Orchard On The Trials, Tribulations And Triumphs Of Gene Therapy HTA

 
• By Eliza Slawther
  
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Convincing payers to fund a $3m therapy is no mean feat, even for innovations with curative potential. Orchard Therapeutics, a company with much experience in this field, tells the Pink Sheet why it values open dialogues, and outlines how health technology assessment methods could be improved.

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Companies must clear many hurdles on the road to market access • Source: Shutterstock

Orchard Therapeutics Limited is among the small handful of companies to have a gene therapy approved in the EU. Its ex-vivo autologous hematopoietic stem cell product Libmeldy (atidarsagene autotemcel) was granted an EU marketing authorization in December 2020 for use as a one-time treatment for children with early-onset metachromatic leukodystrophy (MLD), a rare inherited metabolic disease. (Also see "Advanced Therapies From Orchard & Kite Win EMA Thumbs Up" - Pink Sheet, 16 October, 2020.).

While it is often the regulatory nod that generates buzz for novel therapies, sponsors will know all too well that...

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