Global Orphan Drug Pipeline & Regulatory Insight 2015

Publish Date:- Jan-2015      No Of Pages (2200)

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Rare diseases are also known as Orphan diseases. These diseases are generally categorized as chronic, degenerative, life threatening in nature and normally include certain cancers, metabolic conditions, diseases of the nervous system and musculoskeletal disorders. As per the official definition, rare diseases are classified as those that affect fewer than 200,000 in the United States and less than 5 in 10,000 in the EU. These two regions form a dominant portion of the global market for orphan drugs.

In Japan, any disease with less than 50,000 prevalent cases is defined as a rare disease. In Australia, the perspective is different. The Therapeutic Substances Regulations in Australia defines rare drugs as those which should not be intended for use in more than 2000 patients annually. In Taiwan, the official definition of rare diseases is that these diseases would be termed “rare” if the prevalence rate is 1:10,000 people. For drugs to get an orphan designation in Korea, less than 20,000 people in Korea should suffer from the disease/condition, or no other alternative treatment should be available for the disease in the country.

Owing to the pharmaceutical companies’ inadequate interest in this category of drugs, the term “orphan” was coined for drugs targeted to treat rare diseases. Almost 222 orphan designated drugs in US and 132 in Europe are marketed till date. However, in spite of the increased efforts in this market, there is still a shortage in the number of treatments for most rare disease indications. Thus, the market for rare diseases in the United States, Europe and other regions continue to offer significant potential for growth owing to enormous unmet medical need.

Globally, there was a desire to address the unmet treatment needs of orphan diseases, which transformed into the 1983 U.S. Orphan Drug Act, as well as similar Acts in 1991 in Singapore, 1993 in Japan, 1997 in Australia and in 2000 by the European Union.  The introduction of these acts, along with high-profile philanthropic funding, has made orphan diseases an attractive segment and encouraged investment in R&D for a number of these destructive conditions.

Historically, the development of 70-75% of orphan drugs was mostly in the realm of smaller biotechnology and specialty pharmaceutical companies, and the remaining 25-30% of the orphan drugs were developed by the pharma giants. However, in the past decade, the share of pharma giants in the orphan drug approvals has increased to close to 40-45%.

The US and European region are the major markets for orphan drugs. It is in these two regions that the popularity of these drugs and diseases is increasing rapidly. The US alone accounts for a dominating share of 55%-60% in the global pie, followed by Europe. As the Asian pharmaceutical markets are still in nascent stage, there opportunities for orphan drugs in Asia are also immense and getting increasingly popular. It is expected that by 2017-18, the Asian orphan drugs market would witness steady growth.

 

“Global Orphan Drug Pipeline & Regulatory Insight 2015” Report Highlights & Findings:

  • Global Orphan Drug Market Overview
  • In-depth Insight on Regulatory Framework & for Orphan Drugs by Region
  • Orphan Drug Designation Criteria & Reimbursement Policy by Region
  • Comprehensive Insight on Global Orphan Drug Clinical Pipeline
  • Europe Orphan Drug Clinical Pipeline: 697 Drugs
  • Majority Orphan Designated Drugs in Phase-II : 249
  • Globally Marketed Orphan Drugs: 274


Rare diseases are also known as Orphan diseases. These diseases are generally categorized as chronic, degenerative, life threatening in nature and normally include certain cancers, metabolic conditions, diseases of the nervous system and musculoskeletal disorders. As per the official definition, rare diseases are classified as those that affect fewer than 200,000 in the United States and less than 5 in 10,000 in the EU. These two regions form a dominant portion of the global market for orphan drugs.

In Japan, any disease with less than 50,000 prevalent cases is defined as a rare disease. In Australia, the perspective is different. The Therapeutic Substances Regulations in Australia defines rare drugs as those which should not be intended for use in more than 2000 patients annually. In Taiwan, the official definition of rare diseases is that these diseases would be termed “rare” if the prevalence rate is 1:10,000 people. For drugs to get an orphan designation in Korea, less than 20,000 people in Korea should suffer from the disease/condition, or no other alternative treatment should be available for the disease in the country.

