-
1
-
-
85070103320
-
-
cited, 13, Jul, Available from
-
European Medicines Agency (EMA). Orphan drugs and rare diseases at a glance. [cited 13 Jul 2016]. Available from: http://www.ema.europa.eu/docs/en_GB/document_library/Other/2010/01/WC500069805.pdf
-
(2016)
Orphan drugs and rare diseases at a glance
-
-
-
2
-
-
85080059276
-
-
cited, 14, Jul, Available from
-
NICE. Citizens council report ultra orphan drugs. [cited 14 Jul 2016]. Available from: http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1885144/pdf/bcp0062-0264.pdf
-
(2016)
Citizens council report ultra orphan drugs
-
-
-
3
-
-
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?
-
Available from
-
Hughes W, Palma A, Schuurman A, et al. 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? Orpanet J Rare Dis. 2012;7:1. Available from: http://www.ojrd.com/content/7/1/74
-
(2012)
Orpanet J Rare Dis
, vol.7
, pp. 1
-
-
Hughes, W.1
Palma, A.2
Schuurman, A.3
-
4
-
-
79958849417
-
Pricing and reimbursement of orphan drugs: the need for more transparency
-
Simoens S. Pricing and reimbursement of orphan drugs: the need for more transparency. Orphanet J Rare Dis. 2011;6:42.
-
(2011)
Orphanet J Rare Dis
, vol.6
, pp. 42
-
-
Simoens, S.1
-
6
-
-
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. Eur J Hosp Pharm Pract. 2010;15(4):25–13.
-
(2010)
Eur J Hosp Pharm Pract
, vol.15
, Issue.4
, pp. 25-13
-
-
Heemstra, H.E.1
-
7
-
-
77956169455
-
A comparative study of European rare disease and orphan drug markets
-
Denis A, Mergaert L, Fostier C, et al. A comparative study of European rare disease and orphan drug markets. Health Policy. 2010;97:173–179.
-
(2010)
Health Policy
, vol.97
, pp. 173-179
-
-
Denis, A.1
Mergaert, L.2
Fostier, C.3
-
8
-
-
84949032870
-
Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries
-
Gammie T, Lu CY, Babar ZU-D, et al. Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries. Plos ONE. 2015;10(10):e0140002.
-
(2015)
Plos ONE
, vol.10
, Issue.10
-
-
Gammie, T.1
Lu, C.Y.2
Babar, Z.U.-D.3
-
11
-
-
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: Int J Public Health. 2014;1(3):86–97.
-
(2014)
Rare Dis Orphan Drugs: Int J Public Health
, vol.1
, Issue.3
, pp. 86-97
-
-
Tordrup, D.1
Tzouma, V.2
Kanavos, P.3
-
12
-
-
84962091282
-
G-BA benefit assessment of new orphan drugs in Germany: the first five years
-
Bouslouk M. G-BA benefit assessment of new orphan drugs in Germany: the first five years. Expert Opinion Orphan Drugs. 2016;4(5):453–455. DOI:10.1517/21678707.2016.1166950
-
(2016)
Expert Opinion Orphan Drugs
, vol.4
, Issue.5
, pp. 453-455
-
-
Bouslouk, M.1
-
13
-
-
77956180950
-
Access mechanisms for orphan drugs: A comparative study of selected European countries
-
Available from
-
Garau M, Mestre-Ferrandiz J. Access mechanisms for orphan drugs: A comparative study of selected European countries. OHE Breifing. 2009;52:1–32. Available from: https://www.ohe.org/publications/access-mechanisms-orphan-drugs-comparative-study-selected-european-countries.
-
(2009)
OHE Breifing
, vol.1-32
-
-
Garau, M.1
Mestre-Ferrandiz, J.2
-
14
-
-
85019319506
-
-
Jun, Available from
-
Festøy H, Ognøy A WHO PPRI pharma profile Norway. Jun 2015. Available from: https://legemiddelverket.no/Documents/English/Price%20and%20reimbursement/PPRI_Pharma_Profile_Norway_20150626_final.pdf
-
(2015)
WHO PPRI pharma profile Norway
-
-
Festøy, H.1
Ognøy, A.2
-
15
-
-
84948399431
-
-
Available from
-
NICE. Interim process and methods of the highly specialised technologies programme. 2013. Available from: https://www.nice.org.uk/Media/Default/About/what-we-do/NICE-guidance/NICE-highly-specialised-technologies-guidance/Highly-Specialised-Technologies-Interim-methods-and-process-statements.pdf
-
(2013)
Interim process and methods of the highly specialised technologies programme
-
-
-
17
-
-
84902168946
-
Orphan drug policies: a suitable case for treatment
-
Drummond M, Towse A. Orphan drug policies: a suitable case for treatment. Eur J Health Econ. 2014;15:335–340.
