Will cell and gene therapies disrupt reimbursement?

Cell and gene therapies could prove disruptive in terms of the life-changing benefits they could bring to people with rare diseases and cancers – but they could also mean healthcare systems will have to change the way they pay for therapies. Richard Staines spoke to Andrew Powaleny, director of public affairs at US trade body PhRMA to find out more.

It’s more than a year ago since Spark Therapeutics launched its revolutionary gene therapy Luxturna, a one-shot treatment that can hugely improve sight for people with a rare disease seriously causing impaired vision.

Meanwhile, CAR-T (chimeric antigen receptor T-cell) therapies are being used in cancer, potentially saving the lives of patients with advanced blood cancers who would have almost certainly died in the past.

But these drugs are also phenomenally expensive. Gilead’s CAR-T Yescarta costs $373,000, while Novartis’ Kymriah costs up to $475,000 per patient, and patients can require constant monitoring and intensive care because of the extreme side effects the drugs can cause.

• Read the article in full in pharmaphorum's Deep Dive digital magazine