Category Archives: Orphan Drugs

Orphan Drug Market to Reach $176 Billion by 2020, Says Report

By 2020, orphan drugs are expected to account for 19 percent of the total share of prescription drug sales excluding generics, reaching $176 billion in annual sales, according to EvaluatePharma’s Orphan Drug Report 2014. At 11 percent, this growth is set to be more than double the 5 percent growth rate of the overall prescription market, […]
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Investment Pros Remain Bullish on Orphans

 An illustrious panel of investment professionals spoke optimistically on the orphan disease space in spite of concerns surrounding escalating drug prices in specialty pharmaceuticals. The group consisting of executives from investment banks, hedge funds, venture capital firms and biopharma companies spoke to the National Organization for Rare Disease (NORD) at its s Rare Diseases and […]
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Expanded Access: Myths, Truth and Behavior

Not all companies are big, small companies are scared, and myths drive behavior, stated Kay Holcombe, Senior Vice President, Science Policy at Biotechnology Industry Organization (BIO).
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NIH Translational Research Partnership Yields Promising Therapy

A potential treatment for sickle cell disease has come through the “valley of death” of early stage development due to support from a collaborative partnership established by the National Center for Advancing Translational Sciences (NCATS) at the National Institutes of Health (NIH). The results of early clinical trials demonstrated sufficient effectiveness in addressing the underlying […]
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UK: Patient Group Sets Out Orphan Drug Charter

By Leela Barham. There has been a lot of concern about the decision to give the National Institute for Health and Care Excellence (NICE) the responsibility to look at “highly specialized technologies” (HSTs, or orphan drugs to you and me). That decision was taken just as the NHS across England was going through major reform […]
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