Posted On: 09/21/2016 11:15:37 AM
Post# of 72447
Some interesting stuff in this interview with SRPT's CEO, like
Hmm. Is there any therapy on the market for oral mucositis? What about that awful skin disease HS that causes those abscess tunnels in people's underarms? What about Stage 4 cancer patients whose only hope now might be turning on a cancer-killer gene? hmmmm......
Quote:
BA: It really seems like there’s a new willingness to get these drugs on the market come hell or high water; the FDA and now even NICE appear more flexible than ever before. Is 2016 the year we can say we turned a corner on the regulatory and pricing watchdog side of things in rare disease, where both patients and patients’ groups as well as the biotech/pharmas spending billions on developing medicines are taken ever more into account, rather than just the data handed in for review?
EK: I hope so, because if you think about the FDA, the EMA and the companies, and then you think of the physicians who are like-minded and want to get a therapy onto the market where there are none, generally we all want the same thing.
Hmm. Is there any therapy on the market for oral mucositis? What about that awful skin disease HS that causes those abscess tunnels in people's underarms? What about Stage 4 cancer patients whose only hope now might be turning on a cancer-killer gene? hmmmm......
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