-
1
-
-
0031942634
-
Orphan drug development: International program and study design issues. Optimizing pharmaceutical development: The global experience
-
Haffner ME. Orphan drug development: international program and study design issues. Optimizing pharmaceutical development: the global experience. Drug Inf J 1998;32:93-9.
-
(1998)
Drug Inf J
, vol.32
, pp. 93-99
-
-
Haffner, M.E.1
-
2
-
-
78649647008
-
-
House of Commons. Journals. 2nd session, 7 May Available: (accessed 2010 Apr. 16)
-
House of Commons. Journals. 39th Parliament, 2nd session, no. 90 (7 May 2008). Available: www2.parl.gc.ca/HousePublications /Publication.aspx?Language =E&Mode=1&Parl=39&Ses=2&DocId=3472169 9 9 (accessed 2010 Apr. 16).
-
(2008)
39th Parliament
, Issue.90
-
-
-
3
-
-
0035097082
-
Orphan drug programs/policies in Australia, Japan, and Canada
-
Scott DL, Alder S, Usui E, et al. Orphan drug programs/policies in Australia, Japan, and Canada. Drug Inf J 2001;35:1-16. (Pubitemid 32198548)
-
(2001)
Drug Information Journal
, vol.35
, Issue.1
, pp. 1-16
-
-
Scott, D.L.1
Alder, S.2
Usui, E.3
Lui, K.4
-
4
-
-
0027154172
-
The emergency drug release program: Regulatory aspects of new drug access in Canada
-
Gilron I. The Emergency Drug Release Program: regulatory aspects of new drug access in Canada. CMAJ 1993;148:1151-3. (Pubitemid 23144469)
-
(1993)
Canadian Medical Association Journal
, vol.148
, Issue.7
, pp. 1151-1153
-
-
Gilron, I.1
-
6
-
-
78649648832
-
-
Orphan Drug Policy [letter sent to associations]. Ottawa (ON): Drugs Directorate, File no: 96-037419 Available: (accessed 2010 Jan. 12)
-
Orphan Drug Policy [letter sent to associations]. Ottawa (ON): Drugs Directorate, Health Canada; 1997. File no: 96-037419. Available: www.hc-sc.gc.ca/dhp-mps /prodpharma/applic-demande/pol/orph-pol-eng.php (accessed 2010 Jan. 12).
-
(1997)
-
-
-
7
-
-
0034822074
-
Managing public payment for high-cost, highbenefit treatment: Enzyme replacement therapy for Gaucher's disease in Ontario
-
Clarke JTR, Amato D, Deber RB. Managing public payment for high-cost, highbenefit treatment: enzyme replacement therapy for Gaucher's disease in Ontario. CMAJ 2001;165:595-6.
-
(2001)
CMAJ
, vol.165
, pp. 595-596
-
-
Clarke, J.T.R.1
Amato, D.2
Deber, R.B.3
-
8
-
-
0036588777
-
Orphan drugs: Legal aspects, current situation
-
Lavandeira A. Orphan drugs: legal aspects, current situation. Haemophilia 2002;8:194-8.
-
(2002)
Haemophilia
, vol.8
, pp. 194-198
-
-
Lavandeira, A.1
-
9
-
-
65549112639
-
Orphan drug legislation: Lessons for neglected tropical diseases
-
Villa S, Compagni A, Reich MR. Orphan drug legislation: lessons for neglected tropical diseases. Int J Health Plann Manage 2009;24:27-42.
-
(2009)
Int J Health Plann Manage
, vol.24
, pp. 27-42
-
-
Villa, S.1
Compagni, A.2
Reich, M.R.3
-
10
-
-
22144477549
-
Adopting an orphan
-
Rinaldi A. Adopting an orphan. EMBO Rep 2005;6:507-10.
-
(2005)
EMBO Rep
, vol.6
, pp. 507-510
-
-
Rinaldi, A.1
-
11
-
-
85030599837
-
-
Available: (accessed 2010 July 5)
-
Australian Therapeutic Goods Administration. Drugs designated as orphan drugs. Available: www.tga.gov.au/docs/html/orphand2.htm (accessed 2010 July 5).
-
Drugs Designated As Orphan Drugs
-
-
-
13
-
-
84872660761
-
-
Available: (accessed 2010 July 3)
-
US Food and Drug Administration. Search orphan drug designations and approvals. Available: www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm (accessed 2010 July 3).
-
Search Orphan Drug Designations and Approvals
-
-
-
14
-
-
33747589887
-
Orphan diseases and drugs
-
Badyal D. Orphan diseases and drugs. Indian J Pharmacol 2006;38:299-300.
