99, issn 0021-2571, 2 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

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