-
(2014)
Eur J Health Econ
, vol.15
, pp. 335-340
-
-
Drummond, M.1
Towse, A.2
-
19
-
-
84929324394
-
Principles for consistent value assessment and sustainable funding of orphan drugs in Europe
-
Gutierrez L, Patris J, Hutchings A, et al. Principles for consistent value assessment and sustainable funding of orphan drugs in Europe. Orphanet J Rare Dis. 2015;10:53.
-
(2015)
Orphanet J Rare Dis
, vol.10
, pp. 53
-
-
Gutierrez, L.1
Patris, J.2
Hutchings, A.3
-
20
-
-
80053379101
-
Estimating the budget impact of orphan medicines in Europe: 2010-2020
-
Schey C, Milanova T, Hutchings A. Estimating the budget impact of orphan medicines in Europe: 2010-2020. Orphanet J Rare Dis. 2011;6:62.
-
(2011)
Orphanet J Rare Dis
, vol.6
, pp. 62
-
-
Schey, C.1
Milanova, T.2
Hutchings, A.3
-
21
-
-
84871184065
-
What is wrong with orphan drug policies?
-
Côté A, Keating B. What is wrong with orphan drug policies? Value Health. 2012;15:1185–1191.
-
(2012)
Value Health
, vol.15
, pp. 1185-1191
-
-
Côté, A.1
Keating, B.2
-
22
-
-
84864198277
-
Orphan drugs for rare diseases: is it time to revisit their special market access status?
-
Simoens S, Cassiman D, Dooms M, et al. Orphan drugs for rare diseases: is it time to revisit their special market access status? Drugs. 2012;72(11):1437–1443.
-
(2012)
Drugs
, vol.72
, Issue.11
, pp. 1437-1443
-
-
Simoens, S.1
Cassiman, D.2
Dooms, M.3
-
23
-
-
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):23–29.
-
(2012)
Expert Rev Pharmacoecon Outcomes Res
, vol.12
, Issue.1
, pp. 23-29
-
-
Michel, M.1
Toumi, M.2
-
24
-
-
79959460572
-
Drugs for rare diseases: influenceof orphan designation status on price
-
Picavet E, Dooms M, Cassiman D, et al. Drugs for rare diseases: influenceof orphan designation status on price. Appl Health Econ Health Policy. 2011;9(4):275–279.
-
(2011)
Appl Health Econ Health Policy
, vol.9
, Issue.4
, pp. 275-279
-
-
Picavet, E.1
Dooms, M.2
Cassiman, D.3
-
25
-
-
84900800675
-
Shining a light in the black box of orphan drug pricing
-
Picavet, Picavet E, Morel T, et al. Shining a light in the black box of orphan drug pricing. Orphanet J Rare Dis. 2014;9:62.
-
(2014)
Orphanet J Rare Dis
, vol.9
, pp. 62
-
-
Picavet1
Picavet, E.2
Morel, T.3
-
26
-
-
85165692816
-
-
cited, Jun, Available from
-
IHS PharmOnline International (POLI). [cited Jun 2016]. Available from: https://www.ihs.com/products/pharmaonline-international.html
-
(2016)
-
-
-
27
-
-
85165698418
-
-
cited, Jul, Available from
-
Ameli. [cited Jul 2016]. Available from: http://www.codage.ext.cnamts.fr/codif/bdm_it/index.php?p_site=AMELI
-
(2016)
-
-
-
28
-
-
85165655274
-
-
cited, Jul, Available from
-
The British National Formulary (BNF). [cited Jul 2016]. Available from: https://www.medicinescomplete.com/mc/login.htm
-
(2016)
-
-
-
29
-
-
85165645199
-
-
cited, Jul, Available from
-
Farmadati. [cited Jul 2016]. Available from: http://www.farmadati.it/
-
(2016)
-
-
-
30
-
-
84962146345
-
-
cited, Jul, Available from
-
WHO. Child Growth Standards: weight for Age. [cited Jul 2016]. Available from: http://www.who.int/childgrowth/standards/weight_for_age/en/
-
(2016)
Child Growth Standards: weight for Age
-
-
-
31
-
-
85165704863
-
-
Available from
-
Alcimed. Study on orphan drugs. Paris. 2005. Available from: http://ec.europa.eu/health/files/orphanmp/doc/pricestudy/final_final_report_part_1_web_en.pdf
-
(2005)
Study on orphan drugs. Paris
-
-
-
33
-
-
84937231816
-
Effectiveness, safety and costs of orphan drugs: an evidence-based review
-
Onakpoya I, Spencer EA, Thompson MJ, et al. Effectiveness, safety and costs of orphan drugs: an evidence-based review. BMJ Open. 2015;5:e007199.