-
(2006)
Indian J Pharmacol
, vol.38
, pp. 299-300
-
-
Badyal, D.1
-
16
-
-
85030884080
-
-
Available: (accessed 2010 July 1)
-
Public Health Agency of Canada. Cancer surveillance on-line. Available: http://dsol-smed.phac-aspc.gc.ca/dsol-smed/cancer/c-time-eng.php (accessed 2010 July 1).
-
Cancer Surveillance On-line
-
-
-
18
-
-
33846691775
-
Current issues in gastrointestinal stromal tumors: Incidence, molecular biology, and contemporary treatment of localized and advanced disease
-
Raut CP, Morgan JA, Ashley SW. Current issues in gastrointestinal stromal tumors: incidence, molecular biology, and contemporary treatment of localized and advanced disease. Curr Opin Gastroenterol 2007;23:149-58.
-
(2007)
Curr Opin Gastroenterol
, vol.23
, pp. 149-158
-
-
Raut, C.P.1
Morgan, J.A.2
Ashley, S.W.3
-
19
-
-
0037905488
-
Clinical trials and rare diseases
-
Lagakos SW. Clinical trials and rare diseases. N Engl J Med 2003;348:2455-6.
-
(2003)
N Engl J Med
, vol.348
, pp. 2455-2456
-
-
Lagakos, S.W.1
-
20
-
-
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
-
Clarke JT. 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. CMAJ 2006; 174: 189-90.
-
(2006)
CMAJ
, vol.174
, pp. 189-190
-
-
Clarke, J.T.1
-
21
-
-
33645399023
-
Outpatient gatifloxacin therapy and dysglycemia in older adults
-
Park-Wyllie LY, Juurlink DN, Kopp A, et al. Outpatient gatifloxacin therapy and dysglycemia in older adults. N Engl J Med 2006;354:1352-61.
-
(2006)
N Engl J Med
, vol.354
, pp. 1352-1361
-
-
Park-Wyllie, L.Y.1
Juurlink, D.N.2
Kopp, A.3
-
22
-
-
20144365496
-
Cardiovascular risk associated with celecoxib in a clinical trial for colorectal adenoma prevention
-
Solomon SD, McMurray JJ, Pfeffer MA, et al. Cardiovascular risk associated with celecoxib in a clinical trial for colorectal adenoma prevention. N Engl J Med 2005;352:1071-80.
-
(2005)
N Engl J Med
, vol.352
, pp. 1071-1080
-
-
Solomon, S.D.1
McMurray, J.J.2
Pfeffer, M.A.3
-
23
-
-
19744380776
-
Cardiovascular events associated with rofecoxib in a colorectal adenoma chemoprevention trial
-
[Published erratum in: N Engl J Med 2006;355:221]
-
Bresalier RS, Sandler RS, Quan H, et al. Cardiovascular events associated with rofecoxib in a colorectal adenoma chemoprevention trial. [Published erratum in: N Engl J Med 2006;355:221]. N Engl J Med 2005;352:1092-102.
-
(2005)
N Engl J Med
, vol.352
, pp. 1092-1102
-
-
Bresalier, R.S.1
Sandler, R.S.2
Quan, H.3
-
24
-
-
34249676430
-
Evaluation of serious adverse drug reactions: A proactive pharmacovigilance program (RADAR) vs safety activities conducted by the Food and Drug Administration and pharmaceutical manufacturers
-
Bennett CL, Nebeker JR, Yarnold PR, et al. Evaluation of serious adverse drug reactions: a proactive pharmacovigilance program (RADAR) vs safety activities conducted by the Food and Drug Administration and pharmaceutical manufacturers. Arch Intern Med 2007;167:1041-9.
-
(2007)
Arch Intern Med
, vol.167
, pp. 1041-1049
-
-
Bennett, C.L.1
Nebeker, J.R.2
Yarnold, P.R.3
-
25
-
-
78649671703
-
-
Friedreich's Ataxia Research Alliance. Available: (accessed 2009 Mar.10)
-
Friedreich's Ataxia Research Alliance. Research and Clinical Programs. Available: www.curefa.org/registry.html (accessed 2009 Mar.10).
-
Research and Clinical Programs
-
-
-
26
-
-
33748980639
-
The French 'observatoire' on Gaucher's disease
-
Jaussaud R. The French 'observatoire' on Gaucher's disease. Eur J Intern Med 2006; 17 :S6-8.
