On 15 November, the UK’s Medicines and Healthcare products Regulatory Agency became the first regulator to issue a marketing authorization for a CRISPR/Cas9-based gene therapy, three years after its inventors secured a Nobel Prize for this revolutionary scientific development.
UK Authorizes Vertex’s Casgevy, The World’s First CRISPR/Cas9 Gene Therapy
Vertex’s Casgevy has made history as the world’s first approved CRISPR gene editing therapy after the UK’s MHRA authorized the medicine ahead of its US and European regulatory counterparts.
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Pharmaceutical companies are being encouraged to reach out to NICE in relation to its HTA Innovation Lab, which provides a sandbox environment in which the health technology assessment body can test new methods of evaluating “innovative and disruptive” therapies.
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Health technology assessment body NICE said it has taken on feedback about the implications of allowing higher cost-effectiveness thresholds for some medicines after senior health economists offered diverging views on its methods.
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Cell and gene therapy manufacturers must consider the practicalities of their product within the context of a health care system before it comes onto the market to be successful, experts from Novartis, AstraZeneca and England’s National Health Service say.
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The UK branded drug industry is calling for adjustments to be made to the UK voluntary scheme framework for branded medicines, after the payment rate increased to 22.9% for 2025. Meanwhile, the generics industry says the scheme is functioning “as intended” and opposes any changes.
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Eli Lilly’ will request a re-examination after the European Medicines Agency declined to recommend its Alzheimer’s disease drug Kisunla for EU approval.
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The new global GCP guideline, ICH E6(R3), enables researchers and clinical trial administrators to tailor their documentation processes, but also opens the door for more scrutiny during GCP inspections.
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A German ordinance implementing the EU Health Technology Assessment Regulation offers little clarity on how far joint clinical assessment reports should be considered by national authorities.