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Re: Amatuer17 post# 81192

Monday, 11/28/2016 3:30:43 PM

Monday, November 28, 2016 3:30:43 PM

Post# of 459486
If the Cures Act passes (the House votes on it Wednesday), AVXL 2-73 for Rett may have faster approval. Here are some key provisions:

1. Authorizes innovation prize competitions to advance biomedical science for diseases that are serious and represent a significant burden. (Section 2002)
2. Provides opportunities for new researchers and promotes earlier research independence. (Section 2021)
3. Supports high-risk, high-reward research that has the potential to transform the scientific field. (Section 2036)
4. Expedites the development and review of drugs for subgroups of rare disease patients by authorizing FDA to rely on data submitted in previously approved applications involving similar gene targeting technology. (Section 3012)
5.Reauthorizes the pediatric priority review voucher program incentivizing the development of drugs for rare pediatric diseases. (Section 3013)

If the FDA approves the drug for Rett, physicians may then write prescriptions off label.
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