by Natalie Morrison
LONDON, 5 Oct (APM) - Novartis is confident its ongoing discussions with authorities in England concerning access to its CAR-T therapy Kymriah in adults will lead to a positive decision this year, a company spokesperson told APM on Friday as Gilead reached an agreement on Yescarta.
Kymriah (tisagenlecleucel) and Yescarta (axicabtagene ciloleucel) are the only two products approved in a new class of treatment known as CAR-T - cancer therapies made from the patients’ own T-cells which are re-engineered to attack blood cancers.
Both received European regulatory approval last month for adult diffuse large B-cell lymphoma (DLBCL) indication, but were swiftly rejected by England's health technology assessment (HTA) body NICE in draft guidances after they were deemed too costly (APMHE 59754
, APMHE 59454
However, on Friday, NHS England announced that NICE struck a new commercial deal with Gilead that means its CAR-T will be made available to UK patients within coming weeks under the UK’s Cancer Drugs Fund (CDF), a managed access programme for cancer drugs.
Asked via email whether the approval signals a similar decision to come for Kymriah, a Novartis spokesperson told APM: “For adult patients with r/r DLBCL, we are continuing to build on our close collaboration with NICE and NHS England to reconsider their preliminary decision and facilitate access for NHS patients to this new therapy as soon as possible.
“We are confident that our ongoing discussions will be productive towards reaching a positive decision for patients in the UK this year.”
Novartis was unable to comment as to whether it will have to bring the cost of Kymriah within NICE’s well-known spending thresholds - £30,000 per quality adjusted life year (QALY) as a standard and £50,000 for end of life treatments. NICE decided not to grant ‘end of life’ criteria for the adult indication when it said ‘no’ to reimbursement back in September (APMHE 59761
However, Kymriah did receive clearance last month for use on NHS England's CDF in its other approved indication, acute lymphoblastic leukaemia (ALL) in children and young adults, when it was suggested the list price should be £282,000 (APMHE 59548
The same list price is reportedly being used in DLBCL, although the discount afforded to the NHS on this list price is unknown for either indication.
It is still unclear how Yescarta will be priced under the CDF, and neither Gilead or NICE have revealed its list price. When Yescarta was originally rejected, NICE said treatment cost was above the well-known £50,000 per quality adjusted life year (QALY) spending threshold.
APM asked Novartis if it expects a decision for Kymriah similar to Yescarta.
The spokesperson said: “We cannot comment on the products of other companies. As recently as a month ago, Novartis secured access for our CAR-T cell therapy, Kymriah, for paediatric and young adult ALL patients in the UK, working closely with NHS, NICE and through the Cancer Drug Fund.
“As the company that demonstrated leadership in this area, Novartis understands the unique opportunities and challenges presented by this completely new way of treating cancer.”
CDF for all
The fact both of the UK green lights for CAR-T so far have happened under the CDF could be a sign of the changing reimbursement landscape, if experts are to be believed.
Being included on the CDF does not mean full routine NHS commissioning - rather it offers a temporary funding option for promising drugs that cannot be deemed cost effective at the time of NICE appraisal. Companies can then collect the necessary evidence or strike the right commercial deal to support routine reimbursement.
Experts at this week’s TOPRA symposium in Stockholm discussed the difficulties for HTA bodies and payers of treatments being approved on more limited data than ever before, due in part to accelerated access schemes, yet coming in costlier than their predecessors.
CAR-T therapies have become somewhat of a poster child for this predicament, with both Yescarta and Kymriah being approved on data sets in just dozens of patients, both with high upfront costs.
Niklas Hedberg, chair of Europe’s HTA network EUnetHTA told APM that flexible pricing models will become increasingly important to pay for these costly but uncertain therapies, for instance those which allow for the collection of real world data (APMHE 60023
) - a criteria the CDF fits.
Meanwhile the recently-retired chair of the European Medicines Agency’s (EMA) CHMP, Tomas Salmonson, told APM that payers and HTA bodies should recognise that there are uncertainties surrounding any new product.
He had just told an audience at the symposium that conditional approvals should become "the norm" for any new product, calling for a bigger focus on post-approval data (APMHE 59999