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Orphan Drugs Strategic Approaches for the Industry

  • Publication Date:March 2011
  • Publisher:Espicom
  • Product Type: Report
  • Pages:52
  • ISBN:978-1-85822-418-3

Orphan Drugs Strategic Approaches for the Industry

A complete and perceptive oversight of orphan drugs and their strategic potential is provided in this new report

With many companies facing the “patent cliff”, orphan drugs offer potentially high returns, lower development costs and less intense competition. So what are the issues affecting this sector at a time of challenge for the branded industry?

Orphan Drugs Snapshot
In recent years, there has been a surge of interest in this area as, despite the increasing number of products, the unmet medical needs of patients with rare diseases remain high. Scientific advances, regulatory and government support, active patient advocacy organisations and commercial factors all contribute to this increase in interest in rare diseases.

Estimates of the number of rare diseases range from approximately 5,700 to almost 7,000. These diseases affect an estimated 622 million people across the globe. Most rare diseases have genetic origins; others are caused by infection, including infectious diseases that are more common outside developed nations, toxic agents, or have other causes.

A complete and perceptive oversight of orphan drugs and their strategic potential is provided in this new report:

  • Market perspectives and trends – provides an analysis of the orphan drug market, detailing the drivers for recent growth, regulatory trends, and profiles of key companies and their pipelines
  • Regulatory landscape – provides an in-depth analysis of current regulations in major markets (US, EU, Australia and Japan)
  • Leading companies – summarises leading and emerging companies and their pipelines including a summary of innovative areas of research (antisense therapy, gene therapy, cellular therapy, therapeutic vaccines) in which orphan drugs play a key role in bringing new drugs to the market
  • Challenges – provides an analysis of the preclinical and clinical development challenges, as well as pricing and reimbursement issues, for orphan drugs
  • Outlook – summarises the future outlook for the orphan drug sector

This new strategy report will tell you...

  • What the definitions of orphan drugs are in different geographic regions
  • Why orphan drugs represent potentially attractive targets for drug development
  • Who the leading companies in orphan drug development are and why
  • What the regulatory landscape for orphan drug developments look like and how this affects future growth
  • How orphan drug development, pricing and reimbursement differ from more traditional drug development
  • Why the orphan drug sector has grown in recent years.

More Information...

Use this insightful report to...

  • Identify why the orphan drug sector is currently undergoing high levels of growth
  • Discuss the regulatory and legislative support for orphan drug development and its impact on growth within the sector
  • Learn which companies are well established within the orphan drug space, which are emerging and what their pipelines look like
  • Discover which companies are forming alliances with large pharma to help develop their orphan drug pipelines
  • Analyse the orphan drug market now and in the future"
CHAPTER 1 - The landscape for orphan drug development
Executive Summary
Rare diseases
Causes of rare diseases
Neglected diseases
Drugs for rare diseases
Orphan drug designations and approvals in the US
Orphan drug designations and approvals in the EU
Interest in developing drugs for rare diseases
Scientific advances8
Government support
Patient advocates10
Challenges in the development of drugs for rare diseases

CHAPTER 2 - the market environment: regulation, legislation and incentives for orphan
drug development
Executive Summary
Legislation in the US
EXPERT COMMENTARY: Dr Tim Coté, Director, FDA Office of Orphan Products Development ?14
Clinical data
Accelerating approval
Post-marketing approval commitments
Applying for orphan drug designation
Criticism of orphan drug legislation in the US
Regulation in other markets
EU Orphan Drug Regulations
Company background
Regulatory environment for orphan products
Market access18
Market leaders
Australian Orphan Drug Regulations
Japanese Orphan Drug Regulations
Harmonisation of US and EU regulation and advice
Incentives for orphan drug development

CHAPTER 3 - Key pharma players and their str ategies ?22
Executive Summary
Leading biotech and specialty pharma companies in the orphan space ?23
Genzyme, MA, US
Products and product pipeline
Manufacturing issues
The future for Genzyme
BioMarin, CA, US
Shire plc, UK
Swedish Orphan Biovitrum, Sweden
Actelion Pharmaceuticals Ltd, Switzerland26
Sigma-Tau Group, Italy
Other big biotech players with interests in orphan drugs
Celgene Corporation, NJ, US
Gilead Sciences, CA, US
Emerging biotech companies
Alexion Pharmaceuticals, CT, US
ViroPharma Inc, PA, US
InterMune Inc, CA, US
Protalix Biotherapeutics, Israel
Incyte Corporation, DE, US
Acorda Therapeutics, NY, US
Amicus Therapeutics, NJ, US
Santhera Pharmaceuticals, Switzerland
Innovative technology platforms
Antisense therapy
EXPERT COMMENTARY: Luc Dochez, Chief Business Officer, Prosensa ?
Company background
Research funding
Exon skipping in DMD
Patient organisations
Gene therapy
Cell therapies
Therapeutic vaccines
Large pharma companies in the orphan space36
Business models
EXPERT COMMENTARY: Paolo Bassanini, Investor & External Relations, mondoBIOTECH ?
mondoBIOTECH holding AG
Licensing activities
Working with regulators
Diagnosis of rare diseases
Interest in drugs for rare diseases

CHAPTER 4 - Challenges and opport unities in the orphan drug market ?
Executive Summary
Challenges in discovery and development of drugs for rare diseases ?43
Preclinical development of drugs for rare diseases
Clinical development of drugs for rare diseases
Challenges in trial design and conduct for rare diseases
Innovation in clinical trial design
Finding patients: registries and biobanks
Orphan drugs, diagnostics and personalised medicine
Pricing, market access and reimbursement
Reimbursement in the US
Reimbursement in the EU
Access to investigational products

the author

List of Tables
Table 1. Examples of rare diseases caused by defects in a single gene
Table 3. Examples of government funded projects to accelerate research into rare diseases
Table 4. Prevalence criteria for the definition of rare diseases in the United States, Australia, Japan and the European Union
Table 5. Incentives introduced for orphan drugs in the US, EU, Australia and Japan
Table 6. Genzyme’s key orphan products for rare diseases23
Table 7. BioMarin’s key products
Table 8. Shire’s key orphan products
Table 9. Actelion’s key orphan products
Table 10. Sigma-Tau’s key orphan products and pipeline
Table 11. M&A between biotech companies and their partners for drugs for rare diseases since 2008
Table 12. Examples of companies developing antisense therapies in orphan indications
Table 13. Examples of companies developing gene therapies in orphan indications
Table 14. Examples of companies developing stem cell-based therapies in orphan diseases
Table 15. Examples of companies developing therapeutic vaccines for rare cancers
Table 16. M&A between big pharma and partners for drugs for rare diseases since 2008
Table 17. List of companies receiving FDA orphan drug designations in 2010
Table 18. Examples of the annual costs for some orphan products

List of Charts
Figure 1. Prevalence of rare diseases in Europe
Figure 2. Orphan drug designations and approvals over the last 10 years
Figure 3. Proportions of small molecule and biologics receiving orphan drug approvals
Figure 4. Therapeutic areas for orphan medicinal product designations and approvals in the EU
Figure 5. Reasons for the increasing interest in drugs to treat rare diseases
Figure 6. Treatment alternatives for Gaucher disease in the US
Figure 7. FDA orphan drug designations and approvals for the largest pharma companies
Figure 8. Attributes of 55 pivotal clinical trials carried out for 30 drugs approved by the FDA between 2007 and 2009
Figure 9. Average times taken for orphan drug development and approval for 30 drugs approved in the US between 2007 and 2009
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