Works matching DE "ORPHAN drug laws"

Results: 93

Why do health technology assessment coverage recommendations for the same drugs differ across settings? Applying a mixed methods framework to systematically compare orphan drug decisions in four European countries.

Published in:

2017

By:

Nicod, Elena

Publication type:

journal article

Orphan drugs policies: a suitable case for treatment.

Published in:

2014

By:

Drummond, Michael;
Towse, Adrian

Publication type:

Editorial

The implementation of health technology assessment principles in public decisions concerning orphan drugs.

Published in:

European Journal of Clinical Pharmacology, 2020, v. 76, n. 6, p. 755, doi. 10.1007/s00228-020-02855-7

By:

Brenna, Elenka;
Polistena, Barbara;
Spandonaro, Federico

Publication type:

Article

Orphan drugs, orphan diseases. The first decade of orphan drug legislation in the EU.

Published in:

European Journal of Clinical Pharmacology, 2013, v. 69, n. 4, p. 1009, doi. 10.1007/s00228-012-1423-2

By:

Joppi, Roberta;
Bertele', Vittorio;
Garattini, Silvio

Publication type:

Article

Time to revisit the orphan drug law.

Published in:

2012

By:

Garattini, Silvio

Publication type:

Editorial

The impact of orphan drug policies in treating rare diseases.

Published in:

Health Information & Libraries Journal, 2019, v. 36, n. 2, p. 179, doi. 10.1111/hir.12256

By:

Weerasooriya, Shalini U.

Publication type:

Article

ARE WE ADOPTING THE ORPHANS, OR CREATING THEM? MEDICAL ETHICS AND LEGAL JURISPRUDENTIAL GUIDANCE FOR PROPOSED CHANGES TO THE ORPHAN DRUG ACT.

Published in:

Washington University Jurisprudence Review, 2017, v. 9, n. 2, p. 295

By:

RAW, LYDIA

Publication type:

Article

Regulatory Framework for Drug Development in Rare Diseases.

Published in:

Journal of Clinical Pharmacology, 2022, v. 62, p. S15, doi. 10.1002/jcph.2171

By:

Korth‐Bradley, Joan M.

Publication type:

Article

Investigating the landscape of US orphan product approvals.

Published in:

2018

By:

Miller, Kathleen L.;
Lanthier, Michael

Publication type:

journal article

Adopting Solutions to Orphan Drug Shortages.

Published in:

U.C. Davis Law Review, 2015, v. 48, n. 5, p. 2077

By:

Helen Liu

Publication type:

Article

Capture and passive predation in times of COVID-19 pandemic.

Published in:

Public Choice, 2022, v. 193, n. 3/4, p. 163, doi. 10.1007/s11127-022-01005-0

By:

Guennif, Samira

Publication type:

Article

Rare opportunities appear on the horizon to treat rare diseases.

Published in:

Nature Medicine, 2010, v. 16, n. 3, p. 241, doi. 10.1038/nm0310-241

By:

Torres, Christian

Publication type:

Article

Rarely mentioned: how we arrived at the quantitative definition of a rare disease.

Published in:

Baylor University Medical Center Proceedings, 2022, v. 35, n. 4, p. 498, doi. 10.1080/08998280.2022.2048613

By:

Partin, Clyde

Publication type:

Article

Policymaking for Orphan Drugs and Its Challenges.

Published in:

AMA Journal of Ethics, 2015, v. 17, n. 8, p. 776, doi. 10.1001/journalofethics.2015.17.8.pfor2-1508

By:

Rhee, Taeho Greg

Publication type:

Article

Orphan drug incentives in the pharmacogenomic context: policy responses in the US and Canada.

Published in:

Journal of Law & the Biosciences, 2015, v. 2, n. 2, p. 263, doi. 10.1093/jlb/lsv013

By:

Gibson, Shannon;
von Tigerstrom, Barbara

Publication type:

Article

RECAPTURING THE ORPHAN DRUG ACT: AN ANALYSIS OF PROPOSALS.

Published in:

BYU Journal of Public Law, 2023, v. 37, n. 1, p. 165

By:

Trilokekar, Rajdeep

Publication type:

Article

The use of 2D fingerprint methods to support the assessment of structural similarity in orphan drug legislation.

Published in:

Journal of Cheminformatics, 2014, v. 6, n. 1, p. 2, doi. 10.1186/1758-2946-6-5

By:

Franco, Pedro;
Porta, Nuria;
Holliday, John D.;
Willett, Peter

Publication type:

Article

A Matter of Interpretation: How the Orphan Drug Litigation Tests the Limits of 340B Program Guidance.