-
(2006)
Eur J Intern Med
, vol.17
-
-
Jaussaud, R.1
-
27
-
-
78649672710
-
French observatoire on Gaucher disease (FROG) results on 107 patients
-
Jaussaud R, Javier RM, Rose C, et al. French observatoire on Gaucher disease (FROG) results on 107 patients. J Inherit Metab Dis 2007;30:106.
-
(2007)
J Inherit Metab Dis
, vol.30
, pp. 106
-
-
Jaussaud, R.1
Javier, R.M.2
Rose, C.3
-
28
-
-
33745655593
-
Rare disease research gets a boost
-
Hampton T. Rare disease research gets a boost. JAMA 2006;295:2836-8.
-
(2006)
JAMA
, vol.295
, pp. 2836-2838
-
-
Hampton, T.1
-
29
-
-
0037374498
-
The price of innovation: New estimates of drug development costs
-
DiMasi JA, Hansen RW, Grabowski HG. The price of innovation: new estimates of drug development costs. J Health Econ 2003;22:151-85.
-
(2003)
J Health Econ
, vol.22
, pp. 151-185
-
-
DiMasi, J.A.1
Hansen, R.W.2
Grabowski, H.G.3
-
32
-
-
78649640563
-
Alberta teen pleads for $500,000 drug therapy
-
Feb. 28. Available: (accessed 2009 Mar. 10)
-
CBC News. Alberta teen pleads for $500,000 drug therapy. 2008 Feb. 28. Available: www .cbc .ca/canada/calgary/story/2008/02/28/pare-drug.html (accessed 2009 Mar. 10).
-
(2008)
CBC News
-
-
-
33
-
-
78649657523
-
-
Available: (accessed 2010 July 1)
-
Canadian Agency for Drugs and Technologies in Health. Search CDR drug database. Available: www.cadth.ca/index.php/en/cdr/search (accessed 2010 July 1).
-
Search CDR Drug Database
-
-
-
34
-
-
0033651417
-
A comprehensive league table of cost-utility ratios and a sub-table of "panel-worthy" studies
-
Chapman RH, Stone PW, Sandberg EA, et al. A comprehensive league table of cost-utility ratios and a sub-table of "panel-worthy" studies. Med Decis Making 2000;20:451-67.
-
(2000)
Med Decis Making
, vol.20
, pp. 451-467
-
-
Chapman, R.H.1
Stone, P.W.2
Sandberg, E.A.3
-
36
-
-
0025811947
-
Setting health care priorities in Oregon: Cost-effectiveness meets the rule of rescue
-
Hadorn DC. Setting health care priorities in Oregon: cost-effectiveness meets the rule of rescue. JAMA 1991;265:2218-25.
-
(1991)
JAMA
, vol.265
, pp. 2218-2225
-
-
Hadorn, D.C.1
-
37
-
-
70350519055
-
Enzyme therapy for Fabry patients in jeopardy
-
Available: (accessed 2009 Sept. 3)
-
Silversides A. Enzyme therapy for Fabry patients in jeopardy. CMAJ 2009;181(6- 7):E120. Available: www.ecmaj.com/earlyreleases/4aug09-enzyme.shtml (accessed 2009 Sept. 3).
-
(2009)
CMAJ
, vol.181
, Issue.6-7
-
-
Silversides, A.1
-
38
-
-
29544434176
-
Accountability for reasonableness: Opening the black box of process
-
DOI 10.1007/s10728-005-8124-2
-
Hasman A, Holm S. Accountability for reasonableness: opening the black box of process. Health Care Anal 2005;13:261-73. (Pubitemid 43016878)
-
(2005)
Health Care Analysis
, vol.13
, Issue.4
, pp. 261-273
-
-
Hasman, A.1
Holm, S.2
-
39
-
-
0030897506
-
The safety and efficacy of oral methylnaltrexone in preventing morphine-induced delay in oral-cecal transit time
-
Yuan CS, Foss JF, Osinski J, et al. The safety and efficacy of oral methylnaltrexone in preventing morphine-induced delay in oral-cecal transit time. Clin Pharmacol Ther 1997;61:467-75. (Pubitemid 27195949)
-
(1997)
Clinical Pharmacology and Therapeutics
, vol.61
, Issue.4
, pp. 467-475
-
-
Yuan, C.-S.1
Foss, J.F.2
Osinski, J.3
Toledano, A.4
Roizen, M.F.5
Moss, J.6
-
40
-
-
32044463568
-
Adopting orphan drugs - Two dozen years of treating rare diseases
-
20062
-
Haffner ME. Adopting orphan drugs - two dozen years of treating rare diseases. N Engl J Med 20062;354:445-7.
-
N Engl J Med
, vol.354
, pp. 445-447
-
-
Haffner, M.E.1
|