Takeda UK says it is disappointed that cost regulators have issued a negative recommendation for Cancer Drugs Fund therapy Adcetris in relapsed or refractory Hodgkin lymphoma (R/R HL).

Since its UK launch in 2012, more than 500 patients have had access to Adcetris (brentuximab vedotin) via the CDF for the treatment of R/R HL following an autologous stem cell transplant (ASCT) or following at least two previous therapies when ASCT or multi-agent chemotherapy is not a treatment option.

The drug is now being reviewed by the National Institute for Health and Care Excellence (NICE) for these initial launch indications and also for a new one, granted marketing authorisation by the European Commission in June 2016 for the treatment of patients with CD30+ HL at increased risk of relapse or progression following ASCT.

However, following its cost appraisal the Institute has reached the conclusion that evidence around the drug's clinical effectiveness is "immature and limited", and that it could not be considered a cost effective use of NHS resources.

According to Takeda, should this decision be made final within the Final Appraisal Determination expected in January 2017, it would mean that patients in England would be "denied access to a medicine that has transformed treatment paradigms and given many patients the opportunity to achieve a potential cure with or without a stem cell transplant".

It would also deepen inequalities in care across the UK it said, given that access to Adcetris was granted by the Scottish Medicines Consortium and the All Wales Medicine Strategy Group in 2014 and 2015, respectively. The Republic of Ireland has also approved access via its National Cancer Control Programme, while the drug is also available and reimbursed in all other major European countries.

"This draft decision from NICE is hugely disappointing for patients and the clinical community as we are at risk of losing access to a highly effective treatment that has rapidly become standard of care in relapsed or refractory Hodgkin lymphoma," said Dr Graham Collins, consultant in Haematology, Churchill Hospital, Oxford.

"Losing access would mean a retrograde step in therapy; reverting back to an era where clinicians were limited to managing patients with often ineffective and toxic chemotherapies and limiting our ability to offer patients potentially curative stem cell transplants".

"It is also concerning that this decision could potentially deny patients at high risk of relapse post-ASCT access to a valuable new treatment approach that has been shown to improve progression free survival in this patient population."

Jon Neal, business unit director, Oncology, Takeda UK, stressed that the drug "has been a game changer in the management of relapsed or refractory Hodgkin lymphoma and is the only licensed medicine in this setting", and that recently published five-year follow up data show that median overall survival for Adcetris was nearly 3.5 years, "longer than the historical survival data of less than three years quoted within the ACD".

"This is a rare disease that often affects young people and due to the high level of unmet need, brentuximab vedotin was granted a licence based on unprecedented Phase II trial results. This poses a challenge to the NICE methodology which penalises the absence of Phase III data which is often the case for rarer cancer medicines".