Owing to the pharmaceutical companies’ inadequate interest in this category of drugs, the term “orphan” was coined for drugs targeted to treat rare diseases. Almost 222 orphan designated drugs in US and 132 in Europe are marketed till date. However, in spite of the increased efforts in this market, there is still a shortage in the number of treatments for most rare disease indications. Thus, the market for rare diseases in the United States, Europe and other regions continue to offer significant potential for growth owing to enormous unmet medical need.

Globally, there was a desire to address the unmet treatment needs of orphan diseases, which transformed into the 1983 U.S. Orphan Drug Act, as well as similar Acts in 1991 in Singapore, 1993 in Japan, 1997 in Australia and in 2000 by the European Union.  The introduction of these acts, along with high-profile philanthropic funding, has made orphan diseases an attractive segment and encouraged investment in R&D for a number of these destructive conditions.

Historically, the development of 70-75% of orphan drugs was mostly in the realm of smaller biotechnology and specialty pharmaceutical companies, and the remaining 25-30% of the orphan drugs were developed by the pharma giants. However, in the past decade, the share of pharma giants in the orphan drug approvals has increased to close to 40-45%.

The US and European region are the major markets for orphan drugs. It is in these two regions that the popularity of these drugs and diseases is increasing rapidly. The US alone accounts for a dominating share of 55%-60% in the global pie, followed by Europe. As the Asian pharmaceutical markets are still in nascent stage, there opportunities for orphan drugs in Asia are also immense and getting increasingly popular. It is expected that by 2017-18, the Asian orphan drugs market would witness steady growth.

 

“Global Orphan Drug Pipeline & Regulatory Insight 2015” Report Highlights & Findings:

  • Global Orphan Drug Market Overview
  • In-depth Insight on Regulatory Framework & for Orphan Drugs by Region
  • Orphan Drug Designation Criteria & Reimbursement Policy by Region
  • Comprehensive Insight on Global Orphan Drug Clinical Pipeline
  • Europe Orphan Drug Clinical Pipeline: 697 Drugs
  • Majority Orphan Designated Drugs in Phase-II : 249
  • Globally Marketed Orphan Drugs: 274

1. What Are Orphan Drugs?

  1.1 Introduction To Orphan Drug

  1.2 Favorable Parameters For Orphan Drugs Acceptance

  1.3 Economic Viability Of Orphan Drugs

  1.4 Patent & Market Exclusivity Advantage

 

2. Global Orphan Drug Market Outlook

  2.1 Market Overview

    2.1.1 By Biological & Non-Biological Orphan Drugs

    2.1.2 By Therapeutic Segmentation

    2.1.3 By Regional Segmentation

  2.2 Global Orphan Drug Clinical Pipeline Overview

 

3. Orphan Drug Designation Criteria

  3.1 US Orphan Drug Designation Criteria

  3.2 Europe Orphan Drug Designation Criteria

 

4. Reimbursement Policy For Orphan Drug

  4.1 US Orphan Drug Reimbursement Policy

  4.2 Europe Orphan Drug Reimbursement Policy

 

5. US (FDA) Regulatory Framework For Orphan Drugs

  5.1 Content & Format Of A Request For Written Recommendations

  5.2 Provision For Granting & Refusing Written Recommendations

  5.3 Content And Format Of A Request For Orphan Drug Designation

  5.4 Verification Of Orphan Drug Status & Resident Agent For Foreign Sponsor

  5.5 Timing Of Requests For Orphan Drug Designation & Designation Of Already Approved Drugs

  5.6 Deficiency Letters And Granting Orphan Drug Designation

  5.7 Refusal To Grant Orphan Drug Designation

  5.8 Amendment & Change In Ownership To Orphan Drug Designation

  5.9 Publication & Revocation Of Orphan Drug Designations

  5.10 Annual Reports Of Holder Of Orphan Drug Designation

  5.11 Scope & FDA Recognition Of Orphan Drug Exclusive Approval

  5.12 Protocols for Investigations & Availability of Information

 

