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

 
• By Eliza Slawther

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.

hurdle
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 this is the first of many hurdles on the road to patient access – particularly in the EU, where national health technology assessment (HTA) bodies and payer budgets are

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