-
1
-
-
0003187521
-
Regulation (EC) No 141/2000 of the European Parliament and the Council of 16 December 1999 on orphan medicinal products
-
L 18/1
-
Regulation (EC) No 141/2000 of the European Parliament and the Council of 16 December 1999 on orphan medicinal products. European Commission, Official Journal of the European Communities 2000 L 18/1
-
(2000)
Official Journal of the European Communities
-
-
Commission, E.1
-
2
-
-
84872428805
-
The portal for rare diseases and orphan drugs
-
2011. Ref Type: Electronic Citation
-
The portal for rare diseases and orphan drugs. Orphanet, Orphanet 2011 9 5 2011. Ref Type: Electronic Citation
-
(2011)
Orphanet
, pp. 9-5
-
-
Orphanet1
-
3
-
-
79960182535
-
Phase I: Overview of the conditions for marketing orphan drugs in Europe
-
Alcimed. Study on orphan drugs 2010. Ref Type: Electronic Citation
-
Phase I: overview of the conditions for marketing orphan drugs in Europe. Alcimed. Study on orphan drugs, Koning Boudewijnstichting 2006 18 3 2010. Ref Type: Electronic Citation
-
(2006)
Koning Boudewijnstichting
, pp. 18-3
-
-
-
5
-
-
78649700786
-
Rare diseases, orphan drugs and their regulation: Questions and misconceptions
-
10.1038/nrd3275. 21060315
-
Rare diseases, orphan drugs and their regulation: questions and misconceptions. E Tambuyzer, Nat Rev Drug Discov 2010 9 921 929 10.1038/nrd3275 21060315
-
(2010)
Nat Rev Drug Discov
, vol.9
, pp. 921-929
-
-
Tambuyzer, E.1
-
6
-
-
22144477549
-
Adopting an orphan
-
DOI 10.1038/sj.embor.7400450
-
Adopting an orphan. A Rinaldi, EMBO Rep 2005 6 507 510 10.1038/sj.embor.7400450 15940282 (Pubitemid 40973956)
-
(2005)
EMBO Reports
, vol.6
, Issue.6
, pp. 507-510
-
-
Rinaldi, A.1
-
7
-
-
79960196412
-
Drugs for rare diseases - Orphan designation status influences price
-
10.2165/11531800-000000000-00000. 21174479
-
Drugs for rare diseases - orphan designation status influences price. E Picavet M Dooms D Cassiman S Simoens, Appl Health Econ Health Policy 2011 9 1 5 10.2165/11531800-000000000-00000 21174479
-
(2011)
Appl Health Econ Health Policy
, vol.9
, pp. 1-5
-
-
Picavet, E.1
Dooms, M.2
Cassiman, D.3
Simoens, S.4
-
9
-
-
23944501764
-
Potential interactions of the Orphan Drug Act and pharmacogenomics: A flood of orphan drugs and abuses?
