-
1
-
-
84873079257
-
Orphan drugs: The regulatory environment
-
Franco P. Orphan drugs: the regulatory environment. Drug Discov Today. 2013;18(3-4):163-72.
-
(2013)
Drug Discov Today
, vol.18
, Issue.3-4
, pp. 163-172
-
-
Franco, P.1
-
2
-
-
79960002064
-
Accelerating access to treatments for rare diseases
-
Dunoyer M. Accelerating access to treatments for rare diseases. Nat Rev Drug Discov. 2011;10(7):475-6.
-
(2011)
Nat Rev Drug Discov.
, vol.10
, Issue.7
, pp. 475-476
-
-
Dunoyer, M.1
-
3
-
-
44849143056
-
Why rare diseases are an important medical and social issue
-
Schieppati A, Henter JI, Daina E, Aperia A. Why rare diseases are an important medical and social issue. Lancet. 2008;371(9629):2039-41.
-
(2008)
Lancet
, vol.371
, Issue.9629
, pp. 2039-2041
-
-
Schieppati, A.1
Henter, J.I.2
Daina, E.3
Aperia, A.4
-
4
-
-
77449097555
-
Rare diseases and legislation in China
-
Wang JB, Guo JJ, Yang L, Zhang YD, Sun ZQ, Zhang YJ. Rare diseases and legislation in China. Lancet. 2010;375(9716):708-9.
-
(2010)
Lancet
, vol.375
, Issue.9716
, pp. 708-709
-
-
Wang, J.B.1
Guo, J.J.2
Yang, L.3
Zhang, Y.D.4
Sun, Z.Q.5
Zhang, Y.J.6
-
5
-
-
84876743099
-
Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU
-
Joppi R, Bertele V, Garattini S. Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU. Eur J Clin Pharmacol. 2013;69(4):1009-24.
-
(2013)
Eur J Clin Pharmacol.
, vol.69
, Issue.4
, pp. 1009-1024
-
-
Joppi, R.1
Bertele, V.2
Garattini, S.3
-
6
-
-
74049148749
-
Orphan products: An emerging trend in drug approvals
-
Cote T, Kelkar A, Xu K, Braun MM, Phillips MI. Orphan products: an emerging trend in drug approvals. Nat Rev Drug Discov. 2010;9(1):84.
-
(2010)
Nat Rev Drug Discov.
, vol.9
, Issue.1
, pp. 84
-
-
Cote, T.1
Kelkar, A.2
Xu, K.3
Braun, M.M.4
Phillips, M.I.5
-
8
-
-
84945919084
-
Current progress in the management of rare diseases and orphan drugs in China
-
Gong S, Jin S. Current progress in the management of rare diseases and orphan drugs in China. Intractable Rare Dis Res. 2012;1(2):45-52.
-
(2012)
Intractable Rare Dis Res.
, vol.1
, Issue.2
, pp. 45-52
-
-
Gong, S.1
Jin, S.2
-
10
-
-
58149522302
-
Medicine prices, availability, and affordability in 36 developing and middle-income countries: A secondary analysis
-
Cameron A, Ewen M, Ross-Degnan D, Ball D, Laing R. Medicine prices, availability, and affordability in 36 developing and middle-income countries: a secondary analysis. Lancet. 2009;373(9659):240-9.
-
(2009)
Lancet
, vol.373
, Issue.9659
, pp. 240-249
-
-
Cameron, A.1
Ewen, M.2
Ross-Degnan, D.3
Ball, D.4
Laing, R.5
-
11
-
-
77954736394
-
Prices, availability and affordability of essential medicines in rural areas of Hubei Province, China
-
Yang H, Dib HH, Zhu M, Qi G, Zhang X. Prices, availability and affordability of essential medicines in rural areas of Hubei Province, China. Health Policy Plan. 2010;25(3):219-29.
-
(2010)
Health Policy Plan
, vol.25
, Issue.3
, pp. 219-229
-
-
Yang, H.1
Dib, H.H.2
Zhu, M.3
Qi, G.4
Zhang, X.5
-
12
-
-
84925490315
-
Medicine prices, availability, and affordability in the Shaanxi Province in China: Implications for the future
-
Jiang M, Zhou Z, Wu L, et al. Medicine prices, availability, and affordability in the Shaanxi Province in China: implications for the future. Int J Clin Pharm. 2015;37(1):12-7.
-
(2015)
Int J Clin Pharm.
, vol.37
, Issue.1
, pp. 12-17
-
-
Jiang, M.1
Zhou, Z.2
Wu, L.3
-
13
-
-
85007490066
-
-
Accessed 17 December 2015
-
World Health Organization. Description of essential medicines. 2015. http://apps.who.int/medicinedocs/en/d/Js4875e/5.2.html. Accessed 17 December 2015.
