by Thomas Meek

LONDON, 11 Sep (APM) - NICE's current methods are able to deal with cell and gene therapies that have curative potential in chronic illness, according to a senior figure at the English health technology assessment (HTA) body.

NICE, which provides healthcare guidance for the National Health Service (NHS) in England and Wales, can take into account the benefits provided by these products, which are becoming increasingly common with advances in medical research, said Nick Crabb, programme director, scientific affairs at the organisation.

"I don't believe that our methods are fundamentally incapable of dealing with those sorts of situations," he said on Wednesday during a virtual conference on advanced therapies hosted by the Westminster Health Forum.

NICE has already recommended several advanced therapies that have shown the potential to have a long-term effect from a one-off treatment. These include Spark/Novartis' Luxturna (voretigene neparvovec) for a genetic form of vision loss; Orchard Therapeutics' Strimvelis for a rare immunodeficiency; and the CAR-T cancer therapies Kymriah (tisagenlecleucel) from Novartis and Yescarta (axicabtagene ciloleucel) from Gilead.

All these treatments have very high price tags for a one-off treatment, prompting debate on how they should be assessed by HTA bodies and they should be reimbursed by healthcare systems.

Crabb said HTA is feasible "as long as we can use time horizons in our modelling that take account of all of the differences in terms of costs and all the differences in terms of benefits and between the existing sort chronic treatments and the new therapy".

He pointed out that NICE used existing processes to make available Strimvelis and Yescarta. Strimvelis was recommended via the organisation's highly specialised technologies (HST) programme, which is intended for treatments in very rare conditions. Drugs reviewed as part of the HST programme are assessed against a maximum threshold of £100,000 to £300,000 per quality-adjusted life year (QALY) rather than the standard £30,000.

This meant NICE could recommend reimbursement for the treatment despite its list price of nearly €600,000.

And Kymriah was recommended for reimbursement via the Cancer Drugs Fund (CDF), a managed access programme that allows conditional reimbursement for a cancer treatment for a period of time while more data is collected on its benefit.

Gilead also agreed a commercial access arrangement for the product, which meant the NHS paid a discount on its list price.

Initiatives like the CDF have meant improved access, said Crabb. "If we go back a few years, we did not have things like the Cancer Drugs Fund, we weren't doing things like managed access arrangements then - that would have translated to a no [for Kymriah]. But fortunately, things have moved on and we do have those facilities in place now."

Review

Some industry figures have raised concerns about the use of current HTA processes for advanced therapies, however, in particular in areas of rare disease (APMHE 66397). Earlier this year Janssen's UK director of health economics, market access, reimbursement, government affairs, patient engagement, Jennifer Lee, told APM that NICE has not caught up with the modern medicine pipeline (APMHE 66403).

NICE has acknowledged its need to keep up to date and is currently in the middle of a review of its methodologies, although this has been delayed due to Covid-19 (APMHE 68826).

The organisation's chief executive Gillian Leng told APM this summer the review should speed up access for patients to new and promising health technologies (APMHE 67954).

"We want to support market access. And we want to simplify, if we can, the health technology evaluation process so it is clearer and more explicit to people."

Beyond HTA, innovative payment models that go further than simple discounts can be used to support the use of curative advanced therapies, said Crabb.

Stressing that he was speaking from a personal viewpoint, he said: "I think that we may need to start showing more interest in innovative payment models.

"So not just managing risk by discounts while the evidence is evolving a bit more - you're going a bit beyond that.

"If something appears to be a cure for a chronic illness but you're hoping that it's going to have a lifelong effect but you've only got two years of actual follow-up data and we really don't know what's going to happen, then perhaps there is a case for innovative models based on annuities for example."

New payment models, such as outcomes-based payments, have been championed by industry in recent years.

The UK has made some efforts to increase its flexibility on pricing through the launch last year of the voluntary scheme for branded medicines pricing and access (VPAS), which replaced the pharmaceutical pricing regulation scheme (PPRS).

It includes a new NHS England commercial framework that features additional 'commercial flexibility' to support access to new medicines, including an enhanced commercial access arrangement (CAA) that allows firms - for a fee - to make more complex payment agreements based on outcomes, dosages or patient populations in a confidential way (APMHE 62901).

Crabb said there is infrastructure in place for more complex payment models but there needs to be some use cases to "really drive us forward".

"I think when we get something where there's a really compelling case for patients to make a product available, but it needs something more complex and innovative on the payment model front. I think that's being confronted and specific cases, I believe, will drive the progress."

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