Orphan Drugs: Market Environment Products and Companies

NEW YORK, Jan. 3, 2013 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:

http://www.reportlinker.com/p01075850/Orphan-Drugs-Market-Environment-Products-and-Companies .html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Drug_and_Medication

For the pharma and diagnostic industries, rare diseases can provide significant commercial success. Examples of blockbuster orphan drugs include Novartis' leukaemia drug, Glivec/Gleevec (imatinib), with global sales of US$4,695 million in 2011; and Actelion's Tracleer (bosentan), the current gold standard in pulmonary arterial hypertension with sales of CHF1,522 million (US$1,598 million) in 2011.

The combination of government incentives to develop drugs for rare diseases and the promise of commercial opportunity will continue to fuel the industry's interest in orphan drugs; and provide hope to patients with debilitating conditions and high unmet medical needs. In addition, the fast track approval system allows companies to enter the market faster and more cost-effectively with the potential to extend indications.

Significant unmet clinical need

While orphan diseases are rare, patients with rare diseases are numerous. There are up to 7,000 identifiable rare diseases, affecting an estimated 622 million people around the world. There is a clear need to provide rare disease patients with the same quality of care as other patients, and governments have provided incentives to companies developing orphan drugs to address a significant public health need. As effective diagnostics play a greater role in accurately diagnosing orphan diseases, the addressable patient population will expand.

One of the biggest challenges for rare disease patients is obtaining the correct diagnosis, which can be extremely difficult and can take years, or even decades. An increasing number of companies developing drugs for rare diseases are working with diagnostic companies, or their own diagnostic divisions, to establish biomarkers and develop companion diagnostics for patient selection. Companion diagnostics can assist with identifying patients for clinical trials of a novel drug, and may be approved simultaneously as part of a conditional drug approval.

A comprehensive overview of orphan drugs and their strategic importance is provided in this valuable new report

Market conditions provides an analysis of the orphan drug market, detailing the drivers for recent growth, prevalence and scientific advances

Regulatory landscape provides an in-depth analysis of current regulations in major markets (US, EU, Australia and Japan) along with pricing and reimbursement information and orphan drug approvals

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Orphan Drugs: Market Environment Products and Companies