-
(2015)
Description of Essential Medicines
-
-
-
14
-
-
84938384222
-
An appeal to the global health community for a tripartite innovation: An "Essential diagnostics list," "Health in all policies," and "See-through 21(st) century science and ethics"
-
Dove ES, Barlas IO, Birch K, et al. An Appeal to the Global Health Community for a Tripartite Innovation: An "Essential Diagnostics List," "Health in All Policies," and "See-Through 21(st) Century Science and Ethics". Omics. 2015;19(8):435-42.
-
(2015)
Omics
, vol.19
, Issue.8
, pp. 435-442
-
-
Dove, E.S.1
Barlas, I.O.2
Birch, K.3
-
15
-
-
78650240029
-
Availability of and access to orphan drugs: An international comparison of pharmaceutical treatments for pulmonary arterial hypertension, fabry disease, hereditary angioedema and chronic myeloid leukaemia
-
Blankart CR, Stargardt T, Schreyogg J. Availability of and access to orphan drugs: an international comparison of pharmaceutical treatments for pulmonary arterial hypertension, Fabry disease, hereditary angioedema and chronic myeloid leukaemia. Pharmacoeconomics. 2011;29(1):63-82.
-
(2011)
Pharmacoeconomics
, vol.29
, Issue.1
, pp. 63-82
-
-
Blankart, C.R.1
Stargardt, T.2
Schreyogg, J.3
-
16
-
-
84872660761
-
-
Accessed 31 July 2013
-
U.S. Foo. and Drug Administration. Search Orphan Drug Designations and Approvals. 2013. http//www.accessdata.fda.gov/scripts/opdlisting/oopd/index.cfm. Accessed 31 July 2013
-
(2013)
Search Orphan Drug Designations and Approvals
-
-
-
17
-
-
0008348082
-
-
Accessed 17 July 2013
-
European Medicines Agency. European public assessment reports: Orphan medicines. 2013. http://www.ema.europa.eu/ema/index.jsp?curl=pages%2Fmedicines%2Flanding%2Fepar-search.jsp&mid=WC0b01ac058001d125&searchTab=searchByAuthType&alreadyLoaded=true&isNewQuery=true&status=Authorised&status=Withdrawn&status=Suspended&status=Refused&keyword=Enter+keywords&searchType=name&taxonomyPath=&treeNumber=&searchGenericType=orphan. Accessed 17 July 2013
-
(2013)
European Public Assessment Reports: Orphan Medicines
-
-
-
18
-
-
85007423773
-
-
Accessed 27 March 2014
-
Ministry of Health, Labour and Welfare of Japan. List of products designated as orphan drugs for diseases 2014. 2014. http://www.mhlw.go.jp/english/policy/health-medical/pharmaceuticals/orphan-drug.html. Accessed 27 March 2014.
-
(2014)
List of Products Designated as Orphan Drugs for Diseases 2014
-
-
-
19
-
-
85007426431
-
-
Accessed 28 December 2013
-
Food and Drug Administration of China. Data Search. 2013. http://app1.sfda.gov.cn/datasearch/face3/dir.html. Accessed 28 December 2013.
-
(2013)
Data Search
-
-
-
20
-
-
84879306396
-
-
Accessed 2 May 2013
-
World Health Organization. ATC/DDD Index 2013. 2013. http://www.whoccno/atc-ddd-index/ Accessed 2 May 2013.
-
(2013)
ATC/DDD Index 2013
-
-
-
21
-
-
77954243603
-
Emergence of orphan drugs in the United States: A quantitative assessment of the first 25 years
-
Braun MM, Farag-El-Massah S, Xu K, Cote TR. Emergence of orphan drugs in the United States: a quantitative assessment of the first 25 years. Nat Rev Drug Discov. 2010;9(7):519-22.
-
(2010)
Nat Rev Drug Discov.
, vol.9
, Issue.7
, pp. 519-522
-
-
Braun, M.M.1
Farag-El-Massah, S.2
Xu, K.3
Cote, T.R.4
-
22
-
-
84880643025
-
-
Accessed 2 December 2013
-
U.S. Foo. and Drug Administration. What Are Generic Drugs? 2009. http://www.fda.gov/Drugs/ResourcesForYou/Consumers/BuyingUsingMedicineSafely/UnderstandingGenericDrugs/ucm144456.htm. Accessed 2 December 2013
-
(2009)
What are Generic Drugs?
-
-
-
24
-
-
84872896308
-
-
Accessed 30 May 2013
-
National Bureau of Statistics of China. Income of Urban and Rural Residents in 2011. 2012. http://www.stats.gov.cn/english/NewsEvents/201201/t20120130-26566.html. Accessed 30 May 2013.
