Research and Markets: US Orphan Drug Clinical Pipeline Insight 2014 – New Report on the $49 Billion Industry

In Press Release by Cecile

Research and Markets 840 420

August 20th, 2014

Research and Markets ( has announced the addition of the “US Orphan Drug Clinical Pipeline Insight 2014” report to their offering.

In 2013, the US market for orphan drugs was estimated worth US$ 49 Billion. With factors like government support, high profitability and many more, this market is expected to surpass US$ 60 Billion by 2018.

The orphan drugs enjoy significant competitive advantages owing to the market exclusivity period after the drug has been authorized. Through this clause, the manufacturer of orphan drugs is given a monopoly status in the market because according to the law, no other company is allowed to market the orphan drugs during the exclusivity period. Additionally, this monopolistic power is further strengthened with the fact that no other alternative health technology exists for many orphan drugs.

In US, the research & development of orphan drugs is facilitated by public programs, government regulations, and policies. The major incentives for research & development of orphan drug include grants, research support, fee waivers, tax incentives, and orphan drug market exclusivity. In a highly specialized market like that of orphan drugs, though the impact of market exclusivity in the US has been positive yet relatively modest, a special platform for continued orphan drug development has been laid down by the economic incentives and public support mechanisms.

US Orphan Drug Pipeline Insight 2014 – Report Highlight:

  • US Orphan Drug Market Insight & Orphan Drug Designation Criteria
  • Reimbursement Policy & FDA Regulatory Framework
  • US Orphan Drug Pipeline by Phase, Indication & Company
  • Marketed Orphan Drug Clinical Insight
  • US Orphan Drug Clinical Pipeline: 570 Drugs
  • Marketed Orphan Drugs in US: 203 Drugs
  • Majority Orphan Drugs in PHASE-II Development: 207 Drugs

Key Topics Covered:

1. Introduction to US Orphan Market

  • Market Overview
  • Clinical Pipeline Overview

2. US Orphan Drug Regulatory Framework

  • Criteria for Orphan Drug Designation
  • Reimbursement Policy for Orphan Drugs
  • FDA Regulation

3. US Orphan Drug Clinical Pipeline by Phase, Indication & Company

  • Unknown
  • Research
  • Preclinical
  • Clinical
  • Phase-I
  • Phase-I/II
  • Phase-II
  • Phase-II/III
  • Phase-III
  • Preregistration
  • Registered

4. Marketed Orphan Drugs Clinical Insight by Indication & Company

5. Suspended & Discontinued Orphan Drugs in Clinical Pipeline by Phase, Indication & Company

  • No development reported
  • Discontinued
  • Market Withdrawal
  • Suspended

6. Competitive Landscape

  • Alexion
  • Biogen Idec
  • Bristol Myers Squibb
  • Celgene
  • Eli Lilly
  • Glaxosmithkline
  • Merck
  • Novartis Pharmaceuticals
  • Pfizer
  • Roche
  • Rare Disease Therapeutics
  • Sanofi