-
1
-
-
84971656899
-
-
Orphan Designation. European Medicines Agency, London Accessed 26 October 2015
-
Orphan Designation. European Medicines Agency, London. http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general-content-000029.jsp&mid=WC0b01ac05800240ce. Accessed 26 October 2015.
-
-
-
-
2
-
-
80053379101
-
Estimating the budget impact of orphan medicines in Europe: 2010-2020
-
10.1186/1750-1172-6-62 21951518 3191371
-
Schey C, Milanova T, Hutchings A. Estimating the budget impact of orphan medicines in Europe: 2010-2020. Orphanet J Rare Dis. 2011;6:62. doi: 10.1186/1750-1172-6-62.
-
(2011)
Orphanet J Rare Dis
, vol.6
, pp. 62
-
-
Schey, C.1
Milanova, T.2
Hutchings, A.3
-
3
-
-
84971614450
-
-
Register of designated Orphan Medicinal Products Accessed 5 October 2015
-
Register of designated Orphan Medicinal Products. European Commission. Directorate General Health and Food Safety. http://ec.europa.eu/health/documents/community-register/html/orphreg.htm. Accessed 5 October 2015.
-
European Commission. Directorate General Health and Food Safety
-
-
-
4
-
-
84971614454
-
-
Orphan medicines designation (COMP). EURORDIS, Paris Accessed 5 October 2015.
-
Orphan medicines designation (COMP). EURORDIS, Paris. www.eurordis.org/content/orphan-drug-designation. Accessed 5 October 2015.
-
-
-
-
5
-
-
84971633821
-
-
List of orphan drugs with marketing authorizations Accessed 5 October 2015
-
List of orphan drugs with marketing authorizations. Orphanet Portal. http://www.orpha.net/consor/cgi-bin/Drugs-ListOrphanDrugs.php?lng=EN. Accessed 5 October 2015.
-
Orphanet Portal
-
-
-
6
-
-
2542465496
-
-
How to Apply for Designation as an Orphan Product (Orphan Drug Act) Accessed 5 October 2015
-
How to Apply for Designation as an Orphan Product (Orphan Drug Act). Food and Drug Administration, HHS. 1992. http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/ucm135122.htm. Accessed 5 October 2015.
-
(1992)
Food and Drug Administration, HHS
-
-
-
7
-
-
84971604919
-
-
REGULATION (EC) No 141/2000 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 16 December 1999 on orphan medicinal products Accessed 26 October 2015
-
REGULATION (EC) No 141/2000 OF THE EUROPEAN PARLIAMENT AND OF THE COUNCIL of 16 December 1999 on orphan medicinal products. Europena Parliament and the Council. 2000. http://ec.europa.eu/health/files/eudralex/vol-1/reg-2000-141/reg-2000-141-en.pdf. Accessed 26 October 2015.
-
(2000)
Europena Parliament and the Council
-
-
-
8
-
-
78249288172
-
Balancing economic, ethical and equity concerns in orphan drugs and rare diseases
-
McCabe C. Balancing economic, ethical and equity concerns in orphan drugs and rare diseases. EJHP Practice. 2010;16(4):22-5.
-
(2010)
EJHP Practice
, vol.16
, Issue.4
, pp. 22-25
-
-
McCabe, C.1
-
9
-
-
84866564048
-
Paying for the Orphan Drug System: Break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments?
-
doi: 10.1186/1750-1172-7-74
-
Hughes-Wilson W, Palma A, Schuurman A, Simoens S. Paying for the Orphan Drug System: Break or bend? Is it time for a new evaluation system for payers in Europe to take account of new rare disease treatments? Orphanet J Rare Dis. 2012;7:74. doi: 10.1186/1750-1172-7-74.
-
(2012)
Orphanet J Rare Dis.
, vol.7
, pp. 74
-
-
Hughes-Wilson, W.1
Palma, A.2
Schuurman, A.3
Simoens, S.4
-
10
-
-
69149107165
-
Preferred reporting items for systematic reviews and meta-analyses: The PRISMA statement
-
19622511 w64
-
Moher D, Liberati A, Tetzlaff J, Altman DG. Preferred reporting items for systematic reviews and meta-analyses: the PRISMA statement. Ann Intern Med. 2009;151(4):264-9. w64.
-
(2009)
Ann Intern Med
, vol.151
, Issue.4
, pp. 264-269
-
-
Moher, D.1
Liberati, A.2
Tetzlaff, J.3
Altman, D.G.4
-
11
-
-
84867335647
-
Challenges to orphan drugs access in Eastern Europe: The case of Bulgaria
-
22939047
-
Iskrov G, Miteva-Katrandzhieva T, Stefanov R. Challenges to orphan drugs access in Eastern Europe: the case of Bulgaria. Health Policy. 2012;108(1):10-8.
-
(2012)
Health Policy
, vol.108
, Issue.1
, pp. 10-18
-
-
Iskrov, G.1
Miteva-Katrandzhieva, T.2
Stefanov, R.3
-
12
-
-
84899490637
-
Insight into reimbursement decision-making criteria in Bulgaria: Implications for orphan drugs
-
Iskrov GG, Raycheva RD, Stefanov RS. Insight into reimbursement decision-making criteria in Bulgaria: implications for orphan drugs. Folia Med. 2013;55(3-4):80-6.
-
(2013)
Folia Med
, vol.55
, Issue.3-4
, pp. 80-86
-
-
Iskrov, G.G.1
Raycheva, R.D.2
Stefanov, R.S.3
-
13
-
-
84908159091
-
Rare diseases and orphan drugs: Latvian story
-
10.1186/s13023-014-0147-z 25231378 4172941
-
Logviss K, Krievins D, Purvina S. Rare diseases and orphan drugs: Latvian story. Orphanet J Rare Dis. 2014;9(1):147. doi: 10.1186/s13023-014-0147-z.
-
(2014)
Orphanet J Rare Dis
, vol.9
, Issue.1
, pp. 147
-
-
Logviss, K.1
Krievins, D.2
Purvina, S.3
-
14
-
-
84868266807
-
An insight on differences in availability and reimbursement of orphan medicines among Serbia, Bulgaria and Sweden
-
Pavlović N, Stanimirov B, Stojančević M, Paut-Kusturica M, Stoimenova A, Goločorbin-Kon S, et al. An insight on differences in availability and reimbursement of orphan medicines among Serbia, Bulgaria and Sweden. Biotechnol Biotechnol Equip. 2012;26(5):3236-41.