-
Potential interactions of the Orphan Drug Act and pharmacogenomics: a flood of orphan drugs and abuses? D Loughnot, Am J Law Med 2005 31 365 380 16146295 (Pubitemid 41192518)
-
(2005)
American Journal of Law and Medicine
, vol.31
, Issue.2-3
, pp. 365-380
-
-
Loughnot, D.1
-
10
-
-
41249093220
-
Exploring emerging technologies using metaphors - A study of orphan drugs and pharmacogenomics
-
10.1016/j.socscimed.2008.01.012. 18308439
-
Exploring emerging technologies using metaphors - a study of orphan drugs and pharmacogenomics. W Boon E Moors, Soc Sci Med 2008 66 1915 1927 10.1016/j.socscimed.2008.01.012 18308439
-
(2008)
Soc Sci Med
, vol.66
, pp. 1915-1927
-
-
Boon, W.1
Moors, E.2
-
11
-
-
33747050446
-
Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products
-
DOI 10.1111/j.1365-2125.2006.02654.x
-
Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products. JW Dear P Lilitkarntakul DJ Webb, Br J Clin Pharmacol 2006 62 264 271 10.1111/j.1365-2125.2006.02654.x 16934041 (Pubitemid 44215525)
-
(2006)
British Journal of Clinical Pharmacology
, vol.62
, Issue.3
, pp. 264-271
-
-
Dear, J.W.1
Lilitkarntakul, P.2
Webb, D.J.3
-
12
-
-
0010280366
-
-
Department of Health London, Department of Health. 2010. Ref Type: Report
-
Pharmaceutical Price Regulation Scheme. Department of Health, London, Department of Health 2010 18 3 2010. Ref Type: Report
-
(2010)
Pharmaceutical Price Regulation Scheme
, pp. 18-3
-
-
-
13
-
-
79960187438
-
AWMSG policy on ultra-orphan drugs
-
All Wales Medicines Strategy Group 2011. Ref Type: Electronic Citation
-
AWMSG policy on ultra-orphan drugs. All Wales Medicines Strategy Group, All Wales Medicines Strategy Group 2011 21 2 2011. Ref Type: Electronic Citation
-
(2011)
All Wales Medicines Strategy Group
, pp. 21-2
-
-
-
14
-
-
34247504244
-
Assessing the economic challenges posed by orphan drugs
-
DOI 10.1017/S0266462307051550, PII S0266462307051550
-
Assessing the economic challenges posed by orphan drugs. MF Drummond DA Wilson P Kanavos P Ubel J Rovira, Int J Technol Assess Health Care 2007 23 36 42 17234015 (Pubitemid 46648298)
-
(2007)
International Journal of Technology Assessment in Health Care
, vol.23
, Issue.1
, pp. 36-42
-
-
Drummond, M.F.1
Wilson, D.A.2
Kanavos, P.3
Ubel, P.4
Rovira, J.5
-
15
-
-
84872428805
-
The portal for rare diseases and orphan drugs
-
2010. Ref Type: Electronic Citation
-
The portal for rare diseases and orphan drugs. Orphanet, Orphanet 2010 11 6 2010. Ref Type: Electronic Citation
-
(2010)
Orphanet
, pp. 11-6
-
-
Orphanet1
-
16
-
-
84859005778
-
Orphan drugs. Relating price determination to disease prevalence
-
10.1136/bmj.c4615. 20736283
-
Orphan drugs. Relating price determination to disease prevalence. A Messori A Cicchetti L Patregani, BMJ 2010 341 4615 10.1136/bmj.c4615 20736283
-
(2010)
BMJ
, vol.341
, pp. 34615
-
-
Messori, A.1
Cicchetti, A.2
Patregani, L.3
-
17
-
-
78549264507
-
The problem of orphan drugs
-
10.1136/bmj.c6456. 21081597
-
The problem of orphan drugs. RE Ferner DA Hughes, BMJ 2010 341 6456 10.1136/bmj.c6456 21081597
-
(2010)
BMJ
, vol.341
, pp. 36456
-
-
Ferner, R.E.1
Hughes, D.A.2
-
18
-
-
76949104143
-
Factors associated with success of market authorisation applications for pharmaceutical drugs submitted to the European Medicines Agency
-
10.1007/s00228-009-0756-y. 19936724
-
Factors associated with success of market authorisation applications for pharmaceutical drugs submitted to the European Medicines Agency. J Regnstrom F Koenig B Aronsson T Reimer K Svendsen S Tsigkos,, et al. Eur J Clin Pharmacol 2010 66 39 48 10.1007/s00228-009-0756-y 19936724