Published in:

Journal of Health Care Compliance, 2014, v. 16, n. 5, p. 5

By:

Jackson, Travis

Publication type:

Article

Addressing the authorization of orphan drugs will not fix reimbursement problems: The elephant is still in the room.

Published in:

Canadian Pharmacists Journal, 2013, v. 146, n. 5, p. 245, doi. 10.1177/1715163513499127

By:

Embrett, Mark

Publication type:

Article

Pharma exploits orphan drug rules.

Published in:

Chemistry & Industry, 2010, n. 23, p. 6

By:

Dorey, Emma

Publication type:

Article

What Is the Purpose of the Orphan Drug Act?

Published in:

2017

By:

Herder, Matthew

Publication type:

journal article

Gaucher disease in Mexico. Epidemiologic overview.

Published in:

Acta Pediatrica de Mexico, 2011, v. 32, n. 5, p. 277

By:

Carbajal-Rodriguez, Luis;
Voirol-Garcia, Aitana;
Mora-Magaña, Ignacio;
Rodriguez-Herrera, Raymundo;
Zarco-Roman, Jorge

Publication type:

Article

The EU as a Political Determinant of Global Health: The Case of Research and Development Incentives for Orphan Medicines and Biotechnology.

Published in:

Journal of Health Politics, Policy & Law, 2024, v. 49, n. 5, p. 911, doi. 10.1215/03616878-11257032

By:

Perehudoff, Katrina;
Ippel, Kaja Anastazja

Publication type:

Article

NORD. A beacon of hope for people with rare disorders.

Published in:

Rare Tumors, 2010, v. 2, n. 1, p. 39, doi. 10.4081/rt.2010.e13

By:

Putkowski, Stefanie

Publication type:

Article

The Pediatric Research Equity Act Moves Into Adolescence.

Published in:

2017

By:

Bourgeois, Florence T.;
Hwang, Thomas J.

Publication type:

journal article

Research Into Rare Diseases of Childhood.

Published in:

2014

By:

Batshaw, Mark L.;
Groft, Stephen C.;
Krischer, Jeffrey P.

Publication type:

Opinion

Orphan Drug Pricing and Payer Management in the United States: Are We Approaching the Tipping Point?

Published in:

American Health & Drug Benefits, 2010, v. 3, n. 1, p. 15

By:

Hyde, Rebecca;
Dobrovolny, Diana

Publication type:

Article

THE 340B PROGRAM: A FEDERAL PROGRAM IN DESPERATE NEED OF REVISION AFTER TWO-AND-A-HALF DECADES OF UNCERTAINTY.

Published in:

Journal of Health Care Law & Policy, 2019, v. 22, n. 2, p. 25

By:

FISHER, NICHOLAS C.

Publication type:

Article

European regulation on orphan medicinal products: 10 years of experience and future perspectives.

Published in:

2011

By:

Committee for Orphan Medicinal Products and the European Medicines;
Westermark, Kerstin;
Holm, Birthe Byskov;
Söderholm, Mirjam;
Llinares-Garcia, Jordi;
Rivière, Frida;
Aarum, Stiina;
Butlen-Ducuing, Florence;
Tsigkos, Stelios;
Wilk-Kachlicka, Agnieszka;
N'Diamoi, Cinzia;
Borvendég, János;
Lyons, David;
Sepodes, Bruno;
Bloechl-Daum, Brigitte;
Lhoir, André;
Todorova, Mariana;
Kkolos, Ioannis;
Kubáčková, Kateřina;
Bosch-Traberg, Heidrun

Publication type:

review

Rare diseases, orphan drugs and their regulation: questions and misconceptions.

Published in:

2010

By:

Tambuyzer, Erik

Publication type:

review

Orphan products: an emerging trend in drug approvals.

Published in:

2010

By:

Coté, Timothy;
Kelkar, Aditya;
Kui Xu;
Braun, M. Miles;
Phillips, M. Ian;
Coté, Timothy;
Xu, Kui

Publication type:

Letter

Orphan Drug Designation and Exclusivity for "Same Drugs".

Published in:: Journal of Law, Medicine & Ethics, 2019, v. 47, n. 2, p. 347, doi. 10.1177/1073110519857293
Publication type:: Article

Approximating Future Generic Entry for New Drugs.

Published in:

Journal of Law, Medicine & Ethics, 2019, v. 47, n. 1, p. 177, doi. 10.1177/1073110519840499

By:

Beall, Reed F.;
Darrow, Jonathan J.;
Kesselheim, Aaron S.;
Cook-Deegan, Robert;
Majumder, Mary A.;
McGuire, Amy L.

