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Refinado por: assunto: Rare Diseases remover
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1
Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries
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Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries

Gammie, Todd ; Lu, Christine Y ; Babar, Zaheer Ud-Din Garattini, Silvio

PloS one, 2015-10, Vol.10 (10), p.e0140002-e0140002 [Periódico revisado por pares]

United States: Public Library of Science

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2
Socio-economic burden of rare diseases: A systematic review of cost of illness evidence
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Socio-economic burden of rare diseases: A systematic review of cost of illness evidence

Angelis, Aris ; Tordrup, David ; Kanavos, Panos

Health policy (Amsterdam), 2015-07, Vol.119 (7), p.964-979 [Periódico revisado por pares]

Ireland: Elsevier Ireland Ltd

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3
Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework
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Artigo
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Value-Based Reimbursement Decisions for Orphan Drugs: A Scoping Review and Decision Framework

Paulden, Mike ; Stafinski, Tania ; Menon, Devidas ; McCabe, Christopher

PharmacoEconomics, 2015-03, Vol.33 (3), p.255-269 [Periódico revisado por pares]

Cham: Springer International Publishing

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4
Rare diseases and effective treatments: are we delivering?
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Artigo
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Rare diseases and effective treatments: are we delivering?

Luzzatto, Lucio ; Hollak, Carla E M ; Cox, Timothy M ; Schieppati, Arrigo ; Licht, Christoph ; Kääriäinen, Helena ; Merlini, Giampaolo ; Schaefer, Franz ; Simoens, Steven ; Pani, Luca ; Garattini, Silvio ; Remuzzi, Giuseppe

The Lancet (British edition), 2015-02, Vol.385 (9970), p.750-752 [Periódico revisado por pares]

England: Elsevier Ltd

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5
Estimating the budget impact of orphan medicines in Europe: 2010 - 2020
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Artigo
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Estimating the budget impact of orphan medicines in Europe: 2010 - 2020

Schey, Carina ; Milanova, Tsveta ; Hutchings, Adam

Orphanet journal of rare diseases, 2011-09, Vol.6 (1), p.62-62, Article 62 [Periódico revisado por pares]

England: BioMed Central Ltd

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6
Estimating the clinical cost of drug development for orphan versus non-orphan drugs
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Artigo
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Estimating the clinical cost of drug development for orphan versus non-orphan drugs

Jayasundara, Kavisha ; Hollis, Aidan ; Krahn, Murray ; Mamdani, Muhammad ; Hoch, Jeffrey S ; Grootendorst, Paul

Orphanet journal of rare diseases, 2019-01, Vol.14 (1), p.12-12, Article 12 [Periódico revisado por pares]

England: BioMed Central Ltd

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7
Societal Preferences for Funding Orphan Drugs in the United Kingdom: An Application of Person Trade-Off and Discrete Choice Experiment Methods
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Societal Preferences for Funding Orphan Drugs in the United Kingdom: An Application of Person Trade-Off and Discrete Choice Experiment Methods

Bourke, Siobhan M. ; Plumpton, Catrin O. ; Hughes, Dyfrig A.

Value in health, 2018-05, Vol.21 (5), p.538-546 [Periódico revisado por pares]

United States: Elsevier Inc

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8
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?
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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, Wills ; Palma, Ana ; Schuurman, Ad ; Simoens, Steven

Orphanet journal of rare diseases, 2012-09, Vol.7 (1), p.74-74 [Periódico revisado por pares]

England: BioMed Central

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9
Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients' health needs?
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Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients' health needs?

Rodriguez-Monguio, R ; Spargo, T ; Seoane-Vazquez, E

Orphanet journal of rare diseases, 2017-01, Vol.12 (1), p.1-1, Article 1 [Periódico revisado por pares]

England: BioMed Central

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10
Do investors value the FDA orphan drug designation?
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Do investors value the FDA orphan drug designation?

Miller, Kathleen L

Orphanet journal of rare diseases, 2017-06, Vol.12 (1), p.114-114, Article 114 [Periódico revisado por pares]

England: BioMed Central Ltd

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