Works matching DE "ORPHAN drugs"

Results: 839

FDA Advisory Committee Review of Supplemental Indications and Reasons for Non-Approval, 2008-2022.

Published in:

Journal of Law, Medicine & Ethics, 2025, v. 53, n. 1, p. 137, doi. 10.1017/jme.2025.53

By:

Zhang, Audrey D.;
Chaudhry, Nikhil;
Ramachandran, Reshma;
Ross, Joseph S.;
Schwartz, Jason L.

Publication type:

Article

Regulatory Framework for Orphan Drug Development in India.

Published in:

IUP Law Review, 2025, v. 15, n. 2, p. 14, doi. 10.71329/IUPLR/2025.15.2.14-26

By:

Mazumdar, Ritwika;
Tyagi, Bhawna

Publication type:

Article

EMA approved orphan medicines since the implementation of the orphan legislation.

Published in:

Orphanet Journal of Rare Diseases, 2025, v. 20, n. 1, p. 1, doi. 10.1186/s13023-025-03756-7

By:

Hahl, Eveliina;
Kurko, Terhi;
Koskinen, Hanna;
Airaksinen, Marja;
Sarnola, Kati

Publication type:

Article

Unlocking access: a comprehensive analysis of medicines accessibility for rare diseases in Thailand.

Published in:

Orphanet Journal of Rare Diseases, 2025, v. 20, n. 1, p. 1, doi. 10.1186/s13023-025-03754-9

By:

Suwattanapreeda, Siriwat;
Hirunrassamee, Sanita;
Sooksriwong, Chaoncin;
Maluangnon, Kusawadee;
Chuachantra, Thirapich;
Kuchaisit, Krissana;
Osirisakul, Niti

Publication type:

Article

Voreloxin Gets Orphan Drug Status for Acute Myeloid Leukemia.

Published in:: Oncology (08909091), 2009, v. 23, n. 14, p. 1221
Publication type:: Article

FDA Grant's Orphan Drug Designation for TNFerade.

Published in:: Oncology (08909091), 2009, v. 23, n. 14, p. 1221
Publication type:: Article

Decitabine Receives US Orphan Drug Designation for AML.

Published in:: Oncology (08909091), 2006, v. 20, n. 10, p. 1282
Publication type:: Article

Orphan Drug Status Granted to Sorafenib for Liver Cancer.

Published in:: Oncology (08909091), 2006, v. 20, n. 6, p. 650
Publication type:: Article

Economic evaluation of orphan drug Lutetium-Octreotate vs. Octreotide long-acting release for patients with an advanced midgut neuroendocrine tumour in the Netherlands.

Published in:

European Journal of Health Economics, 2021, v. 22, n. 6, p. 991, doi. 10.1007/s10198-021-01303-2

By:

Hagendijk, Marije E.;
van der Schans, Simon;
Boersma, Cornelis;
Postma, Maarten J.;
van der Pol, Simon

Publication type:

Article

Predictors of negotiated prices for new drugs in Germany.

Published in:

2020

By:

Gandjour, Afschin;
Schüßler, Sofia;
Hammerschmidt, Thomas;
Dintsios, Charalabos-Markos

Publication type:

journal article

Funding orphan medicinal products beyond price: sustaining an ecosystem.

Published in:

2019

By:

de Sola-Morales, Oriol

Publication type:

Editorial

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

Early benefit assessment (EBA) in Germany: analysing decisions 18 months after introducing the new AMNOG legislation.

Published in:

European Journal of Health Economics, 2014, v. 15, n. 6, p. 577, doi. 10.1007/s10198-013-0495-y

By:

Ruof, Jörg;
Schwartz, Friedrich;
Schulenburg, J.-Matthias;
Dintsios, Charalabos-Markos

Publication type:

Article

The impact of new (orphan) drug approvals on premature mortality from rare diseases in the United States and France, 1999-2007.

Published in:

European Journal of Health Economics, 2013, v. 14, n. 1, p. 41, doi. 10.1007/s10198-011-0349-4

By:

Lichtenberg, Frank

Publication type:

Article

The challenges of Chagas Disease-- grim outlook or glimmer of hope.

