Tag Archives: Orphan Drugs

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 […]
Posted in Europe, Global, Guest Blog, healthcare, Op-Ed, Orphan Drugs, Patient Communication, patient education, pricing, Regulatory | Also tagged , , , , , , , | Leave a comment

PharmExec's Orphan Drug Pipeline Picks

Orphan drugs were given short shrift in PharmExec’s 2014 Pipeline Report, but a couple of pipeline candidates targeting small populations did make the list, and are expected to earn big dollars in the next few years. In the US, private and government payers haven’t yet had the nerve to balk at rare disease drug prices. […]
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The List So Far: FDA Breakthrough Designations

With announcements pouring in since the beginning of this year, FDA’s fourth and newest expedited approval pathway for medicines has begun to bear fruit. Established in 2012 within the FDA Safety and Innovation Act (FDASIA), Breakthrough Therapy designations have so far totaled 25 as of the end of July. With a separate senior management team […]
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Orphan Drug Laws: A Pharmacist's Perspective

Orphan Drug Laws:  A Pharmacists’ Perspective Guest blogger Dr. Albert Wertheimer Professor and Director, Health Services Research Temple University Philadelphia PA Pharm Exec’s sister organization CBI hosted its annual Orphan Drug Innovation Summit in Philadelphia on July 17-18.  This is a topic that I have professional interest in, as well as a general curiosity.  I […]
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The Orphan Drug Act at 30 Years: What's Next?

30 years ago last week, the US Orphan Drug Act came into being, and with it, a door of possibilities opened up for patients with literally thousands of untreated diseases. The anniversary marks the beginning of a journey where medicines for rare diseases have gained a foothold in the portfolios of drug companies large and […]
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