GlaxoSmithKline made headlines when it got approval for its gene therapy Strimvelis in Europe, but due to a small patient population and high price tag, the drug has only been used once. So far, despite higher levels of safety and efficacy than previous iterations, the new wave of gene therapies still face commercial hurdles.
Spark Therapeutics is now the next company to take on this challenge. The biotech is currently awaiting approval of its treatment for a rare genetic form of blindness — a potential one-time cure. But pricing is going to be the most closely watched aspect of this drug.
In our latest special edition we are exploring the pricing and commercialization conundrum, as well as taking a closer look at how this field has evolved over the last two decades.
We hope you enjoy reading and please provide us with any feedback. You can reach us at BioPharma Dive by Twitter or via email.
Lisa LaMotta
Editor, BioPharma Dive
E-mail
News and Trends
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Feature Story
After decades of setbacks, a slew of next-gen gene therapies are ready to hit the U.S. market, prompting questions about manufacturing and pricing.
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Feature Story
A pricing conundrum and ethical decisions are clouding an already hazy path to market for many gene therapy drugs and providers.
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At the forefront of drug discovery and development, small to midsize biotech/biopharma companies are the leaders of innovation.
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Feature Story
With an approval of Spark Therapeutics' gene therapy for a rare eye disease rapidly approaching, new questions about pricing are being raised.
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Feature Story
In a field shaped by small patient populations and eye-popping cost considerations, understanding gene therapy's promise and challenges comes down, in part, to the numbers.
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While many are optimistic about gene editing's ability to cure disease, it seems not enough realize the more dangerous aspects of treatment.
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Featured Resources
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Meeting patient engagement goals in gene therapy clinical trials is the pinnacle of recruiting success. Sally Amanuel, Sr. VP Clinical Study Start-up and Regulatory Affairs at Worldwide Clinical Trials, shows how to build strategies for cross-border patient transfer in rare diseases. Watch how →
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Gene therapy is one of the most promising innovations in clinical care. Yet its value is under greater scrutiny today. Worldwide Clinical Trials developed a blueprint for clarifying the real world value of gene therapies. Start building now →
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Value-based assessments are increasingly vital for market access to new drugs. Yet do revolutionary gene therapies really need them? Reflecting on the first marketable gene therapy, Worldwide Clinical Trials Chief Medical Officer Michael Murphy, MD, PhD, has answers. Watch now →
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What We're Reading
The New York Times
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MIT Technology Review
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Science Daily
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