-
(2012)
Biotechnol Biotechnol Equip
, vol.26
, Issue.5
, pp. 3236-3241
-
-
Pavlović, N.1
Stanimirov, B.2
Stojančević, M.3
Paut-Kusturica, M.4
Stoimenova, A.5
Goločorbin-Kon, S.6
-
15
-
-
84868378943
-
Market uptake of orphan drugs - A European analysis
-
1:STN:280:DC%2BC38jks1Cntg%3D%3D 22731105
-
Picavet E, Annemans L, Cleemput I, Cassiman D, Simoens S. Market uptake of orphan drugs - A European analysis. J Clin Pharm Ther. 2012;37(6):664-7.
-
(2012)
J Clin Pharm Ther
, vol.37
, Issue.6
, pp. 664-667
-
-
Picavet, E.1
Annemans, L.2
Cleemput, I.3
Cassiman, D.4
Simoens, S.5
-
16
-
-
84864118282
-
Quantum of effectiveness evidence in FDA's approval of orphan drugs
-
Sasinowski F. Quantum of effectiveness evidence in FDA's approval of orphan drugs. Drug Inf J. 2012;46:238-63.
-
(2012)
Drug Inf J
, vol.46
, pp. 238-263
-
-
Sasinowski, F.1
-
17
-
-
84871184065
-
What is wrong with orphan drug policies?
-
23244823
-
Côté A, Keating B. What is wrong with orphan drug policies? Value Health. 2012;15(8):1185-91.
-
(2012)
Value Health
, vol.15
, Issue.8
, pp. 1185-1191
-
-
Côté, A.1
Keating, B.2
-
18
-
-
84862263456
-
Transferability of National Institute for Health and Clinical Excellence recommendations for pharmaceutical therapies in oncology to Central-Eastern European countries
-
10.1111/j.1365-2354.2012.01351.x 1:STN:280:DC%2BC38rlslGluw%3D%3D
-
Kalo Z, Landa K, Dolezal T, Voko Z. Transferability of National Institute for Health and Clinical Excellence recommendations for pharmaceutical therapies in oncology to Central-Eastern European countries. Eur J Cancer Care. 2012;21(4):442-9. doi: 10.1111/j.1365-2354.2012.01351.x.
-
(2012)
Eur J Cancer Care
, vol.21
, Issue.4
, pp. 442-449
-
-
Kalo, Z.1
Landa, K.2
Dolezal, T.3
Voko, Z.4
-
19
-
-
33747050446
-
Are rare diseases still orphans or happily adopted? the challenges of developing and using orphan medicinal products
-
10.1111/j.1365-2125.2006.02654.x 16934041 1885144
-
Dear JW, Lilitkarntakul P, Webb DJ. Are rare diseases still orphans or happily adopted? The challenges of developing and using orphan medicinal products. Br J Clin Pharmacol. 2006;62(3):264-71. doi: 10.1111/j.1365-2125.2006.02654.x.
-
(2006)
Br J Clin Pharmacol
, vol.62
, Issue.3
, pp. 264-271
-
-
Dear, J.W.1
Lilitkarntakul, P.2
Webb, D.J.3
-
20
-
-
84881540834
-
Systematic review of available evidence on 11 high-priced inpatient orphan drugs
-
10.1186/1750-1172-8-124 23947946 3751719
-
Kanters TA, de Sonneville-Koedoot C, Redekop WK, Hakkaart L. Systematic review of available evidence on 11 high-priced inpatient orphan drugs. Orphanet J Rare Dis. 2013;8:124. doi: 10.1186/1750-1172-8-124.
-
(2013)
Orphanet J Rare Dis
, vol.8
, pp. 124
-
-
Kanters, T.A.1
De Sonneville-Koedoot, C.2
Redekop, W.K.3
Hakkaart, L.4
-
21
-
-
84923593364
-
Generating health technology assessment evidence for rare diseases
-
10.1017/s0266462314000464 25407328
-
Facey K, Granados A, Guyatt G, Kent A, Shah N, van der Wilt GJ, et al. Generating health technology assessment evidence for rare diseases. Int J Technol Assess Health Care. 2014;30(4):416-22. doi: 10.1017/s0266462314000464.
-
(2014)
Int J Technol Assess Health Care
, vol.30
, Issue.4
, pp. 416-422
-
-
Facey, K.1
Granados, A.2
Guyatt, G.3
Kent, A.4
Shah, N.5
Van Der Wilt, G.J.6
-
22
-
-
84894505841
-
Strategies for postmarketing surveillance of drugs for rare diseases
-
1:CAS:528:DC%2BC2cXmtlWmsA%3D%3D 24193169
-
Kesselheim AS, Gagne JJ. Strategies for postmarketing surveillance of drugs for rare diseases. Clin Pharmacol Ther. 2014;95(3):265-8.
-
(2014)
Clin Pharmacol Ther
, vol.95
, Issue.3
, pp. 265-268
-
-
Kesselheim, A.S.1
Gagne, J.J.2
-
24
-
-
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
-
10.1503/cmaj.050706 16415465 1329458
-
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(2):189-90. doi: 10.1503/cmaj.050706.
-
(2006)
CMAJ
, vol.174
, Issue.2
, pp. 189-190
-
-
Clarke, J.T.1
-
25
-
-
84866354190
-
An evaluation framework for funding drugs for rare diseases
-
22999151
-
Winquist E, Bell CM, Clarke JTR, Evans G, Martin J, Sabharwal M, et al. An evaluation framework for funding drugs for rare diseases. Value Health. 2012;15(6):982-6.
-
(2012)
Value Health
, vol.15
, Issue.6
, pp. 982-986
-
-
Winquist, E.1
Bell, C.M.2
Clarke, J.T.R.3
Evans, G.4
Martin, J.5
Sabharwal, M.6
-
26
-
-
66749180384
-
Evidence and values: Requirements for public reimbursement of drugs for rare diseases - A case study in oncology
-
e282-e94
-
Laupacis A. Evidence and values: requirements for public reimbursement of drugs for rare diseases - A case study in oncology. Can J Clin Pharmacol. 2009;16(2):e282-e94.
