How can healthcare systems pay for regenerative medicine? NICE has some ideas
England's watchdog for healthcare cost effectiveness NICE has looked ahead to the impact of regenerative medicines currently in pharma company pipelines.
These treatments could offer huge benefits for patients, but could also be extremely costly. NICE has put together a hypothetical - but entirely plausible scenario - where a CAR-T drug is priced above £500,000 per patient.
NICE wanted to explore whether it could use existing health economic approaches to appraising regenerative and cell therapies, or whether it needed to alter them significantly for these coming technologies.
The organisation has concluded that it can use the same methods and decision making approaches, but has suggested some new models for paying for such 'step change' treatments.
NICE carried out the study following a recommendation by the Department of Health Regenerative Medicine Expert Group. It asked academics from the University of York to create a hypothetical case of a new and unlicensed cell therapies for treating a type of leukaemia in children and young adults to explore the possibilities.
They set up a special expert panel to consider chimeric antigen receptor (CAR) T-cell therapy in relapsed or refractory B-cell acute lymphoblastic leukaemia in children in young adults. The study concerned a hypothetical example product, with characteristics based on early clinical data for related real products supplemented with hypothetical evidence.
This therapy in unlicensed, at the early stages of development, with little data and an unknown price, NICE said.
The hypothetical example involved a product that gave up to 10 additional quality adjusted life years, representing a 10 year life extension at full health, costing over £500,000 per patient.
However NICE noted in its report that it did not consider this to be indicative of the real-life price of such products should they become commercially available.
NICE also noted that as is the case with conventional therapies, products with high prices that give modest improvements in patient outcomes would not be considered cost-effective.
Dr Nick Crabb, programme director – scientific affairs at NICE, said: "Products with these, albeit hypothetical, characteristics would represent major advances in therapy, offering profound benefits to eligible patients compared with the current NHS standard of care.
"Step-change improvements in patient outcomes from cancer treatments of this magnitude are rarely seen with conventional therapies. But the introduction of these products may also present financial challenges.
"Methods will need to be developed that allow healthcare systems to find ways of making them available to patients in a financially sustainable way."
In its report NICE suggested a "lifetime leasing" model, where in this case one-off acquisition costs were replaced with monthly payments of £2,756 as a possible solution.
The report authors noted that in this case they did not consider a 1.5% discount rate should be applied in cost-effectiveness calculations due to uncertainties over the treatment's benefits.
This lower rate would have made cost-effectiveness calculations more favourable compared with the standard 3.5% rate used across the public sector.
This issue was also raised in a parliamentary committee hearing by charities concerned about the UK's deliberations on vaccine cost-effectiveness last week.
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