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Drug Discovery in Rare Diseases
Date:9/20/2012

NEW YORK, Sept. 20, 2012 /PRNewswire/ -- Reportlinker.com announces that a new market research report is available in its catalogue:

 

Drug Discovery in Rare Diseases

http://www.reportlinker.com/p0968548/Drug-Discovery-in-Rare-Diseases.html#utm_source=prnewswire&utm_medium=pr&utm_campaign=Drug_Discovery_and_Development

 

In comparison with major diseases, the targeting of rare diseases poses many different challenges, necessitating consideration of bespoke R&D strategies for drug discovery efforts to be successful. This report examines the role that low disease prevalence plays in determining the most suitable R&D path.Understand the growing interest in developing new treatments for rare diseases, and why low patient numbers do not preclude commercial viability.Review the regulatory environment governing the development of orphan drugs in different countries.Identify the key challenges that are presented by low patient prevalences.Assess how the research strategy chosen can be influenced by the disease prevalence.Compare the scenarios in which repurposing of existing drugs offers advantages over the development of novel drugs, and vice versa.Approximately 7,000 rare diseases have been identified, but only a very small proportion of these are currently well treated. Orphans represent a greater proportion of all new BLAs than they do of NMEs submitted as NDAs. Most orphan approvals are not first approvals of new drugs but are new orphan indications for previously approved drugs.It is possible to obtain orphan drug designation for conditions with a total prevalence greater than that defined by legislation, but only if medically justifiable subsets can be defined with a lower (overall) prevalence. Pediatric subsets are most commonly u
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Related medicine technology :

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3. Outsourcing in Drug Discovery: The Contract Research Organization (CRO) Market, 5th Edition
4. AstraZeneca And The Broad Institute Partner To Advance Discovery Of Antibacterial And Antiviral Agents
5. Drug Discovery Technologies, BCC Research
6. Antigen Discovery Inc. Awarded Phase I SBIR Grant to Develop a Fully Integrated Microfluidic Point of Care Diagnostic Device
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9. Antigen Discovery, Inc. Awarded Phase II SBIR Grant to Identify Biomarkers Associated with Protection against Malaria
10. Omeros Announces Discovery in MASP-2 Program
11. Discovery Labs Receives US Patent for Method to Promote Mucus Clearance with Pulmonary Surfactant
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