-
(2009)
Can J Clin Pharmacol
, vol.16
, Issue.2
-
-
Laupacis, A.1
-
27
-
-
77953423858
-
Orphan drug pricing and payer management in the United States: Are we approaching the tipping point?
-
25126305 4106547
-
Hyde R, Dobrovolny D. Orphan drug pricing and payer management in the United States: are we approaching the tipping point? Am Health Drug Benefits. 2010;3(1):15-22.
-
(2010)
Am Health Drug Benefits
, vol.3
, Issue.1
, pp. 15-22
-
-
Hyde, R.1
Dobrovolny, D.2
-
28
-
-
79952606431
-
Priority setting for orphan drugs: An international comparison
-
20961647
-
Rosenberg-Yunger ZRS, Daar AS, Thorsteinsdóttir H, Martin DK. Priority setting for orphan drugs: an international comparison. Health Policy. 2011;100(1):25-34.
-
(2011)
Health Policy
, vol.100
, Issue.1
, pp. 25-34
-
-
Rosenberg-Yunger, Z.R.S.1
Daar, A.S.2
Thorsteinsdóttir, H.3
Martin, D.K.4
-
29
-
-
84925513451
-
Value-based reimbursement decisions for orphan drugs: A scoping review and decision framework
-
10.1007/s40273-014-0235-x 25412735 4342524
-
Paulden M, Stafinski T, Menon D, McCabe C. Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework. Pharmacoeconomics. 2015;33(3):255-69. doi: 10.1007/s40273-014-0235-x.
-
(2015)
Pharmacoeconomics
, vol.33
, Issue.3
, pp. 255-269
-
-
Paulden, M.1
Stafinski, T.2
Menon, D.3
McCabe, C.4
-
30
-
-
84890312186
-
A pilot study of multicriteria decision analysis for valuing orphan medicines
-
24326170
-
Sussex J, Rollet P, Garau M, Schmitt C, Kent A, Hutchings A. A pilot study of multicriteria decision analysis for valuing orphan medicines. Value Health. 2013;16(8):1163-9.
-
(2013)
Value Health
, vol.16
, Issue.8
, pp. 1163-1169
-
-
Sussex, J.1
Rollet, P.2
Garau, M.3
Schmitt, C.4
Kent, A.5
Hutchings, A.6
-
31
-
-
78649652812
-
Policy alternatives for treatments for rare diseases
-
E787-E92 20624867 2988562
-
Panju AH, Bell CM. Policy alternatives for treatments for rare diseases. CMAJ. 2010;182(17):E787-E92.
-
(2010)
CMAJ
, vol.182
, Issue.17
-
-
Panju, A.H.1
Bell, C.M.2
-
32
-
-
79952581846
-
Access to orphan drugs despite poor quality of clinical evidence
-
21395641 3080635
-
Dupont AG, Van Wilder PB. Access to orphan drugs despite poor quality of clinical evidence. Br J Clin Pharmacol. 2011;71(4):488-96.
-
(2011)
Br J Clin Pharmacol
, vol.71
, Issue.4
, pp. 488-496
-
-
Dupont, A.G.1
Van Wilder, P.B.2
-
33
-
-
84949040660
-
Orphan drug considerations in Health Technology Assessment in eight European countries
-
Tordrup D, Tzouma V, Kanavos P. Orphan drug considerations in Health Technology Assessment in eight European countries. Rare Dis Orphan Drugs. 2014;1(3):86-97.
-
(2014)
Rare Dis Orphan Drugs
, vol.1
, Issue.3
, pp. 86-97
-
-
Tordrup, D.1
Tzouma, V.2
Kanavos, P.3
-
34
-
-
27744461277
-
Drugs for exceptionally rare diseases: Do they deserve special status for funding?
-
1:STN:280:DC%2BD2MrlvVKrsg%3D%3D 16203824
-
Hughes DA, Tunnage B, Yeo ST. Drugs for exceptionally rare diseases: Do they deserve special status for funding? QJM. 2005;98(11):829-36.
-
(2005)
QJM
, vol.98
, Issue.11
, pp. 829-836
-
-
Hughes, D.A.1
Tunnage, B.2
Yeo, S.T.3
-
35
-
-
79958849417
-
Pricing and reimbursement of orphan drugs: The need for more transparency
-
2011 doi: 10.1186/1750-1172-6-42
-
Simoens S. Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis. 2011;6:42. doi: 10.1186/1750-1172-6-42.
-
Orphanet J Rare Dis.
, vol.6
, pp. 42
-
-
Simoens, S.1
-
36
-
-
84873476022
-
Cost-effectiveness assessment of orphan drugs: A scientific and political conundrum
-
23329382
-
Simoens S, Picavet E, Dooms M, Cassiman D, Morel T. Cost-effectiveness assessment of orphan drugs: a scientific and political conundrum. Appl Health Econ Health Policy. 2013;11(1):1-3.
-
(2013)
Appl Health Econ Health Policy
, vol.11
, Issue.1
, pp. 1-3
-
-
Simoens, S.1
Picavet, E.2
Dooms, M.3
Cassiman, D.4
Morel, T.5
-
37
-
-
66349107417
-
Evidence and values: Requirements for public reimbursement of drugs for rare diseases - A case study in oncology
-
e273-e81 19439771
-
Drummond M, Evans B, LeLorier J, Karakiewicz P, Martin D, Tugwell P, et al. Evidence and values: requirements for public reimbursement of drugs for rare diseases - A case study in oncology. Can J Clin Pharmacol. 2009;16(2):e273-e81.
-
(2009)
Can J Clin Pharmacol
, vol.16
, Issue.2
-
-
Drummond, M.1
Evans, B.2
LeLorier, J.3
Karakiewicz, P.4
Martin, D.5
Tugwell, P.6
-
38
-
-
84902168946
-
Orphan drugs policies: A suitable case for treatment
-
24435513
-
Drummond M, Towse A. Orphan drugs policies: a suitable case for treatment. Eur J Health Econ. 2014;15(4):335-40.
-
(2014)
Eur J Health Econ
, vol.15
, Issue.4
, pp. 335-340
-
-
Drummond, M.1
Towse, A.2
-
39
-
-
84871205095
-
What is wrong with orphan drug policies? Suggestions for ways forward
-
23244822
-
Kanavos P, Nicod E. What is wrong with orphan drug policies? Suggestions for ways forward. Value Health. 2012;15(8):1182-4.
