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Ann: Rett Syndrome NDA accepted for Priority Review by FDA, page-24

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    Be it here in Australia, or in the US, doctors are the ones who make decisions on prescriptions, based on their professional judgement. This includes off-label prescription.

    However, it is not doctors who are the main barrier to off-label access; it is the payers, especially in the case of high-priced drugs. Most insurance companies will not provide reimbursement for high-priced drugs in non-approved indications.

    While we don’t know how trofinetide will be priced, it is likely to be unaffordable for most people if it’s not covered by insurance.
 
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