Global Orphan Drug Pipeline Analysis

Publish Date:- Dec-2013       No Of Pages (1690)

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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 & Europe, this pool of patients is gradually growing and orphan drugs are becoming an extremely attractive business proposition for the pharmaceuticals industry. With close to 65-70 Million people in the US & Europe 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. As the Asian pharmaceutical markets are still in growth stage, the opportunities for orphan drugs in Asia are also immense and getting increasingly popular. It is expected that by 2020, the Asian orphan drugs market would witness steady growth.

Global 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 Orphan Drug market based upon development process.

Following parameters for each orphan designated drug profile in development phase are covered in “Global 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



Global Orphan Drug Pipeline by Clinical Phase:

• Research: 3

• Preclinical: 71

• Clinical: 4

• Phase-I: 61

• Phase-I/II: 71

• Phase-II: 198

• Phase-II/III: 20

• Phase-III: 103

• Preregistration: 26

• Registration: 17

• Marketed: 244

• Unknown: 1


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 & Europe, this pool of patients is gradually growing and orphan drugs are becoming an extremely attractive business proposition for the pharmaceuticals industry. With close to 65-70 Million people in the US & Europe 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. As the Asian pharmaceutical markets are still in growth stage, the opportunities for orphan drugs in Asia are also immense and getting increasingly popular. It is expected that by 2020, the Asian orphan drugs market would witness steady growth.

Global 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 Orphan Drug market based upon development process.

Following parameters for each orphan designated drug profile in development phase are covered in “Global 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



Global Orphan Drug Pipeline by Clinical Phase:

• Research: 3

• Preclinical: 71

• Clinical: 4

• Phase-I: 61

• Phase-I/II: 71

• Phase-II: 198

• Phase-II/III: 20

• Phase-III: 103

• Preregistration: 26

• Registration: 17

• Marketed: 244

• Unknown: 1

1. Global Orphan Drugs Market Overview



2. Orphan Drug Clinical Development Phase: Unknown

  2.1 Overview

  2.2 Orphan Drug Profile in Clinical Phase



3. Orphan Drug Clinical Development Phase: Research

  3.1 Overview

  3.2 Orphan Drug Profile in Clinical Phase



4. Orphan Drug Clinical Development Phase: Preclinical

  4.1 Overview

  4.2 Orphan Drug Profile in Clinical Phase



5. Orphan Drug Clinical Drug Development Phase: Clinical

  5.1 Overview

  5.2 Orphan Drug Profile in Clinical Phase



6. Orphan Drug Clinical Development Phase: Phase-I

  6.1 Overview

  6.2 Orphan Drug Profile in Clinical Phase



7. Orphan Drug Clinical Development Phase: Phase-I/II

  7.1 Overview

  7.2 Orphan Drug Profile in Clinical Phase



8. Orphan Drug Clinical Development Phase: Phase-II

  8.1 Overview

  8.2 Orphan Drug Profile in Clinical Phase



9. Orphan Drug Clinical Development Phase: Phase-II/III

  9.1 Overview

  9.2 Orphan Drug Profile in Clinical Phase



10. Orphan Drug Clinical Development Phase: Phase-III

  10.1 Overview

  10.2 Orphan Drug Profile in Clinical Phase



11. Orphan Drug Clinical Development Phase: Pre-Registration

  11.1 Overview

  11.2 Orphan Drug Profile in Clinical Phase



12. Orphan Drug Clinical Development Phase: Registered

  12.1 Overview

  12.2 Orphan Drug Profile in Clinical Phase



13. Marketed Orphan Drug

  13.1 Overview

  13.2 Marketed 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