Published in:

2007

By:

Tarleton, Rick L.;
Reithinger, Richard;
Urbina, Julio A.;
Kitron, Uriel;
Gürtler, Ricardo E.;
Gürtler, Ricardo E

Publication type:

journal article

Removal of the EMA orphan designation upon request of the sponsor: cui prodest?

Published in:

European Journal of Clinical Pharmacology, 2021, v. 77, n. 7, p. 1057, doi. 10.1007/s00228-021-03096-y

By:

Montanaro, Nicola;
Bonaldo, Giulia;
Motola, Domenico

Publication type:

Article

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

Regulatory characteristics and pivotal study design of US Food and Drug Administration approval of drugs for major vs. minor cancer.

Published in:

European Journal of Clinical Pharmacology, 2019, v. 75, n. 9, p. 1193, doi. 10.1007/s00228-019-02695-0

By:

Yamashita, Kenji;
Kaneko, Masayuki;
Narukawa, Mamoru

Publication type:

Article

Policies and availability of orphan medicines in outpatient care in 24 European countries.

Published in:

European Journal of Clinical Pharmacology, 2018, v. 74, n. 7, p. 895, doi. 10.1007/s00228-018-2457-x

By:

Sarnola, Kati;
Ahonen, Riitta;
Martikainen, Jaana E.;
Timonen, Johanna

Publication type:

Article

Time to revisit the orphan drug law.

Published in:

2012

By:

Garattini, Silvio

Publication type:

Editorial

Factors associated with success of market authorisation applications for pharmaceutical drugs submitted to the European Medicines Agency.

Published in:

European Journal of Clinical Pharmacology, 2010, v. 66, n. 1, p. 39, doi. 10.1007/s00228-009-0756-y

By:

Regnstrom, Jan;
Koenig, Franz;
Aronsson, Bo;
Reimer, Tatiana;
Svendsen, Kristian;
Tsigkos, Stelios;
Flamion, Bruno;
Eichler, Hans-Georg;
Vamvakas, Spiros

Publication type:

Article

Predictors of orphan drug approval in the European Union.

Published in:

European Journal of Clinical Pharmacology, 2008, v. 64, n. 5, p. 545, doi. 10.1007/s00228-007-0454-6

By:

Heemstra, Harald E.;
de Vrueh, Remco L.;
van Weely, Sonja;
Büller, Hans A.;
Leufkens, Hubert G. M.

Publication type:

Article

Medicines for children licensed by the European Medicines Agency (EMEA): the balance after 10 years.

Published in:

European Journal of Clinical Pharmacology, 2006, v. 62, n. 11, p. 947, doi. 10.1007/s00228-006-0193-0

By:

Ceci, A.;
Felisi, M.;
Baiardi, P.;
Bonifazi, F.;
Catapano, M.;
Giaquinto, C.;
Nicolosi, A.;
Sturkenboom, M.;
Neubert, A.;
Wong, I.

Publication type:

Article

Hereditary Angio-Oedema for Dermatologists.

Published in:

2019

By:

Bygum, Anette

Publication type:

journal article

Polypharmacological repurposing approach identifies approved drugs as potential inhibitors of Mycobacterium tuberculosis.

Published in:

Biochemical Journal, 2023, v. 480, n. 14, p. 1079, doi. 10.1042/BCJ20230143

By:

Singh, Jasdeep;
Quadir, Neha;
Vashishtha, Shubham;
Chakraborty, Ankan;
Alam, Anwar;
Kundu, Bishwajit;
Ahmad, Uzair;
Sundar, Durai;
Ehtesham, Nasreen Z.;
Hasnain, Seyed E.

Publication type:

Article

Price and reimbursement for orphan medicines and managed entry agreements: does Italy need a framework?

Published in:

Global & Regional Health Technology Assessment, 2021, v. 8, n. 1, p. 114, doi. 10.33393/grhta.2021.2278

By:

Jommi, Claudio;
Addis, Antonio;
Martini, Nello;
Nicod, Elena;
Pani, Marcello;
Scopinaro, Annalisa;
Vogler, Sabine

Publication type:

Article

FDA Grants Accelerated Approval to Talquetamab-Tgvs for Relapsed or Refractory Multiple Myeloma.