-
(2012)
Value Health
, vol.15
, Issue.8
, pp. 1182-1184
-
-
Kanavos, P.1
Nicod, E.2
-
40
-
-
84867391574
-
Dutch doctors call for EU evaluation of cost effectiveness of high cost orphan drugs
-
doi: 10.1136/bmj.e5461
-
Sheldon T. Dutch doctors call for EU evaluation of cost effectiveness of high cost orphan drugs. BMJ. 2012;345:e5461. doi: 10.1136/bmj.e5461.
-
(2012)
BMJ.
, vol.345
, pp. e5461
-
-
Sheldon, T.1
-
41
-
-
17544371544
-
Managing drugs for rare genetic diseases: Trends and insights
-
7-5460, 63-64; quiz 66-67
-
Zitter M. Managing drugs for rare genetic diseases: trends and insights. Manag Care. 2005;14(2):52-4, 7-5460, 63-64; quiz 66-67.
-
(2005)
Manag Care.
, vol.14
, Issue.2
, pp. 52-54
-
-
Zitter, M.1
-
42
-
-
34250719095
-
Assessing the economic challenges posed by orphan drugs: A response to McCabe et al. [2]
-
Drummond MF, Wilson DA, Kanavos P, Ubel PA, Rovira J. Assessing the economic challenges posed by orphan drugs: a response to McCabe et al. [2]. Int J Technol Assess Health Care. 2007;23(3):401-4.
-
(2007)
Int J Technol Assess Health Care
, vol.23
, Issue.3
, pp. 401-404
-
-
Drummond, M.F.1
Wilson, D.A.2
Kanavos, P.3
Ubel, P.A.4
Rovira, J.5
-
43
-
-
34250772257
-
Assessing the economic challenges posed by orphan drugs: A comment on Drummond et al. [1]
-
17579945
-
McCabe C, Tsuchiya A, Claxton K, Raftery J. Assessing the economic challenges posed by orphan drugs: a comment on Drummond et al. [1]. Int J Technol Assess Health Care. 2007;23(3):397-401.
-
(2007)
Int J Technol Assess Health Care
, vol.23
, Issue.3
, pp. 397-401
-
-
McCabe, C.1
Tsuchiya, A.2
Claxton, K.3
Raftery, J.4
-
44
-
-
79952044389
-
Economic considerations in the provision of treatments for rare diseases
-
doi: 10.1007/978-90-481-9485-8-13
-
McCabe C, Edlin R, Round J. Economic considerations in the provision of treatments for rare diseases. Adv Exp Med Biol. 2010;686:211-22. doi: 10.1007/978-90-481-9485-8-13.
-
(2010)
Adv Exp Med Biol.
, vol.686
, pp. 211-222
-
-
McCabe, C.1
Edlin, R.2
Round, J.3
-
45
-
-
77956169455
-
A comparative study of European rare disease and orphan drug markets
-
20800761
-
Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S. A comparative study of European rare disease and orphan drug markets. Health Policy. 2010;97(2-3):173-9.
-
(2010)
Health Policy
, vol.97
, Issue.2-3
, pp. 173-179
-
-
Denis, A.1
Mergaert, L.2
Fostier, C.3
Cleemput, I.4
Simoens, S.5
-
46
-
-
77956290687
-
Issues surrounding orphan disease and orphan drug policies in Europe
-
20804226
-
Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S. Issues surrounding orphan disease and orphan drug policies in Europe. Appl Health Econ Health Policy. 2010;8(5):343-50.
-
(2010)
Appl Health Econ Health Policy
, vol.8
, Issue.5
, pp. 343-350
-
-
Denis, A.1
Mergaert, L.2
Fostier, C.3
Cleemput, I.4
Simoens, S.5
-
47
-
-
84909642930
-
Opportunity cost of funding drugs for rare diseases: The cost-effectiveness of eculizumab in paroxysmal nocturnal hemoglobinuria
-
10.1177/0272989x14539731
-
Coyle D, Cheung MC, Evans GA. Opportunity cost of funding drugs for rare diseases: the cost-effectiveness of eculizumab in paroxysmal nocturnal hemoglobinuria. Med Decis Making. 2014. doi: 10.1177/0272989x14539731.
-
(2014)
Med Decis Making
-
-
Coyle, D.1
Cheung, M.C.2
Evans, G.A.3
-
48
-
-
78249254289
-
Variations in access and use of orphan drugs among EU member states
-
Heemstra HE. Variations in access and use of orphan drugs among EU member states. EJHP Practice. 2010;16(4):25-7.
-
(2010)
EJHP Practice
, vol.16
, Issue.4
, pp. 25-27
-
-
Heemstra, H.E.1
-
49
-
-
84907940929
-
Orphan drugs expenditure in the Netherlands in the period 2006-2012
-
10.1186/s13023-014-0154-0 25304026 4195995
-
Kanters TA, Steenhoek A, Hakkaart L. Orphan drugs expenditure in the Netherlands in the period 2006-2012. Orphanet J Rare Dis. 2014;9:154. doi: 10.1186/s13023-014-0154-0.
-
(2014)
Orphanet J Rare Dis
, vol.9
, pp. 154
-
-
Kanters, T.A.1
Steenhoek, A.2
Hakkaart, L.3
-
50
-
-
84896704935
-
Estimating the budget impact of orphan drugs in Sweden and France 2013-2020
-
10.1186/1750-1172-9-22 24524281 3930763
-
Hutchings A, Schey C, Dutton R, Achana F, Antonov K. Estimating the budget impact of orphan drugs in Sweden and France 2013-2020. Orphanet J Rare Dis. 2014;9:22. doi: 10.1186/1750-1172-9-22.
-
(2014)
Orphanet J Rare Dis
, vol.9
, pp. 22
-
-
Hutchings, A.1
Schey, C.2
Dutton, R.3
Achana, F.4
Antonov, K.5
-
51
-
-
84881482075
-
Sustainable rare diseases business and drug access: No time for misconceptions
-
Rollet P, Lemoine A, Dunoyer M. Sustainable rare diseases business and drug access: no time for misconceptions. Orphanet J Rare Dis. 2013;8(1):1-9.