-
(2012)
Income of Urban and Rural Residents in 2011
-
-
-
25
-
-
85007491932
-
-
Accessed 30 November 2013
-
Ministry of Human Resources and Social Security of the People's Republic of China. Drug lists of National Basic Medical Insurance. 2009. http://www.gov.cn/gzdt/2009-11/30/content-1476934.htm. Accessed 30 November 2013
-
(2009)
Drug Lists of National Basic Medical Insurance
-
-
-
26
-
-
84859326107
-
Rare diseases and orphan drugs
-
Melnikova I. Rare diseases and orphan drugs. Nat Rev Drug Discov. 2012; 11(4):267-8.
-
(2012)
Nat Rev Drug Discov.
, vol.11
, Issue.4
, pp. 267-268
-
-
Melnikova, I.1
-
27
-
-
78751571157
-
Emerging issues in public health: A perspective on China's healthcare system
-
Ling RE, Liu F, Lu XQ, Wang W. Emerging issues in public health: a perspective on China's healthcare system. Public Health. 2011;125(1):9-14.
-
(2011)
Public Health
, vol.125
, Issue.1
, pp. 9-14
-
-
Ling, R.E.1
Liu, F.2
Lu, X.Q.3
Wang, W.4
-
28
-
-
84891930582
-
The ecology of medical care in Beijing
-
Shao S, Zhao F, Wang J, et al. The ecology of medical care in Beijing. PLoS ONE. 2013;8(12):e82446.
-
(2013)
PLoS ONE
, vol.8
, Issue.12
-
-
Shao, S.1
Zhao, F.2
Wang, J.3
-
29
-
-
84992421885
-
Management strategy for raising the accessibility to orphan drugs in China
-
Gong SW, Zhang L, Jin S, Li LL. Management strategy for raising the accessibility to orphan drugs in China. Chin J Hospital Admin. 2010;26(2):126-30.
-
(2010)
Chin J Hospital Admin.
, vol.26
, Issue.2
, pp. 126-130
-
-
Gong, S.W.1
Zhang, L.2
Jin, S.3
Li, L.L.4
-
30
-
-
77954244068
-
China: Public health genomics
-
Zheng S, Song M, Wu L, et al. China: public health genomics. Public Health Genom. 2010;13(5):269-75.
-
(2010)
Public Health Genom.
, vol.13
, Issue.5
, pp. 269-275
-
-
Zheng, S.1
Song, M.2
Wu, L.3
-
31
-
-
84986133005
-
Orphan drugs: Pricing, reimbursement and patient access
-
Barak A, Shankar NJ. Orphan drugs: pricing, reimbursement and patient access. Int J Pharmaceut Healthcare Market. 2011;5(4):299-317
-
(2011)
Int J Pharmaceut Healthcare Market
, vol.5
, Issue.4
, pp. 299-317
-
-
Barak, A.1
Shankar, N.J.2
-
32
-
-
84856487084
-
Access to orphan drugs in Europe: Current and future issues
-
Michel M, Toumi M. Access to orphan drugs in Europe: current and future issues. Expert Rev Pharmacoecon Outcomes Res. 2012;12(1):23-9.
-
(2012)
Expert Rev Pharmacoecon Outcomes Res.
, vol.12
, Issue.1
, pp. 23-29
-
-
Michel, M.1
Toumi, M.2
-
35
-
-
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?
-
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.
-
(2012)
Orphanet J Rare Dis.
, vol.7
, pp. 74
-
-
Hughes-Wilson, W.1
Palma, A.2
Schuurman, A.3
Simoens, S.4
-
36
-
-
27744461277
-
Drugs for exceptionally rare diseases: Do they deserve special status for funding?
-
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
-
37
-
-
84873476022
-
Cost-effectiveness assessment of orphan drugs: A scientific and political conundrum
-
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
-
38
-
-
85007490051
-
Orphan drugs Part 1 - Patient care to the individual level
-
Pierce D. Orphan Drugs Part 1 - Patient Care to the Individual Level. Oncol Issues. 2010;25(6):44.
-
(2010)
Oncol Issues
, vol.25
, Issue.6
, pp. 44
-
-
Pierce, D.1
-
39
-
-
85007423741
-
-
Accessed 17 July 2014
-
The LPL Deficiency Association. Patient Assistance Programs. 2014. http://lplda.org/lpld-patients-and-caregivers/resources/patient-assistance/. Accessed 17 July 2014
-
(2014)
Patient Assistance Programs
-
-
-
40
-
-
84940645721
-
-
Accessed 19 July 2014
-
Food and Drug Administration of China. 2013 China Drug Review Annual Report. 2014. http://www.cde.org.cn/news.do?method=viewInfoCommon&id=313280. Accessed 19 July 2014
-
(2014)
2013 China Drug Review Annual Report
-
-
|