Published in:: ONS Voice, 2023, p. 1
Publication type:: Article

FDA Approves Pralsetinib for Non-Small Cell Lung Cancer With RET Gene Fusions.

Published in:: ONS Voice, 2023, p. 2
Publication type:: Article

Revision of pharmaceutical legislation in the context of antimicrobial resistance and challenges facing the introduction of innovative antimicrobial medical products in Europe.

Published in:

Pharmacia (0428-0296), 2024, v. 71, n. 1, p. 1, doi. 10.3897/pharmacia.71.e122414

By:

Toshev, Atanas;
Petkova-Gueorguieva, Elina;
Mihaylova, Anna;
Lebanova, Hristina;
Stoev, Svetoslav;
Balkanski, Stefan;
Peikova, Lily;
Syarova, Svetlana;
Madzharov, Vasil;
Gueorguiev, Stanislav

Publication type:

Article

CATALYST PHARMS., INC. V. BECERRA: WHEN THE FOOD AND DRUG ADMINISTRATION REPEATEDLY IGNORES THE PLAIN LANGUAGE OF THE ORPHAN DRUG ACT (ODA).

Published in:

Journal of Law & Health, 2023, v. 36, n. 2, p. 139

By:

Yifan Wang

Publication type:

Article

Speaker Abstracts.

Published in:: Basic & Clinical Pharmacology & Toxicology, 2013, v. 113, p. 4, doi. 10.1111/bcpt.12127
Publication type:: Article

Efficacy and Safety of Lomitapide in Hypercholesterolemia.

Published in:

American Journal of Cardiovascular Drugs, 2017, v. 17, n. 4, p. 299, doi. 10.1007/s40256-017-0214-7

By:

Liu, Xin;
Men, Peng;
Wang, Yuhui;
Zhai, Suodi;
Zhao, Zhigang;
Liu, George

Publication type:

Article

Lomitapide.

Published in:: American Journal of Cardiovascular Drugs, 2011, v. 11, n. 5, p. 347, doi. 10.2165/11533560-000000000-00000
Publication type:: Article

Mipomersen.

Published in:: American Journal of Cardiovascular Drugs, 2010, v. 10, n. 4, p. 271, doi. 10.2165/11533460-000000000-00000
Publication type:: Article

ROS1 rearrangements are uncommon in biliary tract cancers.

Published in:

Oncology Letters, 2020, v. 20, n. 6, p. N.PAG, doi. 10.3892/ol.2020.12179

By:

Mazzoni, Francesca;
Petreni, Paolo;
Vasile, Enrico;
Panebianco, Michele;
Casadei-Gardini, Andrea;
Negri, Francesca;
Lunghi, Alice;
Pillozzi, Serena;
Vivaldi, Caterina;
Gervasi, Erika;
Frassineti, Giovanni Luca;
Messerini, Luca;
Jocollé, Genny;
Bisagni, Alessandra;
Antonuzzo, Lorenzo;
Rossi, Giulio

Publication type:

Article

A scoping review of the role of managed entry agreements in upcoming drugs for amyotrophic lateral sclerosis: learning from the case of spinal muscular atrophy.

Published in:

Amyotrophic Lateral Sclerosis & Frontotemporal Degeneration, 2025, v. 26, n. 1/2, p. 48, doi. 10.1080/21678421.2024.2400522

By:

García-Parra, Beliu;
Guiu, Josep M.;
Povedano, MÓnica;
Modamio, Pilar

Publication type:

Article

Reimbursement Status and Recommendations Related to Orphan Drugs in European Countries.

Published in:

Frontiers in Pharmacology, 2019, v. 10, p. 1, doi. 10.3389/fphar.2019.01279

By:

Stawowczyk, Ewa;
Malinowski, Krzysztof Piotr;
Kawalec, Paweł;
Bobiński, Rafał;
Siwiec, Jacek;
Panteli, Dimitra;
Eckhardt, Helene;
Simoens, Steven;
Agusti, Antònia;
Dooms, Marc;
Pilc, Andrzej

Publication type:

Article

Ophthalmic Drug Discovery in the United States over the past Two Decades.