-
(2013)
Orphanet J Rare Dis
, vol.8
, Issue.1
, pp. 1-9
-
-
Rollet, P.1
Lemoine, A.2
Dunoyer, M.3
-
52
-
-
84908218130
-
Reimbursement of orphan drugs in Belgium: What (else) matters?
-
10.1186/s13023-014-0139-z 25208770 4172832
-
Picavet E, Cassiman D, Simoens S. Reimbursement of orphan drugs in Belgium: what (else) matters? Orphanet J Rare Dis. 2014;9(1):139. doi: 10.1186/s13023-014-0139-z.
-
(2014)
Orphanet J Rare Dis
, vol.9
, Issue.1
, pp. 139
-
-
Picavet, E.1
Cassiman, D.2
Simoens, S.3
-
53
-
-
84905915127
-
Application of a policy framework for the public funding of drugs for rare diseases
-
S774-S9
-
Winquist E, Coyle D, Clarke JTR, Evans GA, Seager C, Chan W, et al. Application of a policy framework for the public funding of drugs for rare diseases. J Gen Intern Med. 2014;29(SUPPL. 3):S774-S9.
-
(2014)
J Gen Intern Med
, vol.293
-
-
Winquist, E.1
Coyle, D.2
Clarke, J.T.R.3
Evans, G.A.4
Seager, C.5
Chan, W.6
-
54
-
-
77953354292
-
Budget impact analysis of orphan drugs in Belgium: Estimates from 2008 to 2013
-
10.3111/13696998.2010.491427 20482245
-
Denis A, Mergaert L, Fostier C, Cleemput I, Simoens S. Budget impact analysis of orphan drugs in Belgium: estimates from 2008 to 2013. J Med Econ. 2010;13(2):295-301. doi: 10.3111/13696998.2010.491427.
-
(2010)
J Med Econ
, vol.13
, Issue.2
, pp. 295-301
-
-
Denis, A.1
Mergaert, L.2
Fostier, C.3
Cleemput, I.4
Simoens, S.5
-
55
-
-
80052784333
-
Critical assessment of Belgian reimbursement dossiers of orphan drugs
-
21905759
-
Denis A, Mergaert L, Fostier C, Cleemput I, Hulstaert F, Simoens S. Critical assessment of Belgian reimbursement dossiers of orphan drugs. Pharmacoeconomics. 2011;29(10):883-93.
-
(2011)
Pharmacoeconomics
, vol.29
, Issue.10
, pp. 883-893
-
-
Denis, A.1
Mergaert, L.2
Fostier, C.3
Cleemput, I.4
Hulstaert, F.5
Simoens, S.6
-
56
-
-
84904917381
-
An overview of the orphan medicines market in Turkey
-
Koçkaya G, Wertheimer AI, Kilic P, Tanyeri P, Mert Vural I, Akbulat A, et al. An overview of the orphan medicines market in Turkey. Value in Health Reg Issues. 2014;4(1):47-52.
-
(2014)
Value in Health Reg Issues
, vol.4
, Issue.1
, pp. 47-52
-
-
Koçkaya, G.1
Wertheimer, A.I.2
Kilic, P.3
Tanyeri, P.4
Mert Vural, I.5
Akbulat, A.6
-
57
-
-
84856487084
-
Access to orphan drugs in Europe: Current and future issues
-
10.1586/erp.11.95
-
Morgane M, Toumi M. Access to orphan drugs in Europe: current and future issues. Expert Rev Pharmacoecon Outcomes Res. 2012;12(1):23-9. doi: 10.1586/erp.11.95.
-
(2012)
Expert Rev Pharmacoecon Outcomes Res
, vol.12
, Issue.1
, pp. 23-29
-
-
Morgane, M.1
Toumi, M.2
-
58
-
-
77958187982
-
Review of regulatory recommendations for orphan drug submissions in the netherlands and Scotland: Focus on the underlying pharmacoeconomic evaluations
-
20974323
-
Vegter S, Rozenbaum MH, Postema R, Tolley K, Postma MJ. Review of regulatory recommendations for orphan drug submissions in the netherlands and scotland: focus on the underlying pharmacoeconomic evaluations. Clin Ther. 2010;32(9):1651-61.
-
(2010)
Clin Ther
, vol.32
, Issue.9
, pp. 1651-1661
-
-
Vegter, S.1
Rozenbaum, M.H.2
Postema, R.3
Tolley, K.4
Postma, M.J.5
-
59
-
-
78649700786
-
Rare diseases, orphan drugs and their regulation: Questions and misconceptions
-
1:CAS:528:DC%2BC3cXhtl2itbvO 21060315
-
Tambuyzer E. Rare diseases, orphan drugs and their regulation: Questions and misconceptions. Nat Rev Drug Discov. 2010;9(12):921-9.
-
(2010)
Nat Rev Drug Discov
, vol.9
, Issue.12
, pp. 921-929
-
-
Tambuyzer, E.1
-
60
-
-
34247504244
-
Assessing the economic challenges posed by orphan drugs
-
17234015
-
Drummond MF, Wilson DA, Kanavos P, Ubel P, Rovira J. Assessing the economic challenges posed by orphan drugs. Int J Technol Assess Health Care. 2007;23(1):36-42.
-
(2007)
Int J Technol Assess 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
-
61
-
-
84890346071
-
Eliciting preferences for prioritizing treatment of rare diseases: The role of opportunity costs and framing effects
-
24114738
-
Desser AS, Olsen JA, Grepperud S. Eliciting preferences for prioritizing treatment of rare diseases: the role of opportunity costs and framing effects. Pharmacoeconomics. 2013;31(11):1051-61.
-
(2013)
Pharmacoeconomics
, vol.31
, Issue.11
, pp. 1051-1061
-
-
Desser, A.S.1
Olsen, J.A.2
Grepperud, S.3
-
62
-
-
33645824158
-
Rationing of drugs for rare diseases
-
16605277
-
Hughes D. Rationing of drugs for rare diseases. Pharmacoeconomics. 2006;24(4):315-6.
-
(2006)
Pharmacoeconomics
, vol.24
, Issue.4
, pp. 315-316
-
-
Hughes, D.1
-
63
-
-
27644486590
-
Orphan drugs and the NHS: Should we value rarity?
-
McCabe C, Claxton K, Tsuchiya A. Orphan drugs and the NHS: Should we value rarity? Br Med J. 2005;331(7523):1016-9.
