Latest NICE Funding Decisions

Peter Russell

December 21, 2018

There were positive decisions this week by the National Institute for Health and Care Excellence (NICE) on two leukaemia treatments.

There were also funding announcements on treatments for liver cancer and melanoma.

Tisagenlecleucel-T for Leukaemia

Tisagenlecleucel (Kymriah, Novartis) was recommended for use within the Cancer Drugs Fund (CDF) as an option for treating relapsed or refractory B-cell acute lymphoblastic leukaemia in people aged up to 25 years.

Current treatment is usually blinatumomab (Blincyto, Amgen) or salvage chemotherapy.

In final guidance, NICE said tisagenlecleucel – a CAR T-cell therapy, which uses a patient's own modified T cells to attach to and kill cancer cells – may help patients live longer but a number of uncertainties remained.

It said making the treatment available within the CDF would allow more data to be collected, particularly on overall survival, subsequent stem cell transplant rates, and immunoglobulin usage.

The list price for tisagenlecleucel is £282,000 per infusion, but the manufacturer had agreed a confidential price discount.

Liposomal cytarabine–daunorubicin for Leukaemia

Liposomal cytarabine–daunorubicin (Vyxeos, Jazz Pharmaceuticals) was recommended as an option for untreated therapy-related acute myeloid leukaemia or acute myeloid leukaemia with myelodysplasia-related changes in adults.

In final guidance, NICE said that current treatment was chemotherapy, but that clinical trials had demonstrated that people having liposomal cytarabine–daunorubicin lived longer than people having standard chemotherapy.

The manufacturer had agreed to provide the drug at a discounted price, and the appraisal committee was satisfied that the drug was a cost-effective use of NHS resources for end-of-life treatments.

Liver Cancer Drug

NICE recommended in final guidance the life-extending drug lenvatinib (Lenvima, Eisai) for some adults in England and Wales with untreated unresectable advanced hepatocellular carcinoma.

Patients would be eligible for treatment if they had Child–Pugh grade A liver impairment and an Eastern Cooperative Oncology Group (ECOG) performance status of 0 or 1, NICE said.

Usual treatment is with sorafenib (Nexavar, Bayer), but around a quarter of people stop taking it because they cannot tolerate its side effects. Lenvatinib, which also has common side effects such as hypertension, would offer an alternative first-line treatment option.

NICE estimated that around 900 people each year would be eligible for treatment.

Most patients take three 4mg capsules daily. The list price for lenvatinib is £1,437 for 30x4mg capsules, but the manufacturer agreed to provide the drug to the NHS at a confidential discount.

Pembrolizumab for Melanoma

NICE recommended that pembrolizumab (Keytruda, Merck Sharp & Dohme) should be available within England's CDF as an option for the adjuvant treatment of stage III melanoma with lymph node involvement in adults who have had complete resection.

It said more evidence was needed to address uncertainties over the treatment and it was therefore too early to decide whether the drug should be recommended for routine NHS use.

The drug would be supplied for the CDF at a confidential discount.

Tumour profiling

NICE published final updated guidance recommending three tests as options to help NHS clinicians decide whether to prescribe chemotherapy for some people with early breast cancer.

Previous guidance published in 2013 recommended Oncotype DX Breast Recurrence Score (Genomic Health) as an option for people with oestrogen receptor (ER)-positive, human epidermal growth factor receptor 2 (HER2)-negative and lymph node (LN)-negative, who were assessed as being at intermediate risk of the cancer spreading, and where the decision to prescribe chemotherapy remained unclear.

Updated guidance now also recommends two further tests – EndoPredict (Myriad Genetics), and Prosigna (NanoString Technologies) – as options.

Owing to uncertainty around the impact of the tests on chemotherapy decision-making and longer term patient outcomes, the guidance recommended that test data is collected and made available to the National Cancer Registration and Analysis Service.

Ectopic Pregnancies

New recommendations to help standardise the diagnosis and treatment of ectopic pregnancy were published in draft guidance.

It included signs to look out for during a transvaginal ultrasound that could indicate an ectopic pregnancy, such as an attached mass separate to the ovary.

There were new recommendations on managing small ectopic pregnancies in clinically stable women.

The update also said that women should be advised that the rate of ectopic pregnancy, and the risk of rupture, is similar following expectant and medical management.

Pancreatic Cancer

NICE also updated its quality standard for the diagnosis of pancreatic cancer and management of pancreatic ductal adenocarcinoma in adults aged over 18.

It followed a consultation period earlier this year with interested parties.


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