-
(2010)
Eur J Clin Pharmacol
, vol.66
, pp. 39-48
-
-
Regnstrom, J.1
Koenig, F.2
Aronsson, B.3
Reimer, T.4
Svendsen, K.5
Tsigkos, S.6
-
19
-
-
0037837490
-
The orphan drug backlash
-
12701333
-
The orphan drug backlash. T Maeder, Sci Am 2003 288 80 87 12701333
-
(2003)
Sci Am
, vol.288
, pp. 80-87
-
-
Maeder, T.1
-
20
-
-
20044366257
-
Endothelin antagonism in pulmonary hypertension, heart failure, and beyond
-
DOI 10.1136/hrt.2004.053991
-
Endothelin antagonism in pulmonary hypertension, heart failure, and beyond. T Attina R Camidge DE Newby DJ Webb, Heart 2005 91 825 831 10.1136/hrt.2004.053991 15894792 (Pubitemid 40769240)
-
(2005)
Heart
, vol.91
, Issue.6
, pp. 825-831
-
-
Attina, T.1
Camidge, R.2
Newby, D.E.3
Webb, D.J.4
-
21
-
-
33749333428
-
MarketWatch: Prices and availability of biopharmaceuticals: An international comparison
-
DOI 10.1377/hlthaff.25.5.1353
-
Prices and availability of biopharmaceuticals: an international comparison. PM Danzon MF Furukawa, Health Aff (Millwood) 2006 25 1353 1362 10.1377/hlthaff.25.5.1353 (Pubitemid 44497636)
-
(2006)
Health Affairs
, vol.25
, Issue.5
, pp. 1353-1362
-
-
Danzon, P.M.1
Furukawa, M.F.2
-
22
-
-
31544452135
-
Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy
-
DOI 10.1503/cmaj.050706
-
Is the current approach to reviewing new drugs condemning the victims of rare diseases to death? A call for a national orphan drug review policy. JT Clarke, CMAJ 2006 174 189 190 10.1503/cmaj.050706 16415465 (Pubitemid 43166273)
-
(2006)
Canadian Medical Association Journal
, vol.174
, Issue.2
, pp. 189-190
-
-
Clarke, J.T.R.1
-
23
-
-
50649096569
-
A new model to evaluate the long-term cost effectiveness of orphan and highly specialised drugs following listing on the Australian Pharmaceutical Benefits Scheme: The Bosentan Patient Registry
-
19450082
-
A new model to evaluate the long-term cost effectiveness of orphan and highly specialised drugs following listing on the Australian Pharmaceutical Benefits Scheme: the Bosentan Patient Registry. A Owen J Sprinks A Meehan T Robb M Hardy D Kwasha, Journal of Medical Economics 2008 11 235 243 19450082
-
(2008)
Journal of Medical Economics
, vol.11
, pp. 235-243
-
-
Owen, A.1
Sprinks, J.2
Meehan, A.3
Robb, T.4
Hardy, M.5
Kwasha, D.6
-
24
-
-
3542998742
-
National institute for clinical excellence and its value judgments
-
National Institute for Clinical Excellence and its value judgments. MD Rawlins AJ Culyer, BMJ 2004 329 224 227 10.1136/bmj.329.7459.224 15271836 (Pubitemid 39013284)
-
(2004)
British Medical Journal
, vol.329
, Issue.7459
, pp. 224-227
-
-
Rawlins, M.D.1
Culyer, A.J.2
-
25
-
-
27644486590
-
Orphan drugs and the NHS: Should we value rarity?
-
Orphan drugs and the NHS: should we value rarity? C McCabe K Claxton A Tsuchiya, BMJ 2005 331 1016 1019 10.1136/bmj.331.7523.1016 16254305 (Pubitemid 41552456)
-
(2005)
British Medical Journal
, vol.331
, Issue.7523
, pp. 1016-1019
-
-
McCabe, C.1
Claxton, K.2
Tsuchiya, A.3
-
26
-
-
0035203733
-
Cost-effectiveness analysis and the consistency of decision making: Evidence from pharmaceutical reimbursement in Australia (1991 to 1996)
-
Cost-effectiveness analysis and the consistency of decision making: evidence from pharmaceutical reimbursement in australia (1991 to 1996). B George A Harris A Mitchell, Pharmacoeconomics 2001 19 1103 1109 10.2165/00019053- 200119110-00004 11735677 (Pubitemid 33135698)
-
(2001)
PharmacoEconomics
