The cost of nab-paclitaxel is not justified by its limited benefit, says NICE in draft guidance
NICE has published new draft guidance which recommends that nab-paclitaxel taken with gemcitabine should not be funded by the NHS for previously untreated metastatic pancreatic cancer, because it has limited benefits compared to current treatments and it is more costly.
Nab-paclitaxel, which is taken with gemcitabine, is also known as Abraxane and is marketed by Celgene. It is a novel formulation of paclitaxel, a chemotherapy which works by blocking cell division and promoting cell death. In this formulation, paclitaxel is attached to albumin which helps it move through the walls of blood vessels. NICE is currently looking at how well nab-paclitaxel, taken with gemcitabine, works for people whose pancreatic cancer has spread and who have not received any other treatments.
In the early stages, pancreatic cancer doesn't usually cause any symptoms, which can make it difficult to recognise and means that many people are not diagnosed until the cancer is very advanced. Without treatment, survival may be only 2 to 6 months. Some people may be offered a chemotherapy treatment called FOLFIRINOX (a combination of fluorouracil, leucovorin, irinotecan and oxaliplatin), which is the current standard of care for people who are not too poorly to receive it, but this treatment has serious side effects which means it is not always an appropriate option. When this is the case gemcitabine (recommended by NICE in 2001) is generally used, or gemcitabine taken with capecitabine.
Commenting on the draft guidance, Sir Andrew Dillon, NICE chief executive, said: "Unfortunately the development of new treatments for pancreatic cancer has been very limited in recent years. Although a number of newer treatments including nab-paclitaxel have been introduced, we are disappointed that, when considering the impact of side effects as well as how effective the treatment is the evidence fails to show that it works any better for patients than other treatments already provided by the NHS. It is also more expensive."
Information provided by Celgene, the company which markets the drug, showed that FOLFIRINOX was more clinically effective than nab-paclitaxel with gemcitabine. It also showed that although nab-paclitaxel with gemcitabine was more effective than gemcitabine alone, it resulted in more serious side effects. Nab-paclitaxel with gemcitabine was similar in effectiveness to gemcitabine with capecitabine but may have more side effects.
Stakeholders, including Celgene, healthcare professionals and members of the public are now able to comment on the preliminary recommendations which are available for public consultation.
Clinical experts told the Committee that the side effects for nab-paclitaxel with gemcitabine were more manageable compared to those associated with FOLFIRINOX, but both Celgene and the clinical specialists agreed that nab-paclitaxel with gemcitabine would not be suitable for people who are too poorly.
Until final guidance is issued, NHS bodies should make decisions locally on the funding of specific treatments. Once NICE issues its final guidance on a technology, it replaces local recommendations across the country. This draft guidance does not mean that people currently taking nab-paclitaxel with gemcitabine will stop receiving it. They have the option to continue treatment until they and their clinicians consider it appropriate to stop. Nab-paclitaxel is currently available via the Cancer Drugs Fund.
For more information call the NICE press office on 0300 323 0142 or out of hours on 07775 583 813.
Notes to Editors
Explanation of terms
Paclitaxel formulated as albumin-bound nanoparticles (referred to in this document as nab-paclitaxel; Abraxane, Celgene) has a UK marketing authorisation in combination with gemcitabine for the first-line treatment of adults with metastatic adenocarcinoma of the pancreas.
Side effects of nab-paclitaxel with gemcitabine include a drop in blood cells causing an increased risk of infection, bleeding problems, tiredness and breathlessness, numbness or tingling in fingers and toes (peripheral neuropathy), gastrointestinal problems; aching joints and muscles, tiredness, feeling sick and hair loss.
About the guidance
The draft guidance will be available from Tuesday 9 September 2014.
The marketer of the drug submitted data from Study CA046 which showed that nab-paclitaxel plus gemcitabine compared with gemcitabine had statistically significantly longer overall survival (median gain of 2.1 months) and progression-free survival (median gain of 1.8 months). A mixed treatment comparison, presented by the company showed that nab-paclitaxel plus gemcitabine was associated with a shorter overall survival and progression-free survival compared with FOLFIRINOX, and with a similar overall survival and progression-free survival compared with gemcitabine plus capecitabine.
The list price of nab-paclitaxel is £246 per 100 mg vial (excluding VAT). The company's submission also states that a 250 mg vial will be available from September 2014 at a price of £615 (excluding VAT). The company estimated the average cost of a 28-day course of treatment to be £1,481 (excluding VAT).
The most plausible cost per QALY (Quality Adjusted Life Year) gained for nab-paclitaxel plus gemcitabine compared with gemcitabine alone was £78,500 per QALY gained. The company's analyses showed that nab-paclitaxel plus gemcitabine was dominated by FOLFIRINOX (that is, nab-paclitaxel was less effective and more costly) and had cost per QALY of £87,100 compared with gemcitabine plus capecitabine.
Nab-paclitaxel plus gemcitabine fulfilled the first end-of-life criterion for short life expectancy, and the third criterion for a small patient population. However, the extension to life for nab-paclitaxel plus gemcitabine compared with all the comparators was shorter than 3 months, and therefore the Committee considered that it did not meet this end-of-life criterion, and that the end-of-life considerations could not be applied.
The SMC is currently considering the use of nab-paclitaxel in combination with gemcitabine for the first-line treatment of adult patients with metastatic adenocarcinoma of the pancreas
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