US Orphan Drug Pipeline Analysis

Publish Date:- Oct-2013       No Of Pages (880)

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In recent years, the pharmaceutical industry has been experiencing a paradigm shift. While a large pool of patients was considered as a major source of revenue for pharma companies in the past, the focus is now gradually shifting to small sections of patients suffering from rare disease. In US, this pool of patients is gradually growing and orphan drugs are becoming an extremely attractive business proposition for the pharmaceuticals industry. With close to 30 million people in the US having some kind of rare disease complaints, the increasing activities around the development of orphan drugs only imply that the appropriate treatments for unmet needs are increasing, thus increasing the profit margins in the future.

US Orphan Drug Pipeline Analysis” by PNS Pharma gives comprehensive insight on the various orphan designated drugs being developed for the treatment of rare disease. Research report covers all the orphan designated drugs being developed in various development phases. This report enables pharmaceutical companies, collaborators and other associated stake holders to identify and analyze the available investment opportunity in the US Orphan Drug market based upon development process.

Following parameters for each drug profile in development phase are covered in “US Orphan Drug Pipeline Analysis” research report:

• Drug Profile Overview

• Alternate Names for Drug

• Active Indication

• Phase of Development

• Mechanism of Action

• Brand Name

• Patent Information

• Orphan Designation by Indication, Country & Organisation

• Country for Clinical Trial

• Owner / Originator/ Licensee/Collaborator

• Administrative Route

• Drug Class

• ATC Codes



US Orphan Drug Pipeline by Clinical Phase:

• Research: 4

• Preclinical: 54

• Phase-I: 68

• Phase-I/II: 62

• Phase-II: 169

• Phase-II/III: 15

• Phase-III: 93

• Preregistration: 28

• Registration: 18

• Marketed: 137


In recent years, the pharmaceutical industry has been experiencing a paradigm shift. While a large pool of patients was considered as a major source of revenue for pharma companies in the past, the focus is now gradually shifting to small sections of patients suffering from rare disease. In US, this pool of patients is gradually growing and orphan drugs are becoming an extremely attractive business proposition for the pharmaceuticals industry. With close to 30 million people in the US having some kind of rare disease complaints, the increasing activities around the development of orphan drugs only imply that the appropriate treatments for unmet needs are increasing, thus increasing the profit margins in the future.

US Orphan Drug Pipeline Analysis” by PNS Pharma gives comprehensive insight on the various orphan designated drugs being developed for the treatment of rare disease. Research report covers all the orphan designated drugs being developed in various development phases. This report enables pharmaceutical companies, collaborators and other associated stake holders to identify and analyze the available investment opportunity in the US Orphan Drug market based upon development process.

Following parameters for each drug profile in development phase are covered in “US Orphan Drug Pipeline Analysis” research report:

• Drug Profile Overview

• Alternate Names for Drug

• Active Indication

• Phase of Development

• Mechanism of Action

• Brand Name

• Patent Information

• Orphan Designation by Indication, Country & Organisation

• Country for Clinical Trial

• Owner / Originator/ Licensee/Collaborator

• Administrative Route

• Drug Class

• ATC Codes



US Orphan Drug Pipeline by Clinical Phase:

• Research: 4

• Preclinical: 54

• Phase-I: 68

• Phase-I/II: 62

• Phase-II: 169

• Phase-II/III: 15

• Phase-III: 93

• Preregistration: 28

• Registration: 18

• Marketed: 137

1. US Orphan Drug Market Overview



2. US Orphan Drug Market Regulatory Framework

  2.1 General Provisions

  2.2 Written Recommendations for Investigations of Orphan Drugs

  2.3 Designation of an Orphan Drug

  2.4 Orphan Drug Exclusive Approval

  2.5 Open Protocols for Investigations

  2.6 Availability of Information



3. Orphan Drug Clinical Phase: Research

  3.1 Overview

  3.2 Orphan Drug Profiles in Clinical Phase



4. Orphan Drug Clinical Phase: Preclinical

  4.1 Overview

  4.2 Orphan Drug Profiles in Clinical Phase



5. Orphan Drug Clinical Phase: Phase-I

  5.1 Overview

  5.2 Orphan Drug Profiles in Clinical Phase



6. Orphan Drug Clinical Phase: Phase-I/II

  6.1 Overview

  6.2 Orphan Drug Profiles in Clinical Phase



7. Orphan Drug Clinical Phase: Phase-II

  7.1 Overview

  7.2 Orphan Drug Profiles in Clinical Phase



8. Orphan Drug Clinical Phase: Phase-II/III

  8.1 Overview

  8.2 Orphan Drug Profiles in Clinical Phase



9. Orphan Drug Clinical Phase: Phase-III

  9.1 Overview

  9.2 Orphan Drug Profiles in Clinical Phase



10. Orphan Drug Clinical Phase: Pre-Registration

  10.1 Overview

  10.2 Orphan Drug Profiles in Clinical Phase



11. Orphan Drug Clinical Phase: Registered

  11.1 Overview

  11.2 Orphan Drug Profiles in Clinical Phase



12. Marketed Orphan Drugs

  12.1 Overview

  12.2 Orphan Drug Profiles


Each Drug Profile has Tables Representing Following Information:

• Alternate Names

• Originator & Owner

• Collaborator

• Technology Provider

• Licensee

• Highest Development Phase

• Indications

• Class

• Mechanism of Action

• ATC code

• Designated Brand Name & Orphan Designation