Drugs, Orphan

Known as: Drug, Orphan, Orphan drugs, drugs orphan 
A compound or product that demonstrates promise for the diagnosis and/or treatment of rare diseases and conditions that affect fewer than 200,000… (More)
National Institutes of Health

Papers overview

Semantic Scholar uses AI to extract papers important to this topic.
2014
2014
BACKGROUND Some orphan drugs can cost hundreds of thousands of dollars annually per patient. As a result, payer sensitivity to… (More)
  • table I
  • table II
Is this relevant?
2012
2012
To assess the methodological quality of Orphan Medicinal Product (OMP) dossiers and discuss possible reasons for the small number… (More)
  • table 1
  • table 2
  • table 3
  • table 3
Is this relevant?
2012
2012
Orphan drug incentives have stimulated research into diseases with significant unmet medical need. Although the targeting of… (More)
  • figure 1
  • figure 2
  • figure 3
Is this relevant?
2011
2011
AIM We analysed the Belgian reimbursement decisions of orphan drugs as compared with those of innovative drugs for more common… (More)
Is this relevant?
Highly Cited
2011
Highly Cited
2011
BACKGROUND Orphan drugs are a growing issue of importance to European healthcare policy makers. The success of orphan drug… (More)
  • figure 1
  • figure 2
  • figure 3
  • table 1
  • figure 4
Is this relevant?
Review
2010
Review
2010
Sustained advocacy efforts driven by patients' organizations to make rare diseases a health priority have led to regulatory and… (More)
Is this relevant?
Highly Cited
2010
Highly Cited
2010
OBJECTIVES This study investigates issues associated with the United States Orphan Drug Act. METHODS A comprehensive orphan… (More)
  • figure 1
  • figure 2
  • table 1
  • table 2
  • figure 3
Is this relevant?
Highly Cited
2007
Highly Cited
2007
Historically, patients with rare diseases have been underserved by commercial drug development. In several jurisdictions… (More)
  • figure 1
  • figure 2
Is this relevant?
Highly Cited
2006
Highly Cited
2006
445 ing or evaluating the work of so many volunteers. Some organizations conduct internal evaluations, and they tend to agree… (More)
Is this relevant?
Review
2005
Review
2005
Ultra-orphan drugs are medicines used to treat exceptionally rare diseases that are chronically debilitating or life-threatening… (More)
  • table 1
  • table 1
  • figure 1
Is this relevant?