Effectiveness, safety and costs of orphan drugs: an evidence-based review

Effectiveness, safety and costs of orphan drugs: an evidence-based review

Item
http://www.wikidata.org/entity/Q26827237
"Threshold-crossing": A Useful Way to Establish the Counterfactual in Clinical Trials?A comparative study of orphan drug prices in Europe.Do payers value rarity? An analysis of the relationship between disease rarity and orphan drug prices in Europe.The REFRACT-LYMA cohort study: a French observational prospective cohort study of patients with mantle cell lymphomaImpact of orphan drugs on Latvian budget.Recommendations from the European Working Group for Value Assessment and Funding Processes in Rare Diseases (ORPH-VAL).Developments in pulmonary arterial hypertension-targeted therapy for chronic thromboembolic pulmonary hypertension.Addressing the challenge of high-priced prescription drugs in the era of precision medicine: A systematic review of drug life cycles, therapeutic drug markets and regulatory frameworks.Factors Contributing to the Efficacy-Effectiveness Gap in the Case of Orphan Drugs for Metabolic Diseases.Clinical Evidence Supporting US Food and Drug Administration Approval of Otolaryngologic Prescription Drug Indications, 2005-2014.Affordable orphan drugs: a role for not-for-profit organizations.Emerging therapies for inherited retinal degeneration.Establishing core outcome sets for phenylketonuria (PKU) and medium-chain Acyl-CoA dehydrogenase (MCAD) deficiency in children: study protocol for systematic reviews and Delphi surveys.Rare disease prevention and treatment: the need for a level playing field.Revealed preferences towards the appraisal of orphan drugs in Poland - multi criteria decision analysis.Characteristics of clinical trials in rare vs. common diseases: A register-based Latvian study.
P2860

Effectiveness, safety and costs of orphan drugs: an evidence-based review

Item
http://www.wikidata.org/entity/Q26827237
description
2015 nî lūn-bûn
@nan
2015 թուականի Յունիսին հրատարակուած գիտական յօդուած
@hyw
2015 թվականի հունիսին հրատարակված գիտական հոդված
@hy
2015年の論文
@ja
2015年論文
@yue
2015年論文
@zh-hant
2015年論文
@zh-hk
2015年論文
@zh-mo
2015年論文
@zh-tw
2015年论文
@wuu
name
Effectiveness, safety and costs of orphan drugs: an evidence-based review
@ast
Effectiveness, safety and costs of orphan drugs: an evidence-based review
@en
Effectiveness, safety and costs of orphan drugs: an evidence-based review
@nl
type
label
Effectiveness, safety and costs of orphan drugs: an evidence-based review
@ast
Effectiveness, safety and costs of orphan drugs: an evidence-based review
@en
Effectiveness, safety and costs of orphan drugs: an evidence-based review
@nl
prefLabel
Effectiveness, safety and costs of orphan drugs: an evidence-based review
@ast
Effectiveness, safety and costs of orphan drugs: an evidence-based review
@en
Effectiveness, safety and costs of orphan drugs: an evidence-based review
@nl
P1433
P1476
P2093
P2860
P304
P31
P3181
P356
P407
P433
P478
P577
P5875
P698
P921
P932
P3181
P356
P1433
P1476
Effectiveness, safety and costs of orphan drugs: an evidence-based review
@en
P2093
P2860
P304
P31
P3181
P356
10.1136/BMJOPEN-2014-007199
P407
P433
P478
P577
P5875
P698
P921
P932