Europe Orphan Drug Market & Clinical Trial Insight 2015

Publish Date:- Jan-2015      No Of Pages (1100)

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The concept of rare diseases and the idea that a special attention needs to be given to this sector has been taking shape in Europe since the 1990s. ever since then the political aspects and initiatives related to orphan medicinal products have been emerging at both the EU level and at the level of member states individually. Many member states in the EU have led the way during the 1990s in the space of orphan medicinal products, the result of which took the shape of the first European legislative text concerning rare diseases called the Orphan Medicinal Product Regulation. This Regulation was adopted in 1999 and came into effect in 2000.

Europe is the second largest market for orphan drugs. In the European region, rare disease is considered to be a priority area and research in this field is given high importance by the EU Framework Programmes for Research and Technological Development (FP) ever since the early 1990s. Increasing the utilization of scarce resources and coordinating research efforts are the basic factors which have been responsible for success in the European rare diseases market. However, the absence of an exhaustive rare disease classification, standard terms of reference and a harmonized regulatory requirement, has always been a challenge in this region, which is currently impacting the global sharing of information, data and samples which would boost the research further.

It has been estimated that Europe has witnessed more than 100 therapies for rare diseases being approved since 2000, when the European Commission introduced incentives to encourage the development of these drugs. The share of R&D for orphan medicinal products development as a proportion of total biopharmaceutical industry’s R&D has been increasing significantly over the years. This is strengthened by the fact that almost all companies which have been set up recently to develop orphan medicinal products have their extensive R&D plants and staff located in the European Union. This shows the significance of the region and its level of attractiveness.

Additionally, the investment in R&D by the European companies for developing orphan medicinal products has increased by more than 200%, while the total number of employees in these companies has recorded an increase of more than 150% since 2000. With the long durations of lead times in the biopharmaceuticals industry for R&D projects, it is most likely that the EU Regulation for Orphan medicinal products would have a significant impact in the coming years.

  

“Europe Orphan Drug Market & Clinical Trial Insight 2015” Report Highlights & Findings:

  • Europe Orphan Drug Market Overview
  • In-depth Insight on Regulatory Framework & for Orphan Drugs
  • Application Procedure for Seeking Orphan Drug Status
  • Comprehensive Insight on Orphan Drug Clinical Pipeline
  • Europe Orphan Drug Clinical Pipeline: 326 Drugs
  • Majority Orphan Designated Drugs in Phase-II : 111
  • Marketed Orphan Drugs in Europe: 130


The concept of rare diseases and the idea that a special attention needs to be given to this sector has been taking shape in Europe since the 1990s. ever since then the political aspects and initiatives related to orphan medicinal products have been emerging at both the EU level and at the level of member states individually. Many member states in the EU have led the way during the 1990s in the space of orphan medicinal products, the result of which took the shape of the first European legislative text concerning rare diseases called the Orphan Medicinal Product Regulation. This Regulation was adopted in 1999 and came into effect in 2000.

Europe is the second largest market for orphan drugs. In the European region, rare disease is considered to be a priority area and research in this field is given high importance by the EU Framework Programmes for Research and Technological Development (FP) ever since the early 1990s. Increasing the utilization of scarce resources and coordinating research efforts are the basic factors which have been responsible for success in the European rare diseases market. However, the absence of an exhaustive rare disease classification, standard terms of reference and a harmonized regulatory requirement, has always been a challenge in this region, which is currently impacting the global sharing of information, data and samples which would boost the research further.

It has been estimated that Europe has witnessed more than 100 therapies for rare diseases being approved since 2000, when the European Commission introduced incentives to encourage the development of these drugs. The share of R&D for orphan medicinal products development as a proportion of total biopharmaceutical industry’s R&D has been increasing significantly over the years. This is strengthened by the fact that almost all companies which have been set up recently to develop orphan medicinal products have their extensive R&D plants and staff located in the European Union. This shows the significance of the region and its level of attractiveness.

Additionally, the investment in R&D by the European companies for developing orphan medicinal products has increased by more than 200%, while the total number of employees in these companies has recorded an increase of more than 150% since 2000. With the long durations of lead times in the biopharmaceuticals industry for R&D projects, it is most likely that the EU Regulation for Orphan medicinal products would have a significant impact in the coming years.