-
(2005)
Br Med J
, vol.331
, Issue.7523
, pp. 1016-1019
-
-
McCabe, C.1
Claxton, K.2
Tsuchiya, A.3
-
64
-
-
33646231273
-
Orphan drugs revisited: Author's response [4]
-
Hughes D. Orphan drugs revisited: Author's response [4]. QJM. 2006;99(5):350-1.
-
(2006)
QJM
, vol.99
, Issue.5
, pp. 350-351
-
-
Hughes, D.1
-
65
-
-
84894321192
-
Limits on use of health economic assessments for rare diseases
-
10.1093/qjmed/hcu016 1:STN:280:DC%2BC2czns1aksw%3D%3D 24453281
-
Hyry HI, Stern AD, Cox TM, Roos JC. Limits on use of health economic assessments for rare diseases. QJM. 2014;107(3):241-5. doi: 10.1093/qjmed/hcu016.
-
(2014)
QJM
, vol.107
, Issue.3
, pp. 241-245
-
-
Hyry, H.I.1
Stern, A.D.2
Cox, T.M.3
Roos, J.C.4
-
66
-
-
27644487456
-
Commissioning for rare diseases: View from the frontline
-
Burls A, Austin D, Moore D. Commissioning for rare diseases: view from the frontline. Br Med J. 2005;331(7523):1019-21.
-
(2005)
Br Med J
, vol.331
, Issue.7523
, pp. 1019-1021
-
-
Burls, A.1
Austin, D.2
Moore, D.3
-
67
-
-
33846968946
-
-
Nice Citizens Council Report - Ultra Orphan Drugs Accessed 5 Oct 2015
-
Nice Citizens Council Report - Ultra Orphan Drugs. National Institute for Clinical Excellence, London. 2004. https://www.nice.org.uk/Media/Default/Get-involved/Citizens-Council/Reports/CCReport04UltraOrphanDrugs.pdf. Accessed 5 Oct 2015.
-
(2004)
National Institute for Clinical Excellence, London
-
-
-
68
-
-
84881344116
-
Prioritizing treatment of rare diseases: A survey of preferences of Norwegian doctors
-
23931945
-
Desser AS. Prioritizing treatment of rare diseases: a survey of preferences of Norwegian doctors. Soc Sci Med. 2013;94:56-62.
-
(2013)
Soc Sci Med
, vol.94
, pp. 56-62
-
-
Desser, A.S.1
-
69
-
-
84925497333
-
Challenges in measuring the societal value of orphan drugs: Insights from a canadian stated preference survey
-
10.1007/s40271-014-0109-5 25586645
-
Dragojlovic N, Rizzardo S, Bansback N, Mitton C, Marra CA, Lynd LD. Challenges in measuring the societal value of orphan drugs: insights from a canadian stated preference survey. Patient. 2015;8(1):93-101. doi: 10.1007/s40271-014-0109-5.
-
(2015)
Patient
, vol.8
, Issue.1
, pp. 93-101
-
-
Dragojlovic, N.1
Rizzardo, S.2
Bansback, N.3
Mitton, C.4
Marra, C.A.5
Lynd, L.D.6
-
70
-
-
77957171034
-
Societal views on orphan drugs: Cross sectional survey of Norwegians aged 40 to 67
-
10.1136/bmj.c4715 20861122 2944922
-
Desser AS, Gyrd-Hansen D, Olsen JA, Grepperud S, Kristiansen IS. Societal views on orphan drugs: cross sectional survey of Norwegians aged 40 to 67. BMJ. 2010;341:c4715. doi: 10.1136/bmj.c4715.
-
(2010)
BMJ
, vol.341
, pp. c4715
-
-
Desser, A.S.1
Gyrd-Hansen, D.2
Olsen, J.A.3
Grepperud, S.4
Kristiansen, I.S.5
-
71
-
-
84856806914
-
Systematic review of comparative effectiveness data for oncology orphan drugs
-
22435748
-
Cheng MM, Ramsey SD, Devine EB, Garrison LP, Bresnahan BW, Veenstra DL. Systematic review of comparative effectiveness data for oncology orphan drugs. Am J Manag Care. 2012;18(1):47-62.
-
(2012)
Am J Manag Care
, vol.18
, Issue.1
, pp. 47-62
-
-
Cheng, M.M.1
Ramsey, S.D.2
Devine, E.B.3
Garrison, L.P.4
Bresnahan, B.W.5
Veenstra, D.L.6
-
72
-
-
33646240617
-
Orphan drugs revisited
-
1:STN:280:DC%2BD283jt1Kkug%3D%3D 16504983
-
McCabe C, Tsuchiya A, Claxton K, Raftery J. Orphan drugs revisited. QJM. 2006;99(5):341-5.
-
(2006)
QJM
, vol.99
, Issue.5
, pp. 341-345
-
-
McCabe, C.1
Tsuchiya, A.2
Claxton, K.3
Raftery, J.4
-
73
-
-
84971656249
-
-
Cost effectiveness and strategic planning (WHO-CHOICE) WHO Accessed 20.10.2015
-
Cost effectiveness and strategic planning (WHO-CHOICE) WHO. http://www.who.int/choice/costs/CER-levels/en/. Accessed 20.10.2015.
-
-
-
-
74
-
-
84956580438
-
HTA Implementation Roadmap in Central and Eastern European Countries
-
doi: 10.1002/hec.3298
-
Kaló Z, Gheorghe A, Huic M, Csanádi M, Kristensen FB. HTA Implementation Roadmap in Central and Eastern European Countries. Health Econ. 2016;25 Suppl 1:179-92. doi: 10.1002/hec.3298.
-
(2016)
Health Econ.
, vol.25
, pp. 179-192
-
-
Kaló, Z.1
Gheorghe, A.2
Huic, M.3
Csanádi, M.4
Kristensen, F.B.5
-
75
-
-
84856894934
-
Pharmacoeconomic analyses of treatments for rare disease
-
Fishman JC, Skrepnek GH. Pharmacoeconomic analyses of treatments for rare disease. Pharm Policy Law. 2012;14(1):51-67.
