CDF Approval for Blood Cancer Drug

Peter Russell

March 15, 2019

A monoclonal antibody treatment for people with relapsed multiple myeloma was approved this week by the National Institute for Health and Care Excellence (NICE) for use on the Cancer Drugs Fund (CDF).

NICE was also involved in talks on breaking an impasse that has prevented a cystic fibrosis (CF) drug from being made available to patients in England.

There was welcome news for some people with breast cancer after pharmaceutical company Roche agreed to fund a treatment under an early medicines' access scheme.

Blood Cancer Drug Approved for CDF

NICE approved daratumumab (Darzalex, Janssen-Cilag) through England's CDF as an option for treating relapsed multiple myeloma in patients who have had three previous therapies.

In final draft guidance, an appraisal committee found that daratumumab, used after previous treatment with agents such as bortezomib (Velcade, Janssen-Cilag) plus dexamethasone, could not be proven to extend the length of time people lived before their disease progressed but might improve health-related quality of life.

Daratumumab is an innovative monoclonal antibody treatment. It kills multiple myeloma cells by recognising CD38, a protein commonly expressed in this particular type of cancer cell, and is the only myeloma treatment of this kind.

NICE estimated that around 2900 people in England would be eligible for treatment with daratumumab each year while it is available through the CDF.

Meindert Boysen, director of NICE's Centre for Health Technology Evaluation, commented: "Some of the cost effectiveness evidence we reviewed suggests daratumumab could notably increase the overall survival of people who have had one previous therapy, but the long-term trial data is limited.

"We have approved daratumumab for use in the Cancer Drugs Fund so people at this stage of their disease can access the treatment while more data is collected."

Shelagh McKinlay, head of patient advocacy at Myeloma UK said: "This is really positive news and is a major step-change in the treatment available to patients at this stage in their myeloma. For too long there has been real unmet need at first relapse in myeloma.  Limited treatment options have meant that patients were missing out at a critical time in their disease pathway."

Early Access for Breast Cancer Drug

Adults in the UK with PD-L1 positive, unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) will be able to have early access to atezolizumab (Tecentriq, Roche) in combination with nab-paclitaxel (Abraxane, Celgene).

Roche agreed to fund atezolizumab following a positive scientific opinion by the UK’s Medicines and Healthcare products Regulatory Agency (MHRA), under the Early Access to Medicines Scheme (EAMS).

The MHRA said a study had shown that a year after starting treatment, 29% of the patients treated with the monoclonal antibody combined with nab-paclitaxel chemotherapy were alive without cancer progression, compared with16% of those treated with chemotherapy alone.

However, the MHRA said that the number of patients studied was small, mainly confined to those under 75, and that data on overall survival are still preliminary.

Lesley Hugo, breast cancer franchise lead at Roche said: "People with TNBC currently have poor clinical outcomes and treatments are limited, with chemotherapy being the main therapeutic option.

"Roche has worked closely with healthcare bodies to deliver access for patients to the innovative medicine atezolizumab as quickly as possible."

An estimated 15% of breast cancers in the UK are classed as TNBC, a small proportion of whom could be eligible to receive atezolizumab.

CAR T-Cell Therapy on CDF for Lymphoma Patients

Also recommended for inclusion on the CDF this week was the chimeric antigen receptor (CAR) T‑cell therapy tisagenlecleucel (Kymriah, Novartis) as an option for treating adult patients with relapsed or refractory diffuse large B-cell lymphoma after two or more lines of systemic therapy.

NICE initially rejected tisagenlecleucel, which has a list price of £282,000 for a single intravenous infusion, on cost grounds. However, the manufacturer has since agreed a confidential price discount.

In final guidance, an appraisal committee said that evidence from a single-arm study with short follow-up, and a small observational study, suggested that people having tisagenlecleucel might live for longer or have more time before their disease relapsed. However, it said a longer follow-up was needed.

Last month, NICE described its positive decision on tisagenlecleucel as "a step forward for personalised medicine".

NHS England said it was working closely with several hospitals across the country to deliver the complex treatment which it is estimated could benefit around 200 people per year.

Talks About Cystic Fibrosis Drug

NICE, NHS England, and the Secretary of State for Health and Social Care, Matt Hancock, held talks earlier this week with pharma company Vertex to try to break the stalemate that has prevented patient access in England to CF drug lumacaftor-ivacaftor (Orkambi).

Vertex chairman Dr Jeffrey Leiden flew into London to give evidence last week to a House of Commons committee investigating why discussions over the treatment had been deadlocked for almost 3 years.

Lumacaftor-ivacaftor is licenced to treat CF in people 12 years and older who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which around 50% of people with CF in the UK have.

An estimated 2834 people in England could benefit from treatment with the systemic protein modulator.

NICE has rejected the drug on cost-effectiveness grounds.

Discussions on an offer by NHS England to pay Vertex £500 million over 5 years to include access to the company's portfolio of CF treatments have so far been fruitless.

Anal Fistula Treatment

NICE updated its interventional procedures guidance on radially emitting laser fibre treatment of an anal fistula.

It said that current evidence on the safety and efficacy of the treatment was limited in quantity and quality and that, although it carried no major safety concerns, the procedure should only be used with special arrangements for clinical governance, consent, and audit or research.

It also said that the procedure should only be carried out by clinicians experienced in cannulating fistulas, and who were trained in the use of lasers.

Mental Illness and Substance Abuse

NICE issued a draft quality standard for consultation on the assessment, management, and care provided to people aged 14 and over who have coexisting severe mental illness and substance misuse.

Consultation on the draft closes on 10th April 2019. The final quality standard is expected to be published in August 2019.

Rheumatoid Arthritis Consultation

NICE produced a draft diagnostics consultation document on therapeutic monitoring of TNF-alpha inhibitors in rheumatoid arthritis.

Guidance will cover the use of enzyme-linked immunosorbent assay (ELISA) tests. The draft said although the tests showed promise, there was currently insufficient evidence to recommend their routine adoption for rheumatoid arthritis.

The closing date for comments from interested parties is 4th April 2019.

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