OrphanDrugs Strategic Approaches for the Industry

Date: March 1, 2011
Pages: 52
Price:
US$ 1,215.00
Publisher: Espicom Business Intelligence
Report type: Strategic Report
Delivery: E-mail Delivery (PDF)
ID: OB2F00C1BF3EN
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OrphanDrugs Strategic Approaches for the Industry
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 from Espicom 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.

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
THE BUSINESS IMPLICATIONS

CHAPTER 1 - THE LANDSCAPE FOR ORPHAN DRUG DEVELOPMENT

Executive Summary
Introduction
  Rare diseases
    Definitions
    Prevalence
    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 advances
Government support
Patient advocates
Markets
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
  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
EXPERT COMMENTARY: Jörn Aldag, CEO, AMT BioPharma
  Company background
  Regulatory environment for orphan products
  Market access
  Market leaders
  Australian Orphan Drug Regulations
  Japanese Orphan Drug Regulations
  Harmonisation of US and EU regulation and advice
  Incentives for orphan drug development
  Conclusions

CHAPTER 3 - KEY PHARMA PLAYERS AND THEIR STRATEGIES

Executive Summary
  Leading biotech and specialty pharma companies in the orphan space
    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, Switzerland
    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
  Pipeline
  Patient organisations
  Gene therapy
  Cell therapies
  Therapeutic vaccines
  Large pharma companies in the orphan space
  Pfizer
  GlaxoSmithKline
  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
  Conclusions
  Appendix

CHAPTER 4 - CHALLENGES AND OPPORT UNITIES IN THE ORPHAN DRUG MARKET

Executive Summary
  Challenges in discovery and development of drugs for rare diseases
    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
    Outlook

FOOTNOTES

BIBLIOGRAPHY

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 diseases
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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