Are you ready for the regulatory changes to early access in France?

Changes to the existing ATU include two distinct pathways for drugs/therapies in clinical development and off-label including use in rare disease, that now include a commitment to collection of data.  By Heather Manna, Director of Regulatory Affairs, RareiTi The most recent French Social Security law of Decembe14, 2020 (Article 78- La Loi de financement de…

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Expanded Access – the Right Applies

Prompted by the current high-profile case of Lisa Stockman Mauriello’s devastating rare disease ALS diagnosis and her family’s fight to access an investigational therapy, tofersen, potentially the only option to alter the course of her disease, we comment on the issues surrounding this case and the possible ethical dilemma this presents whilst applying the long-established…

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