-
(2015)
BMJ Open
, vol.5
, pp. e007199
-
-
Onakpoya, I.1
Spencer, E.A.2
Thompson, M.J.3
-
34
-
-
84987618854
-
Scientific and social value judgments for orphan drugs in health technology assessment
-
Nicod E, Kanavos P. Scientific and social value judgments for orphan drugs in health technology assessment. Int J Technol Assess Health Care. 2016;32(4):1–15.
-
(2016)
Int J Technol Assess Health Care
, vol.32
, Issue.4
, pp. 1-15
-
-
Nicod, E.1
Kanavos, P.2
-
35
-
-
80055038797
-
-
Available from
-
European Parliament Directorate-General for Internal Policies. Differences in costs of and access to pharmaceutical products in the EU. 2011. Available from: http://www.europarl.europa.eu/RegData/etudes/etudes/join/2011/451481/IPOL-ENVI_ET(2011)451481_EN.pdf.
-
(2011)
Differences in costs of and access to pharmaceutical products in the EU
-
-
-
36
-
-
84983442269
-
-
Dec,. Luxembourg: Publications Office of the European Union.,. Available from
-
European Commission. Study on enhanced cross-country coordination in the area of pharmaceutical product pricing. Luxembourg: Publications Office of the European Union. Dec, 2015. Available from: http://ec.europa.eu/health/sites/health/files/systems_performance_assessment/docs/pharmaproductpricing_frep_en.pdf.
-
(2015)
Study on enhanced cross-country coordination in the area of pharmaceutical product pricing
-
-
-
37
-
-
84890312186
-
A pilot study of multicriteria decision analysis for valuing orphan medicines
-
Sussex J, Rollet P, Garau M, et al. A pilot study of multicriteria decision analysis for valuing orphan medicines. Value Health. 2013;16:1163–1169.
-
(2013)
Value Health
, vol.16
, pp. 1163-1169
-
-
Sussex, J.1
Rollet, P.2
Garau, M.3
-
38
-
-
84925513451
-
Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework
-
Paulden M, Stafinski T, Menon D, et al. Value-based reimbursement decisions for orphan drugs: a scoping review and decision framework. Pharmacoeconomics. 2015;33:255–269.
-
(2015)
Pharmacoeconomics
, vol.33
, pp. 255-269
-
-
Paulden, M.1
Stafinski, T.2
Menon, D.3
-
39
-
-
84959106689
-
Can the EVIDEM framework tackle issues raised by evaluating treatments for rare diseases: analysis of issues and policies, and context-specific adaptation
-
Wagner M, Khoury H, Willet J, et al. Can the EVIDEM framework tackle issues raised by evaluating treatments for rare diseases: analysis of issues and policies, and context-specific adaptation. Pharmacoeconomics. 2016;34:285–301.
-
(2016)
Pharmacoeconomics
, vol.34
, pp. 285-301
-
-
Wagner, M.1
Khoury, H.2
Willet, J.3
-
40
-
-
85165653486
-
Value assessment and pricing frameworks for rare disease treatments: new approaches from the literature
-
Nov, Milan, Italy, Available from
-
Palaska C, Hutchings A. Value assessment and pricing frameworks for rare disease treatments: new approaches from the literature. ISPOR 18th Annual European Congress; Nov 2015; Milan, Italy. Available from: http://www.ispor.org/research_pdfs/51/pdffiles/PSY113.pdf
-
(2015)
ISPOR 18th Annual European Congress
-
-
Palaska, C.1
Hutchings, A.2
-
41
-
-
84887541924
-
Funding innovation for treatment for rare diseases: adopting a cost-based yardstick approach
-
Fellows GK, Hollis A. Funding innovation for treatment for rare diseases: adopting a cost-based yardstick approach. Orphanet J Rare Dis. 2013;8:18044.
-
(2013)
Orphanet J Rare Dis
, vol.8
, pp. 18044
-
-
Fellows, G.K.1
Hollis, A.2
-
42
-
-
85165713321
-
-
cited, Oct, Available from
-
US Forex Foreign Exchange Services. [cited Oct 2016]. Available from: http://www.usforex.com/forex-tools/historical-rate-tools/yearly-average-rates
-
(2016)
-
-
|