-
(2012)
Pharm Policy Law
, vol.14
, Issue.1
, pp. 51-67
-
-
Fishman, J.C.1
Skrepnek, G.H.2
-
76
-
-
32944471148
-
Orphan drugs and the NHS: Fairness in health care entails more than cost effectiveness
-
16282416 1283316
-
Sheehan M. Orphan drugs and the NHS: fairness in health care entails more than cost effectiveness. BMJ. 2005;331(7525):1144-5.
-
(2005)
BMJ
, vol.331
, Issue.7525
, pp. 1144-1145
-
-
Sheehan, M.1
-
77
-
-
84861951692
-
How should we model rare disease allocation decisions?
-
22616383
-
London AJ. How should we model rare disease allocation decisions? Hastings Cent Rep. 2012;42(1):3.
-
(2012)
Hastings Cent Rep
, vol.42
, Issue.1
, pp. 3
-
-
London, A.J.1
-
78
-
-
84960887396
-
Potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement process of orphan drugs
-
10.1186/s13023-016-0388-0 26965710 4787054
-
Kolasa K, Zwolinski KM, Kalo Z, Hermanowski T. Potential impact of the implementation of multiple-criteria decision analysis (MCDA) on the Polish pricing and reimbursement process of orphan drugs. Orphanet J Rare Dis. 2016;11:23. doi: 10.1186/s13023-016-0388-0.
-
(2016)
Orphanet J Rare Dis
, vol.11
, pp. 23
-
-
Kolasa, K.1
Zwolinski, K.M.2
Kalo, Z.3
Hermanowski, T.4
-
79
-
-
84925581911
-
Shift of focus in the financing of Hungarian drugs. Reimbursement for orphan drugs for treating rare diseases: Financing of enzyme replacement therapy in Hungary
-
10.1556/oh.2014.30031 25344850
-
Szegedi M, Molnar MJ, Boncz I, Kosztolanyi G. Shift of focus in the financing of Hungarian drugs. Reimbursement for orphan drugs for treating rare diseases: financing of enzyme replacement therapy in Hungary. Orv Hetil. 2014;155(44):1735-41. doi: 10.1556/oh.2014.30031.
-
(2014)
Orv Hetil
, vol.155
, Issue.44
, pp. 1735-1741
-
-
Szegedi, M.1
Molnar, M.J.2
Boncz, I.3
Kosztolanyi, G.4
-
80
-
-
84960899803
-
Challenges faced in transferring economic evaluations to middle income countries
-
doi: 10.1017/s0266462315000604
-
Drummond M, Augustovski F, Kalo Z, Yang BM, Pichon-Riviere A, Bae EY et al. Challenges faced in transferring economic evaluations to middle income countries. Int J Technol Assess Health Care. 2015;31(6):442-8. doi: 10.1017/s0266462315000604.
-
(2015)
Int J Technol Assess Health Care.
, vol.31
, Issue.6
, pp. 442-448
-
-
Drummond, M.1
Augustovski, F.2
Kalo, Z.3
Yang, B.M.4
Pichon-Riviere, A.5
Bae, E.Y.6
-
81
-
-
84971637171
-
Determinants of patient adherence to antihypertensive medication: A multi-national cross-sectional survey
-
Morrison V, Fargher E, Parveen S, Plumpton C. Determinants of patient adherence to antihypertensive medication: a multi-national cross-sectional survey. Ascertaining Barriers for Compliance: policies for safe, effective and cost-effective use of medicines in Europe - Final Report. 2012. p. 78-103.
-
(2012)
Ascertaining Barriers for Compliance: Policies for Safe, Effective and Cost-effective Use of Medicines in Europe - Final Report
, pp. 78-103
-
-
Morrison, V.1
Fargher, E.2
Parveen, S.3
Plumpton, C.4
-
82
-
-
84887913110
-
Differential pricing of new pharmaceuticals in lower income European countries
-
10.1586/14737167.2013.847367 24219049
-
Kalo Z, Annemans L, Garrison LP. Differential pricing of new pharmaceuticals in lower income European countries. Expert Rev Pharmacoecon Outcomes Res. 2013;13(6):735-41. doi: 10.1586/14737167.2013.847367.
-
(2013)
Expert Rev Pharmacoecon Outcomes Res
, vol.13
, Issue.6
, pp. 735-741
-
-
Kalo, Z.1
Annemans, L.2
Garrison, L.P.3
-
83
-
-
84879459872
-
Haemophilia care in Europe - A survey of 35 countries
-
10.1111/hae.12125 23557438
-
O'Mahony B, Noone D, Giangrande PL, Prihodova L. Haemophilia care in Europe - a survey of 35 countries. Haemophilia. 2013;19(4):e239-47. doi: 10.1111/hae.12125.
-
(2013)
Haemophilia
, vol.19
, Issue.4
, pp. e239-e247
-
-
O'Mahony, B.1
Noone, D.2
Giangrande, P.L.3
Prihodova, L.4
-
84
-
-
84971623632
-
-
EURORDIS. Improving patient access to orphan drugs in Europe EURORDIS. 2009 Accessed 29.04.2016.
-
EURORDIS. Improving patient access to orphan drugs in Europe EURORDIS. 2009. http://www.eurordis.org/content/improving-patient-access-orphan-drugs-europe. Accessed 29.04.2016.
-
-
-
-
85
-
-
84971637172
-
-
EUCERD. EUCERD Recommendation for a CAVOMP Information Flow. 2012 Accessed 20.04.2016
-
EUCERD. EUCERD Recommendation for a CAVOMP Information Flow. 2012. http://www.eurordis.org/sites/default/files/cavomp.pdf. Accessed 20.04.2016.
-
-
-
-
87
-
-
84942293513
-
Rare disease terminology and definitions-a systematic global review: Report of the ISPOR Rare Disease Special Interest Group
-
International Society for Pharmacoeconomics and Outcomes Research Rare Disease Special Interest Group 26409619
-
Richter T, Nestler-Parr S, Babela R, Khan ZM, Tesoro T, Molsen E, Hughes DA, International Society for Pharmacoeconomics and Outcomes Research Rare Disease Special Interest Group. Rare disease terminology and definitions-a systematic global review: report of the ISPOR Rare Disease Special Interest Group. Value Health. 2015;18(6):906-14.
