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 trials | | 2022 |
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 welfare | Decision 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 2021 | JAMA Health Forum | 2022 |
Discrepancies between FDA documents and ClinicalTrials.gov for Orphan Drug-related clinical trial data | PLOS Global Public Health | 2022 |
Oncologic Rare Disease Drug Development | | 2021 |
Real-world Use of and Spending on New Oral Targeted Cancer Drugs in the US, 2011-2018 | JAMA Internal Medicine | 2021 |
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 Drugs | | 2019 |
Essential list of medicinal products for rare diseases: recommendations from the IRDiRC Rare Disease Treatment Access Working Group | Orphanet Journal of Rare Diseases | 2021 |
Inclusion of Infants and Neonates in Pediatric Orphan Product Approvals | Clinical Pharmacology and Therapeutics | 2021 |
Quantitative retrospective natural history modeling for orphan drug development | Journal of Inherited Metabolic Disease | 2021 |
Market Exclusivity for Drugs with Multiple Orphan Approvals (1983-2017) and Associated Budget Impact in the US | Pharmacoeconomics | 2020 |
Real-World Evidence Utilization in Clinical Development Reflected by US Product Labeling: Statistical Review | Therapeutic Innovation and Regulatory Science | 2020 |
Postmarketing Safety-Related Regulatory Actions for New Therapeutic Biologics Approved in the United States 2002-2014: Similarities and Differences With New Molecular Entities | Clinical Pharmacology and Therapeutics | 2020 |
BIO 300: a promising radiation countermeasure under advanced development for acute radiation syndrome and the delayed effects of acute radiation exposure | Expert Opinion on Investigational Drugs | 2020 |
Manifestaciones osteoarticulares de las esfingolipidosis en adultos | EMC - Aparato Locomotor | 2020 |
Post-marketing safety-related regulatory actions on first-in-class drugs: A double-cohort study | Journal of Clinical Pharmacy and Therapeutics | 2020 |
Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval | Journal of Clinical Pharmacology | 2020 |
Application of orphan drug designation to cancer treatments (2008-2017): a comprehensive and comparative analysis of the USA and EU | BMJ Open | 2019 |
Clinical characteristics of 248 patients with Krabbe disease: quantitative natural history modeling based on published cases | Genetics in Medicine | 2019 |
International collaborative actions and transparency to understand, diagnose, and develop therapies for rare diseases | EMBO Molecular Medicine | 2019 |
An Analysis of Follow-On Development in New Drug Classes, January 1986-June 2018 | Clinical Pharmacology and Therapeutics | 2019 |
Estimating the clinical cost of drug development for orphan versus non-orphan drugs | Orphanet Journal of Rare Diseases | 2019 |
A multidisciplinary review of the policy, intellectual property rights, and international trade environment for access and affordability to essential cancer medications | Globalization and Health | 2019 |
The work, goals, challenges, achievements, and recommendations of orphan medicinal product organizations in India: an interview-based study | Orphanet Journal of Rare Diseases | 2019 |
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 Balamuthia | International Journal for Parasitology: Drugs and Drug Resistance | 2019 |
Ultra-orphan lysosomal storage diseases: A cross-sectional quantitative analysis of the natural history of alpha-mannosidosis | Journal of Inherited Metabolic Disease | 2019 |
Precision Medicines' Impact on Orphan Drug Designation | Clinical and Translational Science | 2019 |
Orphan Drug Designation and Exclusivity for "Same Drugs" | Journal of Law, Medicine and Ethics | 2019 |
FDA's Office of Orphan Products Development: providing incentives to promote the development of products for rare diseases | Journal of Pharmacokinetics and Pharmacodynamics | 2019 |
Pharmaceutical compounding of orphan active ingredients in Belgium: how community and hospital pharmacists can address the needs of patients with rare diseases | Orphanet Journal of Rare Diseases | 2019 |
A cross-sectional quantitative analysis of the natural history of free sialic acid storage disease-an ultra-orphan multisystemic lysosomal storage disorder | Genetics in Medicine | 2019 |
Analysis of factors related to the occurrence of important drug-specific postmarketing safety-related regulatory actions: A cohort study focused on first-in-class drugs | Pharmacoepidemiology and Drug Safety | 2018 |
Getting to the Heart of the Matter: Lysosomal Storage Diseases That Manifest a Cardiac Phenotype | Current Genetic Medicine Reports | 2018 |
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 drugs | Trials | 2018 |
Investigating the landscape of US orphan product approvals | Orphanet Journal of Rare Diseases | 2018 |
Drug Product Considerations for Biosimilars | AAPS Advances in the Pharmaceutical Sciences Series | 2018 |
Recommendations for the design of small population clinical trials | Orphanet Journal of Rare Diseases | 2018 |
A cross-sectional quantitative analysis of the natural history of Farber disease: an ultra-orphan condition with rheumatologic and neurological cardinal disease features | Genetics in Medicine | 2018 |
Clinical and Economic Evaluation of Repository Corticotropin Injection: A Narrative Literature Review of Treatment Efficacy and Healthcare Resource Utilization for Seven Key Indications | Advances in Therapy | 2017 |
The Future Role of the United States in Global Health: Emphasis on Cardiovascular Disease | Journal of the American College of Cardiology | 2017 |
Anthracycline-induced cardiotoxicity in patients with paediatric bone sarcoma and soft tissue sarcoma | Cardiology in the Young | 2017 |
The Use of Social Media in Orphan Drug Development | Clinical Therapeutics | 2017 |
Of patents and patent disputes: The TNFα patent files. Part 1: Humira | Human Antibodies | 2017 |
Quantitative clinical characteristics of 53 patients with MPS VII: a cross-sectional analysis | Genetics in Medicine | 2017 |
Ethical imperatives of timely access to orphan drugs: is possible to reconcile economic incentives and patients' health needs? | Orphanet Journal of Rare Diseases | 2017 |
Participation of the elderly, women, and minorities in pivotal trials supporting 2011-2013 U.S. Food and Drug Administration approvals | Trials | 2016 |