, vol.19
, Issue.11
, pp. 1103-1109
-
-
George, B.1
Harris, A.2
Mitchell, A.3
-
28
-
-
74249105349
-
-
National Institute for Health Clinical Excellence London: National Institute for Health and Clinical Excellence
-
National Institute for Health and Clinical Excellence, Appraising life-extending, end of life treatments London: National Institute for Health and Clinical Excellence 2009
-
(2009)
Appraising Life-extending, End of Life Treatments
-
-
-
29
-
-
13844272256
-
QALY maximisation and people's preferences: A methodological review of the literature
-
DOI 10.1002/hec.924
-
QALY maximisation and people's preferences: a methodological review of the literature. P Dolan R Shaw A Tsuchiya A Williams, Health Econ 2005 14 197 208 10.1002/hec.924 15386656 (Pubitemid 40253634)
-
(2005)
Health Economics
, vol.14
, Issue.2
, pp. 197-208
-
-
Dolan, P.1
Shaw, R.2
Tsuchiya, A.3
Williams, A.4
-
30
-
-
79960200442
-
Citizens Council Report Ultra Orphan Drugs
-
National Institute for Health Clinical Excellence 2010. Ref Type: Electronic Citation
-
Citizens Council Report Ultra Orphan Drugs. National Institute for Health and Clinical Excellence, National Institute for Health and Clinical Excellence 2005 19 3 2010. Ref Type: Electronic Citation
-
(2005)
National Institute for Health and Clinical Excellence
, pp. 19-3
-
-
-
31
-
-
77957171034
-
Societal views on orphan drugs: Cross sectional survey of Norwegians aged 40 to 67
-
10.1136/bmj.c4715. 20861122
-
Societal views on orphan drugs: cross sectional survey of Norwegians aged 40 to 67. AS Desser D Gyrd-Hansen JA Olsen S Grepperud IS Kristiansen, BMJ 2010 341 4715 10.1136/bmj.c4715 20861122
-
(2010)
BMJ
, vol.341
, pp. 34715
-
-
Desser, A.S.1
Gyrd-Hansen, D.2
Olsen, J.A.3
Grepperud, S.4
Kristiansen, I.S.5
-
33
-
-
33745684367
-
A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1
-
17134598
-
A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1. M Connock A Juarez-Garcia E Frew A Mans J Dretzke A Fry-Smith,, et al. Health Technol Assess 2006 10 ii 113 17134598
-
(2006)
Health Technol Assess
, vol.10
-
-
Connock, M.1
Juarez-Garcia, A.2
Frew, E.3
Mans, A.4
Dretzke, J.5
Fry-Smith, A.6
-
34
-
-
45749087693
-
Pharmaceutical risk-sharing agreements
-
DOI 10.2165/00019053-200826070-00002
-
Pharmaceutical risk-sharing agreements. JP Cook JA Vernon R Manning, Pharmacoeconomics 2008 26 551 556 10.2165/00019053-200826070-00002 18563946 (Pubitemid 351871550)
-
(2008)
PharmacoEconomics
, vol.26
, Issue.7
, pp. 551-556
-
-
Cook, J.P.1
Vernon, J.A.2
Manning, R.3
-
35
-
-
79960190101
-
Orphan Drugs Risk Share
-
2010. Ref Type: Electronic Citation
-
Orphan Drugs Risk Share. Scottish Medicines Consortium, Scottish Medicines Consortium 2010 19 3 2010. Ref Type: Electronic Citation
-
(2010)
Scottish Medicines Consortium
, pp. 19-3
-
-
Medicines Consortium, S.1
-
37
-
-
77953704571
-
Multiple sclerosis risk sharing scheme: A costly failure
-
10.1136/bmj.c1672. 20522654
-
Multiple sclerosis risk sharing scheme: a costly failure. J Raftery, BMJ 2010 340 1672 10.1136/bmj.c1672 20522654
-
(2010)
BMJ
, vol.340
, pp. 31672
-
-
Raftery, J.1
-
38
-
-
33646466804
-
Choosing the right incentive strategy for research and development in neglected diseases
-
10.2471/BLT.06.029835. 16710547
-
Choosing the right incentive strategy for research and development in neglected diseases. SM Maurer, Bull World Health Organ 2006 84 376 381 10.2471/BLT.06.029835 16710547
-
(2006)
Bull World Health Organ
, vol.84
, pp. 376-381
-
-
Maurer, S.M.1
|