6. Europe (EMA) Regulatory Framework For Orphan Drugs

  6.1 Committee for Orphan Medicinal Products

  6.2 How to Apply for Orphan Designation in Europe

  6.3 Marketing Authorization & Market Exclusivity

  6.4 Transferring An Orphan Designation To Another Sponsor

  6.5 Mandatory Submission Of Annual Report On Development

  6.6 Incentives For Micro, Small And Medium-Sized Enterprises

  6.7 Fee Reductions For Designated Orphan Medicinal Products

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

 

7. Asia Regulatory Framework For Orphan Drugs

  7.1 Taiwan Rare Disease & Orphan Drug Act

  7.2 Japan Orphan Drug Regulation

 

8. Global Orphan Drug Pipeline by Company, Country, Indication & Phase

  8.1 Unknown

  8.2 Research

  8.3 Preclinical

  8.4 Clinical

  8.5 Phase-I

  8.6 Phase-I/II

  8.7 Phase-II

  8.8 Phase-II/III

  8.9 Phase-III

  8.10 Preregistration

  8.11 Registered

 

9. Marketed Orphan Drug by Company, Country & Indication

 

10. Discontinued & Suspended Orphan Drugs in Clinical Trials

  10.1 No Development Reported

  10.2 Discontinued

  10.3 Market Withdrawal

  10.4 Suspended

 

11. Competitive Landscape

  11.1 AOP Orphan

  11.2 Agenus

  11.3 Alexion

  11.4 Bristol Myers Squibb

  11.5 Biogen Idec

  11.6 Celgene

  11.7 Eli Lilly

  11.8 Genethon

  11.9 Genzyme Corporation

  11.10 Glaxosmithkline

  11.11 Merck

  11.12 Novartis Pharmaceuticals

  11.13 Orphan Europe

  11.14 Pfizer

  11.15 Prosensa

  11.16 Rare Disease Therapeutics

  11.17 Roche

  11.18 Sanofi

  11.19 Shire

  11.20 Teva Pharmaceutical


Figure 1-1: Orphan v/s Non-Orphan Drugs -Phase II to Launch Clinical Development Time

Figure 1-2: Probability of Regulatory Success of Orphan v/s Non-Orphan Drugs

 

Figure 2-1: Global Orphan Drugs Market Value (US$ Billion), 2013-2018

Figure 2-2: US Orphan Drugs Market Value (US$ Billion), 2013-2018

Figure 2-3: Europe Orphan Drugs Market Value (US$ Billion), 2013-2018

Figure 2-4: Asia* Orphan Drugs Market Value (US$ Billion), 2013-2018

Figure 2-5: Biological & Non Biological Orphan Drug Segment (%), 2014 & 2018

Figure 2-6: Biological & Non Biological Orphan Drug Market (US$ Billion), 2013-2018

Figure 2-7: Global Orphan Drugs Market by Therapeutic Area, 2012 & 2018

Figure 2-8: Regional Markets for Orphan Drugs, 2014 & 2018

 

Figure 2-9: Global Orphan Drug Pipeline by Phase (%), 2015

Figure 2-10: Global Orphan Drug Pipeline by Phase (Numbers), 2015

Figure 2-11: No Development Reported Global Orphan Drug Pipeline by Phase (%), 2015

Figure 2-12: No Development Reported Global Orphan Drug Pipeline by Phase (Numbers), 2015

Figure 2-13: Discontinued Global Orphan Drug Pipeline by Phase (%), 2015

Figure 2-14: Discontinued Global Orphan Drug Pipeline by Phase (Numbers), 2015

Figure 2-15: Suspended Global Orphan Drug Pipeline by Phase (%), 2015

Figure 2-16: Suspended Global Orphan Drug Pipeline by Phase (Numbers), 2015

 

Figure 7-1: Japan Orphan Drug/Medical Device Designation System

Figure 7-2: Japan Orphan Drug/Device Designation Process

 

Figure 11-1: AOP Orphan

Figure 11-2: Agenus Pipeline

Figure 11-3: Genethon Drug Pipeline

Figure 11-4: Genzyme Drug Pipeline

Figure 11-5: Orphan Europe Pipeline

Figure 11-6: Prosensa Drug Pipeline

Figure 11-7: Shire Drug Pipeline

 

 

Table 1-1: Orphan Drugs in the US and their Cost    

Table 1-2: Orphan Drugs in the EU and their Cost