-
(2015)
Value Health
, vol.18
, Issue.6
, pp. 906-914
-
-
Richter, T.1
Nestler-Parr, S.2
Babela, R.3
Khan, Z.M.4
Tesoro, T.5
Molsen, E.6
Hughes, D.A.7
-
88
-
-
75149138472
-
European network for health technology assessment, EUnetHTA: Planning, development, and implementation of a sustainable European network for health technology assessment
-
European network for Health Technology Assessment (EUnetHTA) 20030898
-
Kristensen FB, Mäkelä M, Neikter SA, Rehnqvist N, Håheim LL, Mørland B, Milne R, Nielsen CP, Busse R, Lee-Robin SH, Wild C, Espallargues M, Chamova J, European network for Health Technology Assessment (EUnetHTA). European network for health technology assessment, EUnetHTA: planning, development, and implementation of a sustainable European network for health technology assessment. Int J Technol Assess Health Care. 2009;25 Suppl 2:107-16.
-
(2009)
Int J Technol Assess Health Care
, vol.252
, pp. 107-116
-
-
Kristensen, F.B.1
Mäkelä, M.2
Neikter, S.A.3
Rehnqvist, N.4
Håheim, L.L.5
Mørland, B.6
Milne, R.7
Nielsen, C.P.8
Busse, R.9
Lee-Robin, S.H.10
Wild, C.11
Espallargues, M.12
Chamova, J.13
-
89
-
-
84855374189
-
Developing and paying for medicines for orphan indications in oncology: Utilitarian regulation vs equitable care
-
1:STN:280:DC%2BC387hs1eiuw%3D%3D 22215105 3251875
-
Davies JE, Neidle S, Taylor DG. Developing and paying for medicines for orphan indications in oncology: Utilitarian regulation vs equitable care. Br J Cancer. 2012;106(1):14-7.
-
(2012)
Br J Cancer
, vol.106
, Issue.1
, pp. 14-17
-
-
Davies, J.E.1
Neidle, S.2
Taylor, D.G.3
-
90
-
-
84864198277
-
Orphan drugs for rare diseases: Is it time to revisit their special market access status?
-
22747423
-
Simoens S, Cassiman D, Dooms M, Picavet E. Orphan drugs for rare diseases: Is it time to revisit their special market access status? Drugs. 2012;72(11):1437-43.
-
(2012)
Drugs
, vol.72
, Issue.11
, pp. 1437-1443
-
-
Simoens, S.1
Cassiman, D.2
Dooms, M.3
Picavet, E.4
-
91
-
-
84971654522
-
-
What type of evidence is currently being considered in the development of clinical practice guidelines for rare diseases? Accessed 2 October 2015
-
What type of evidence is currently being considered in the development of clinical practice guidelines for rare diseases? Institute for Quality and Efficiency in Health Care: Executive Summaries. 2011. http://www.ncbi.nlm.nih.gov/pubmedhealth/PMH0065182/pdf/PubMedHealth-PMH0065182.pdf. Accessed 2 October 2015.
-
(2011)
Institute for Quality and Efficiency in Health Care: Executive Summaries
-
-
-
92
-
-
79960136544
-
A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: An exploratory study
-
21205401
-
Mentzakis E, Stefanowska P, Hurley J. A discrete choice experiment investigating preferences for funding drugs used to treat orphan diseases: an exploratory study. Health Econ Policy Law. 2011;6(3):405-33.
-
(2011)
Health Econ Policy Law
, vol.6
, Issue.3
, pp. 405-433
-
-
Mentzakis, E.1
Stefanowska, P.2
Hurley, J.3
-
93
-
-
84861100238
-
Orphan drugs for rare diseases: Grounds for special status
-
1:CAS:528:DC%2BC38Xis1Oksb4%3D
-
Picavet E, Dooms M, Cassiman D, Simoens S. Orphan drugs for rare diseases: grounds for special status. Drug Dev Res. 2012;73(3):115-9.
-
(2012)
Drug Dev Res
, vol.73
, Issue.3
, pp. 115-119
-
-
Picavet, E.1
Dooms, M.2
Cassiman, D.3
Simoens, S.4
-
94
-
-
79954488011
-
European policies on orphan diseases and drugs stimulate drug development, but could be improved
-
doi: 10.2165/11601550-000000000-00000
-
European policies on orphan diseases and drugs stimulate drug development, but could be improved. Drugs Ther Perspect. 2011;27(5):24-6. doi: 10.2165/11601550-000000000-00000.
-
(2011)
Drugs Ther Perspect.
, vol.27
, Issue.5
, pp. 24-26
-
-
-
95
-
-
33645875903
-
Rare diseases and essential medicines: Global perspective
-
Hogerzeil HV. Rare diseases and essential medicines: Global perspective. Int J Pharm Med. 2005;19(5-6):285-8.
-
(2005)
Int J Pharm Med
, vol.19
, Issue.5-6
, pp. 285-288
-
-
Hogerzeil, H.V.1
-
96
-
-
84860431228
-
A fair share for the orphans: Ethical guidelines for a fair distribution of resources within the bounds of the 10-year-old European Orphan Drug Regulation
-
21947805
-
Pinxten W, Denier Y, Dooms M, Cassiman JJ, Dierickx K. A fair share for the orphans: ethical guidelines for a fair distribution of resources within the bounds of the 10-year-old European Orphan Drug Regulation. J Med Ethics. 2012;38(3):148-53.
-
(2012)
J Med Ethics
, vol.38
, Issue.3
, pp. 148-153
-
-
Pinxten, W.1
Denier, Y.2
Dooms, M.3
Cassiman, J.J.4
Dierickx, K.5
-
97
-
-
84907210916
-
Health disparities: Exploring the ethics of orphan drugs
-
24733129
-
Kinney J. Health disparities: exploring the ethics of orphan drugs. Am J Health Syst Pharm. 2014;71(9):692-3.
-
(2014)
Am J Health Syst Pharm
, vol.71
, Issue.9
, pp. 692-693
-
-
Kinney, J.1
-
98
-
-
84861928513
-
Which orphans will find a home? the rule of rescue in resource allocation for rare diseases
-
22616398
-
Largent EA, Pearson SD. Which orphans will find a home? The rule of rescue in resource allocation for rare diseases. Hastings Cent Rep. 2012;42(1):27-34.
-
(2012)
Hastings Cent Rep
, vol.42
, Issue.1
, pp. 27-34
-
-
Largent, E.A.1
Pearson, S.D.2
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