53
Citations
32
Journals
2014
First Cited
2022
Last Cited

Articles citing This Document (Page 1 of 2)

ArticleJournalYear
A systematic review of disease prevalence, health-related quality of life, and economic outcomes associated with Friedreich’s Ataxia
FDA Orphan Designations, Approvals, and Regulatory Review Time since the Enactment of the Orphan Drug Act (1983)
Rare disease patients in India are rarely involved in international orphan drug trials2022
Association between control group therapy and magnitude of clinical benefit of cancer drugs.
Converting a drug from off‐label to on‐label use: Government subsidies and patient welfareDecision Sciences
Approvals and Timing of New Formulations of Novel Drugs Approved by the US Food and Drug Administration Between 1995 and 2010 and Followed Through 2021JAMA Health Forum2022
Discrepancies between FDA documents and ClinicalTrials.gov for Orphan Drug-related clinical trial dataPLOS Global Public Health2022
Oncologic Rare Disease Drug Development2021
Real-world Use of and Spending on New Oral Targeted Cancer Drugs in the US, 2011-2018JAMA Internal Medicine2021
Contrasting Evidence to Reimbursement Reality for Off-label use (OLU) of Drug Treatments in Cancer Care – Rationale and Design of the CEIT-OLU-project
Orphan Diseases and Drugs2019
Essential list of medicinal products for rare diseases: recommendations from the IRDiRC Rare Disease Treatment Access Working GroupOrphanet Journal of Rare Diseases2021
Inclusion of Infants and Neonates in Pediatric Orphan Product ApprovalsClinical Pharmacology and Therapeutics2021
Quantitative retrospective natural history modeling for orphan drug developmentJournal of Inherited Metabolic Disease2021
Market Exclusivity for Drugs with Multiple Orphan Approvals (1983-2017) and Associated Budget Impact in the USPharmacoeconomics2020
Real-World Evidence Utilization in Clinical Development Reflected by US Product Labeling: Statistical ReviewTherapeutic Innovation and Regulatory Science2020
Postmarketing Safety-Related Regulatory Actions for New Therapeutic Biologics Approved in the United States 2002-2014: Similarities and Differences With New Molecular EntitiesClinical Pharmacology and Therapeutics2020
BIO 300: a promising radiation countermeasure under advanced development for acute radiation syndrome and the delayed effects of acute radiation exposureExpert Opinion on Investigational Drugs2020
Manifestaciones osteoarticulares de las esfingolipidosis en adultosEMC - Aparato Locomotor2020
Post-marketing safety-related regulatory actions on first-in-class drugs: A double-cohort studyJournal of Clinical Pharmacy and Therapeutics2020
Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing ApprovalJournal of Clinical Pharmacology2020
Application of orphan drug designation to cancer treatments (2008-2017): a comprehensive and comparative analysis of the USA and EUBMJ Open2019
Clinical characteristics of 248 patients with Krabbe disease: quantitative natural history modeling based on published casesGenetics in Medicine2019
International collaborative actions and transparency to understand, diagnose, and develop therapies for rare diseasesEMBO Molecular Medicine2019
An Analysis of Follow-On Development in New Drug Classes, January 1986-June 2018Clinical Pharmacology and Therapeutics2019
Estimating the clinical cost of drug development for orphan versus non-orphan drugsOrphanet Journal of Rare Diseases2019
A multidisciplinary review of the policy, intellectual property rights, and international trade environment for access and affordability to essential cancer medicationsGlobalization and Health2019
The work, goals, challenges, achievements, and recommendations of orphan medicinal product organizations in India: an interview-based studyOrphanet Journal of Rare Diseases2019
Identification of plicamycin, TG02, panobinostat, lestaurtinib, and GDC-0084 as promising compounds for the treatment of central nervous system infections caused by the free-living amebae Naegleria, Acanthamoeba and BalamuthiaInternational Journal for Parasitology: Drugs and Drug Resistance2019
Ultra-orphan lysosomal storage diseases: A cross-sectional quantitative analysis of the natural history of alpha-mannosidosisJournal of Inherited Metabolic Disease2019
Precision Medicines' Impact on Orphan Drug DesignationClinical and Translational Science2019
Orphan Drug Designation and Exclusivity for "Same Drugs"Journal of Law, Medicine and Ethics2019
FDA's Office of Orphan Products Development: providing incentives to promote the development of products for rare diseasesJournal of Pharmacokinetics and Pharmacodynamics2019
Pharmaceutical compounding of orphan active ingredients in Belgium: how community and hospital pharmacists can address the needs of patients with rare diseasesOrphanet Journal of Rare Diseases2019
A cross-sectional quantitative analysis of the natural history of free sialic acid storage disease-an ultra-orphan multisystemic lysosomal storage disorderGenetics in Medicine2019
Analysis of factors related to the occurrence of important drug-specific postmarketing safety-related regulatory actions: A cohort study focused on first-in-class drugsPharmacoepidemiology and Drug Safety2018
Getting to the Heart of the Matter: Lysosomal Storage Diseases That Manifest a Cardiac PhenotypeCurrent Genetic Medicine Reports2018
The Comparative Effectiveness of Innovative Treatments for Cancer (CEIT-Cancer) project: Rationale and design of the database and the collection of evidence available at approval of novel drugsTrials2018
Investigating the landscape of US orphan product approvalsOrphanet Journal of Rare Diseases2018
Drug Product Considerations for BiosimilarsAAPS Advances in the Pharmaceutical Sciences Series2018
Recommendations for the design of small population clinical trialsOrphanet Journal of Rare Diseases2018
A cross-sectional quantitative analysis of the natural history of Farber disease: an ultra-orphan condition with rheumatologic and neurological cardinal disease featuresGenetics in Medicine2018
Clinical and Economic Evaluation of Repository Corticotropin Injection: A Narrative Literature Review of Treatment Efficacy and Healthcare Resource Utilization for Seven Key IndicationsAdvances in Therapy2017
The Future Role of the United States in Global Health: Emphasis on Cardiovascular DiseaseJournal of the American College of Cardiology2017
Anthracycline-induced cardiotoxicity in patients with paediatric bone sarcoma and soft tissue sarcomaCardiology in the Young2017
The Use of Social Media in Orphan Drug DevelopmentClinical Therapeutics2017
Of patents and patent disputes: The TNFα patent files. Part 1: HumiraHuman Antibodies2017
Quantitative clinical characteristics of 53 patients with MPS VII: a cross-sectional analysisGenetics in Medicine2017
Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients' health needs?Orphanet Journal of Rare Diseases2017
Participation of the elderly, women, and minorities in pivotal trials supporting 2011-2013 U.S. Food and Drug Administration approvalsTrials2016