Orphan drug
The use of 2D fingerprint methods to support the assessment of structural similarity in orphan drug legislation.
... In this paper, we have described how fingerprint-based measures of similarity can be used to assess the struc- tural novelty of molecules that are being submitted for consideration as new medicines for rare diseases. ...
11
Do investors value the FDA orphan drug designation?
... The orphan drug designation was created in 1983 when the Orphan Drug Act (ODA) was enacted by Congress, and is administered by the Office of Orphan Products Development at the Food and ...
6
Pediatric Orphan Drug Indications: 2010–2018
... allow drug prices to vary by indication on the basis of factors such as bene fi t to patients and availability of therapeutic ...a drug cannot be predicted with certainty, innovators may choose not to invest ...
11
Examining Why the Canadian Federal Government Placed an Orphan Drug Strategy on Their Decision Agenda Now
... No orphan drug policy has been implemented as of this writing; subsequently no reform has yet been achieved. Instead, the present analysis focuses on the change of the dominant causal story regarding ...
11
Exploring patient and family involvement in the lifecycle of an orphan drug: a scoping review
... Through the literature review, an inventory of existing and proposed opportunities for patients, families, and patient organizations at each stage of the orphan drug lifecycle was compiled. Further ...
14
Incentives for orphan drug research and development in the United States
... the orphan drugs would have been approved in the absence of the ODA incentives ...for orphan drugs before the approval of the ODA ...of orphan drugs per se and as a byproduct of research for ...
7
Challenges in orphan drug development and regulatory policy in China
... and Drug Administration (US FDA) regulatory process for rare disease clinical trials is necessary for developing regulatory policy in ...the Orphan Drug Act of 1983, and has since approved the most ...
8
Development and validation of UV spectrophotometric method for the determination of Nilotinib hydrochloride (An orphan drug)
... Nilotinib hydrochloride,4-methyl-N-[3-(4-methyl-1H-imidazol-1-yl)-5-(trifluoromethyl)phenyl]-3-[(4-pyridin-3- ylpyrimidin-2-yl)amino]benzamidesalt) in the form of the hydrochloride monohydrate salt with trade name ...
7
Shining a light in the black box of orphan drug pricing
... Firstly, orphan drugs with multiple orphan indications are associated with higher ...that orphan drug prices are determined based on the preva- lence of the first indication ...that ...
10
AN INSIGHT ON ORPHAN DRUG DEVELOPMENT STRATEGY IN US AND EUROPE
... for orphan disease causes psychological distress to the patient and the family also, and a feeling of ...of orphan drugs. The Orphan Drug Act (ODA) was came in to force on January 28, 1983, ...
6
What the Orphan Drug Act Has Done Lately for Children With Rare Diseases: A 10-Year Analysis
... an orphan drug designation, a sponsor (eg, an investigator, a company, or an organization) must meet 2 criteria: (1) provide a scienti fi c rationale that a product (P1) can potentially treat or prevent a ...
8
Therapies for Inborn Errors of Metabolism: What Has the Orphan Drug Act Delivered?
... designated orphan drug receives 7 years of exclusive rights to the US mar- ket for the rare-disease ...2142 orphan drugs/biological prod- ucts have been designated, of which 356 have received ...
8
Novel treatments for rare rheumatologic disorders: analysis of the impact of 30 years of the US orphan drug act
... an orphan drug development pathway can vary within same disease entity: both tocilizumab and canakinumab are approved for the treat- ment of systemic juvenile idiopathic ...the orphan program with ...
12
Pressure for drug development in lysosomal storage disorders – a quantitative analysis thirty years beyond the US orphan drug act
... had drug development pressure and what distinguished those with successful development and approvals from diseases not treated or without orphan drug ...for orphan drug designations ...
9
Creation and Development of the Public Service Orphan Drug Human Botulism Immune Globulin
... BIG-IV came into existence through a variety of special circumstances. The high incidence of this rare illness in California enabled a randomized, controlled, pivotal treatment trial to be conducted here. The federal ...
7
Towards an orphan drug policy for the European Union
... In France : The Law of 8.12.1992 modifying book V of the Public Health Code relative to the Pharmacy and drug, article 21, provides for the exceptional use of certain drugs, when they : [r] ...
34
Expert Delphi survey on research and development into drugs for neglected diseases
... diseases, orphan drug acts were created which contain financial and non-financial incentives for the pharmaceutical ...selected orphan drug incentives, or the implementation of a Medical ...
13
Finding a Cure: Incentivizing Partnerships Between Disease Advocacy Groups and Academic Commercial Researchers
... that drug researchers would no longer receive compensation for the patented research tools that they ...in drug development was eroded, it could lead to a decrease in venture capital investments in ...
31
Translational research in retinology
... and Drug Administration granted Orphan Drug designation for RPE cells of Advanced Cell Technology, Inc to initiate its Phase I/II clinical trials using retinal pigment epithelial cells derived from ...
6
ORPHAN DRUGS: NEED OF AN HOUR
... (1). Orphan drugs have been defined in USA as those drugs intended to treat either a rare disease or a more common disease where the sponsor cannot make any profit ...a drug for such disease or condition ...
14