Published in:

Ophthalmic Epidemiology, 2021, v. 28, n. 1, p. 21, doi. 10.1080/09286586.2020.1786591

By:

Gu, David;
Janetos, Timothy M.

Publication type:

Article

Prioritizing Rare Diseases: Psychological Effects Influencing Medical Decision Making.

Published in:

Medical Decision Making, 2017, v. 37, n. 5, p. 567, doi. 10.1177/0272989X17691744

By:

Wiss, Johanna;
Levin, Lars-Ake;
Andersson, David;
Tinghög, Gustav

Publication type:

Article

Voxelotor: First Approval.

Published in:

Drugs, 2020, v. 80, n. 2, p. 209, doi. 10.1007/s40265-020-01262-7

By:

Blair, Hannah A.

Publication type:

Article

Drug Development for Rare Paediatric Epilepsies: Current State and Future Directions.

Published in:

Drugs, 2019, v. 79, n. 18, p. 1917, doi. 10.1007/s40265-019-01223-9

By:

Auvin, Stéphane;
Avbersek, Andreja;
Bast, Thomas;
Chiron, Catherine;
Guerrini, Renzo;
Kaminski, Rafal M.;
Lagae, Lieven;
Muglia, Pierandrea;
Cross, J. Helen

Publication type:

Article

Generic Substitution of Orphan Drugs for the Treatment of Rare Diseases: Exploring the Potential Challenges.

Published in:

Drugs, 2018, v. 78, n. 4, p. 399, doi. 10.1007/s40265-018-0882-x

By:

Di Paolo, Antonello;
Arrigoni, Elena

Publication type:

Article

Factors Contributing to the Efficacy-Effectiveness Gap in the Case of Orphan Drugs for Metabolic Diseases.

Published in:

Drugs, 2017, v. 77, n. 13, p. 1461, doi. 10.1007/s40265-017-0788-z

By:

Schuller, Y.;
Hollak, C.;
Gispen-de Wied, C.;
Stoyanova-Beninska, V.;
Biegstraaten, M.

Publication type:

Article

Pitolisant: First Global Approval.

Published in:

Drugs, 2016, v. 76, n. 13, p. 1313, doi. 10.1007/s40265-016-0620-1

By:

Syed, Yahiya

Publication type:

Article

Ataluren: First Global Approval.

Published in:

2014

By:

Ryan, Nicola

Publication type:

Journal Article

Orphan Drugs for Rare Diseases: Is it Time to Revisit Their Special Market Access Status?

Published in:

2012

By:

Simoens, Steven;
Cassiman, David;
Dooms, Marc;
Picavet, Eline

Publication type:

Journal Article

Pharmacological Management of Epilepsy: Recent Advances and Future Prospects.

Published in:

Drugs, 2008, v. 68, n. 14, p. 1925, doi. 10.2165/00003495-200868140-00001

By:

Landmark, Cecilie Johannessen;
Johannessen, Svein I.

Publication type:

Article

Orphan Drug Pricing and Costs: A Case Study of Kalydeco and Orkambi.

Published in:

Healthcare Policy, 2019, v. 15, n. 1, p. 70

By:

HOLLIS, AIDAN

Publication type:

Article

Some Numbers behind Canada's Decision to Adopt an Orphan Drug Policy: US Orphan Drug Approvals in Canada, 1997-2012.

Published in:

Healthcare Policy, 2016, v. 11, n. 4, p. 70

By:

HERDER, MATTHEW;
MARK KRAHN, TIMOTHY

Publication type:

Article

Reimbursement of Drugs for Rare Diseases through the Public Healthcare System in Canada: Where Are We Now?

Published in:

Healthcare Policy, 2015, v. 11, n. 1, p. 15

By:

MENON, DEVIDAS;
CLARK, DEREK;
STAFINSKI, TANIA

Publication type:

Article