Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval.

Journal of Clinical Pharmacology
Kenji HaradaShunsuke Ono

Abstract

The basic components of regulatory and supporting policies for orphan drug development appear similar between the United States and Japan, but drugs designated as orphan drugs have been different between the 2 countries. The probabilities of development success (ie, marketing approval) in designated orphan drugs have also been significantly different. In this study, we analyzed recent outcomes of development for orphan drugs designated from 1993 to 2017 in Japan, considering their development and approval status in the United States. Our analysis showed that success for orphan drug development in Japan was apparently associated with prior approval status in the United States. Company size, orphan development experience, and patient enrichment were also positively associated with successful marketing approval. Although similar designations and priority review systems for orphan drugs have been enacted, economic incentives and regulatory conditions provided by the systems seem to be different between the 2 countries, which may lead to varied performance in orphan designation and approval. We need to pay close attention to the impact of industrial global development strategies when comparing the outcomes and performance of differe...Continue Reading

References

Oct 3, 2002·Nature Reviews. Drug Discovery·Marlene E HaffnerMarie Moses
Aug 27, 2004·Journal of Health Politics, Policy and Law·Mary K Olson
Feb 21, 2006·British Journal of Clinical Pharmacology·Roberta JoppiSilvio Garattini
Aug 29, 2006·British Journal of Clinical Pharmacology·James W DearDavid J Webb
Oct 25, 2006·The Journal of Biological Chemistry·Kris A DemaliKeith Burridge
Jan 23, 2008·European Journal of Clinical Pharmacology·Harald E HeemstraHubert G M Leufkens
Jun 17, 2008·Lancet·Arrigo SchieppatiAnita Aperia
Nov 27, 2009·Clinical Pharmacology and Therapeutics·Y HiraiS Ono
Nov 26, 2010·Drug Discovery Today·Harald E HeemstraM Miles Braun
Jun 7, 2011·JAMA : the Journal of the American Medical Association·Aaron S KesselheimJerry Avorn
Nov 19, 2011·Drug Discovery Today·Michelle PutzeistHubert G M Leufkens
Mar 31, 2012·Nature Reviews. Drug Discovery·Irena Melnikova
Nov 28, 2014·Orphanet Journal of Rare Diseases·Stuart A Bell, Catrin Tudur Smith
Oct 28, 2017·Drug Discovery Today·Matthias P HoferStiina Aarum

❮ Previous
Next ❯

Software Mentioned

STATA

Related Concepts

Trending Feeds

COVID-19

Coronaviruses encompass a large family of viruses that cause the common cold as well as more serious diseases, such as the ongoing outbreak of coronavirus disease 2019 (COVID-19; formally known as 2019-nCoV). Coronaviruses can spread from animals to humans; symptoms include fever, cough, shortness of breath, and breathing difficulties; in more severe cases, infection can lead to death. This feed covers recent research on COVID-19.

Blastomycosis

Blastomycosis fungal infections spread through inhaling Blastomyces dermatitidis spores. Discover the latest research on blastomycosis fungal infections here.

Nuclear Pore Complex in ALS/FTD

Alterations in nucleocytoplasmic transport, controlled by the nuclear pore complex, may be involved in the pathomechanism underlying multiple neurodegenerative diseases including Amyotrophic Lateral Sclerosis and Frontotemporal Dementia. Here is the latest research on the nuclear pore complex in ALS and FTD.

Applications of Molecular Barcoding

The concept of molecular barcoding is that each original DNA or RNA molecule is attached to a unique sequence barcode. Sequence reads having different barcodes represent different original molecules, while sequence reads having the same barcode are results of PCR duplication from one original molecule. Discover the latest research on molecular barcoding here.

Chronic Fatigue Syndrome

Chronic fatigue syndrome is a disease characterized by unexplained disabling fatigue; the pathology of which is incompletely understood. Discover the latest research on chronic fatigue syndrome here.

Evolution of Pluripotency

Pluripotency refers to the ability of a cell to develop into three primary germ cell layers of the embryo. This feed focuses on the mechanisms that underlie the evolution of pluripotency. Here is the latest research.

Position Effect Variegation

Position Effect Variagation occurs when a gene is inactivated due to its positioning near heterochromatic regions within a chromosome. Discover the latest research on Position Effect Variagation here.

STING Receptor Agonists

Stimulator of IFN genes (STING) are a group of transmembrane proteins that are involved in the induction of type I interferon that is important in the innate immune response. The stimulation of STING has been an active area of research in the treatment of cancer and infectious diseases. Here is the latest research on STING receptor agonists.

Microbicide

Microbicides are products that can be applied to vaginal or rectal mucosal surfaces with the goal of preventing, or at least significantly reducing, the transmission of sexually transmitted infections. Here is the latest research on microbicides.

Related Papers

Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research
M Mildred, S Lee
Value in Health : the Journal of the International Society for Pharmacoeconomics and Outcomes Research
D KorchaginaF Tavella
© 2021 Meta ULC. All rights reserved