Next-gen drugs: A balancing act

GlaxoSmithKline, the world’s sixth largest pharmaceutical company, have sold their rare disease gene therapy portfolio to Orchard Therapeutics due to doubts in the product line’s profitability.

One of these therapies is Strimvelis which treats ‘bubble baby’ disease – an ultra-rare form of severe combined immunodeficiency (SCID) in newborn infants. A single treatment of Strimvelis costs parents US$735,000 out of pocket. Pharma firms, like GSK, are often unable to reach economies of scale required for a sustainable business model as only a few dozen children are diagnosed every year with SCID and even fewer have parents who can afford the cure. Only a handful of patients have been treated since the launch of Strimvelis in 2016.

Rare disease gene therapy treatments are so-called ‘one-and-done’ courses with a remarkably different customer relationship compared to ongoing pharmaceutical dispensing patterns. Orchard Therapeutics CEO Mark Rothera said, ‘I think society has to find a balance between the extraordinary value we are bringing and a sustainable business model. There is a balancing act.’

Read more: Reuters / MIT Tech Review

Image: SkitterPhoto / CC0-1.0 – Single pink pill

This story is taken from the 20 April 2018 edition of The Warren Centre’s Prototype newsletter. Sign up for the Prototype here.