Of interest to our UK readers will be the announcement by Simon Stevens, the chief executive of the NHS, in a speech to the Association of the British Pharmaceutical Industry on 26 April 2018, that preparations are underway to assess making CAR-T therapies available on the NHS. No CAR-T therapies have yet been approved in Europe, but the two treatments already approved by the FDA in the USA (Novartis’ Kymriah and Kite Pharma’s Yescarta) are under assessment by the EMA, and have already been referred by the Department of Health to NICE, the body that decides whether new treatments are cost effective.

The involvement of NICE of course throws up the knotty issue of price for these potential cures. Kymriah and Yescarta are currently listed in the US at $475,000 and $373,000 respectively. Notably, whilst these are big numbers (and whilst inpatient and other ancillary costs also need to be taken into account), these costs are lower than some commentators had estimated they could be.

Stevens indicated that the drug makers would have to work with the NHS, saying “in order for [the therapies] to become widely available, manufacturers need to set fair prices so that they are both affordable and sustainable in the long term”.

It will be interesting to see how NICE assesses these products, given the mock assessments carried out by NICE in 2016 (see here for a summary, and here for the assessment itself). One of the findings of the underlying studies run by the University of York was that in addition to discounting, innovative outcome-based payment methodologies need to be developed. One suggestion was the use of “lifetime leasing” whereby a monthly fee is paid for the duration of treatment benefits (until death). Another option considered is a variant of that which is now in place in the US for Kymriah. Under the US system, Novartis only gets paid if the patient treated with Kymriah through Medicare or Medicaid responds in the first month of treatment. The option considered by NICE in its mock assessment was that payment should only be made for patients in remission.

In addition to cost considerations, work is also underway to ensure that NHS Hospitals in the UK have the necessary services in place to administer these complex therapies once they are approved.