Publication type:

Article

Will crowdfunding and general solicitation spur orphan drug development for biotechs?

Published in:

Formulary, 2013, v. 48, n. 10, p. 343

By:

Loucks, David

Publication type:

Article

Rare Disease Funding: A Rawlsian Approach to Orphan Drug Legislation.

Published in:

Penn Bioethics Journal, 2015, v. 11, n. 2, p. 13

By:

Barghi, Ameen;
Liu, Jeff

Publication type:

Article

Policy Recommendations to Promote Prescription Drug Competition: A Position Paper From the American College of Physicians.

Published in:

2020

By:

Daniel, Hilary;
Serchen, Josh;
Cooney, Thomas G.;
Health and Public Policy Committee of the American College of Physicians

Publication type:

journal article

Fool Me Twice? The Reemergence of Rofecoxib and the Orphan Drug Act.

Published in:

2019

By:

Lee, Theodore T.;
Solomon, Daniel H.;
Kesselheim, Aaron S.

Publication type:

journal article

Intractable and rare diseases research in Asia.

Published in:

BioScience Trends, 2012, v. 6, n. 2, p. 48, doi. 10.5582/bst.2012.v6.2.48

By:

Song, Peipei;
Gao, Jianjun;
Inagaki, Yoshinori;
Kokudo, Norihiro;
Wei Tang

Publication type:

Article

Insurance Companies' Perspectives on the Orphan Drug Pipeline.

Published in:

American Health & Drug Benefits, 2013, v. 6, n. 9, p. 589

By:

Handfield, Robert;
Feldstein, Josh;
Kaddis, Atheer A.

Publication type:

Article

Inadequately met needs.

Published in:: 2011
Publication type:: Editorial

Corrigendum to "The impact of the rare disease and Orphan Drug Act in Taiwan" [J Food Drug Anal 29 (2021) 717-725].

Published in:

Journal of Food & Drug Analysis, 2022, v. 30, n. 2, p. 282, doi. 10.38212/2224-6614.3407

By:

Ning-Chun Hsiang;
Weng-Foung Huang;
Churn-Shiouh Gau;
Teng-Wen Tsai;
Lin-Chau Chang

Publication type:

Article

The impact of the rare disease and Orphan Drug Act in Taiwan.

Published in:

Journal of Food & Drug Analysis, 2021, v. 29, n. 4, p. 717, doi. 10.38212/2224-6614.3383

By:

Ning-Chun Hsiang;
Weng-Foung Huang;
Churn-Shiouh Gau;
Teng-Wen Tsai;
Lin-Chau Chang

Publication type:

Article

Do investors value the FDA orphan drug designation?

Published in:

2017

By:

Miller, Kathleen L.

Publication type:

journal article

Review of 11 national policies for rare diseases in the context of key patient needs.

Published in:

2017

By:

Dharssi, Safiyya;
Wong-Rieger, Durhane;
Harold, Matthew;
Terry, Sharon

Publication type:

journal article

Evolving prevalence of haematological malignancies in orphan designation procedures in the European Union.

Published in:

2017

By:

Polsinelli, Benedetta;
Tsigkos, Stelios;
Naumann-Winter, Frauke;
Mariz, Segundo;
Sepodes, Bruno

Publication type:

Letter

Challenges in orphan drug development and regulatory policy in China.

Published in:

2017

By:

Cheng, Alice;
Zhi Xie;
Xie, Zhi

Publication type:

journal article

The correlation between HTA recommendations and reimbursement status of orphan drugs in Europe.

Published in:

2016

By:

Kawalec, Paweł;
Sagan, Anna;
Pilc, Andrzej

Publication type:

journal article

Novel treatments for rare rheumatologic disorders: analysis of the impact of 30 years of the US orphan drug act.

Published in:

2016

By:

Lutz, Thomas;
Lampert, Anette;
Hoffmann, Georg F.;
Ries, Markus

Publication type:

journal article

Rare diseases and orphan drugs: Latvian story.

Published in:

Orphanet Journal of Rare Diseases, 2014, v. 9, n. 1, p. 2, doi. 10.1186/s13023-014-0147-z

By:

Logviss, Konstantins;
Krievins, Dainis;
Purvina, Santa

Publication type:

Article

The legal imperative for treating rare disorders.

Published in:

Orphanet Journal of Rare Diseases, 2013, v. 8, n. 1, p. 1, doi. 10.1186/1750-1172-8-135

By:

Hyry, Hanna I.;
P. Roos, Jonathan C.;
Manue, Jeremy;
Cox, Timothy M.

Publication type:

Article