Orphan Drugs: Strategic Approaches for the Industry

Published Date: 31/03/2011
Category: Therapies & Markets

Research Report Decription

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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.

Key Questions Answered:

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.

Companies Mentioned:

Actelion Pharmaceuticals Acorda Therapeutics, Amicus Therapeutics, AMT BioPharma, Alexion Pharmaceuticals, BioMarin, Celgene Corporation, GlaxoSmithKline, Genzyme, Incyte Corporation, InterMune, mondoBIOTECH holding AG, Protalix Biotherapeutics, Prosensa, Pfizer, Sigma-Tau Group, Gilead Sciences, Santhera Pharmaceuticals, Shire, Swedish Orphan Biovitrum, ViroPharma

Paper Highlights:

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