  

“Europe Orphan Drug Market & Clinical Trial Insight 2015” Report Highlights & Findings:

  • Europe Orphan Drug Market Overview
  • In-depth Insight on Regulatory Framework & for Orphan Drugs
  • Application Procedure for Seeking Orphan Drug Status
  • Comprehensive Insight on Orphan Drug Clinical Pipeline
  • Europe Orphan Drug Clinical Pipeline: 326 Drugs
  • Majority Orphan Designated Drugs in Phase-II : 111
  • Marketed Orphan Drugs in Europe: 130

1. What Are Orphan Drugs?

  1.1 Introduction to Orphan Drug

  1.2 Favorable Parameters for Orphan Drugs Acceptance

 

2. Europe Orphan Drug Designation Criteria

  2.1 Designation Criteria

  2.2 Sponsors Application Procedure

 

3. Europe Orphan Drug Reimbursement Policy

 

4. Europe Orphan Drug Market Overview

  4.1 Current Market Scenario

  4.2 Europe Orphan Drug Clinical Trial Insight

 

5. Europe Regulatory Framework for Orphan Drugs

  5.1 Committee for Orphan Medicinal Products

  5.2 How to Apply for Orphan Designation in Europe

  5.3 Marketing Authorization & Market Exclusivity

  5.4 Transferring An Orphan Designation To Another Sponsor

  5.5 Mandatory Submission Of Annual Report On Development

  5.6 Incentives For Micro, Small And Medium-Sized Enterprises

  5.7 Fee Reductions For Designated Orphan Medicinal Products

  5.8 Procedure for Orphan Designation & Incentives for R&D (Regulation (EC) No 141/2000)

 

6. Europe Orphan Drug Pipeline by Country, Company, Indication & Phase

  6.1 Research

  6.2 Preclinical

  6.3 Clinical

  6.4 Phase-I

  6.5 Phase-I/II

  6.6 Phase-II

  6.7 Phase-II/III

  6.8 Phase-III

  6.9 Preregistration

  6.10 Registered

 

7. Marketed Orphan Drug in Europe by Country, Company & Indication

 

8. Discontinued & Suspended Orphan Drug Clinical Pipeline

  8.1 No Development Reported

  8.2 Discontinued

  8.3 Market Withdrawal

  8.4 Suspended

 

9. Competitive Landscape

  9.1 AOP Orphan

  9.2 Genethon

  9.3 Genzyme Corporation

  9.4 Glaxosmithkline

  9.5 Merck

  9.6 Novartis Pharmaceuticals

  9.7 Orphan Europe

  9.8 Pfizer

  9.9 Prosensa

  9.10 Shire

  9.11 Teva Pharmaceutical


Figure 4-1: Europe Orphan Drug Market (US$ Billion), 2012-2018

Figure 4-2: Europe Share in Global Orphan Drug Market, 2012 & 2018

Figure 4-3: Biological & Non Biological Orphan Drug Segment (%), 2012 & 2018

Figure 4-4: Biological & Non Biological Orphan Drug Market (US$ Billion), 2012-2018

Figure 4-5: Europe Orphan Pipeline by Phase (%), 2014

Figure 4-6: Europe Orphan Pipeline by Phase (Number), 2014

Figure 4-7: No Development Reported Europe Orphan Pipeline by Phase (%), 2014

Figure 4-8: No Development Reported Europe Orphan Pipeline by Phase (Number), 2014

Figure 4-9: Discontinued Europe Orphan Pipeline by Phase (%), 2014

Figure 4-10: Discontinued Europe Orphan Pipeline by Phase (Number), 2014

Figure 4-11: Suspended Europe Orphan Pipeline by Phase (%), 2014

Figure 4-12: Suspended Europe Orphan Pipeline by Phase (Number), 2014

Figure 9-1: AOP Orphan

Figure 9-2: Genethon Drug Pipeline

Figure 9-3: Genzyme Drug Pipeline

Figure 9-4: Orphan Europe Pipeline

Figure 9-5: Prosensa Drug Pipeline

Figure 9-6: Shire Drug Pipeline