Category Archives: Orphan Drugs

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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Eight Ingredients for a Successful Oncology Drug Development Recipe

by Matthew Cook and Sebastien Morisot In light of the risks and challenges inherent in the development of oncologics, some biopharma companies are beginning to question whether oncology remains an attractive therapeutic area in which to invest limited research, development, and commercialization resources.
Also posted in Agency Insight, Biotech, Europe, FDA, Gene therapy, Guest Blog, leadership, Market Access, pricing, R&D, Regulatory, Sales | Tagged , , | Leave a comment

Pharm Exec's Brand of the Year Applauds the Old and the New

Pharm Exec’s Brand of the Year award is itself a brand with staying power — our first recipient was Merck’s Gardasil papillomavirus vaccine, which eight years later is still going strong as one of the company’s 10 top-selling products.
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Europe Approves First Medicine for Castleman's Disease

The European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has recommended marketing authorization for Sylvant (siltuximab) for adult patients with multicentric Castleman’s disease.
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