NICE has confirmed in final draft guidance that nab-paclitaxel, when given with the chemotherapy drug gemcitabine, should not be funded by the NHS for people whose pancreatic cancer has spread and who have not received any other treatments.
People with newly diagnosed pancreatic cancer are usually offered a combination treatment called FOLFIRINOX, but it is associated with serious side effects and cannot be given to everyone. Other options include gemcitabine either on its own or with another chemotherapy called capecitabine.
Information provided by Celgene showed that although nab-paclitaxel with gemcitabine was more effective than gemcitabine alone, it resulted in more serious side effects. It also caused more side effects than gemcitabine with capecitabine despite having similar effectiveness.
Commenting on the draft guidance, Sir Andrew Dillon, NICE chief executive, said: "Pancreatic cancer has no obvious warning signs so many people are not diagnosed until their cancer is advanced. Prognosis is poor and without treatment people may live for only 2 to 6 months.
"Unfortunately the development of new treatments for pancreatic cancer has been very limited in recent years. Although nab-paclitaxel is more effective than one of the treatment options currently available, it is linked with more side effects. When considering the impact of side effects on a patient's quality of life and the fact that it is also more expensive, nab-paclitaxel cannot be considered an effective use of NHS resources."
The draft guidance was issued in response to an appeal hearing on earlier draft guidance. The Appeal Panel requested further rationale on the Committee's considerations on the weighting of the quality-adjusted life year under NICE's end-of-life policy2.
The Appraisal Committee was also asked to look at whether the current Pharmaceutical Price Regulation Scheme (PPRS) would affect the drug's cost effectiveness. The committee agreed that the rebate provided by the company to the Department of Health could not be taken into account as there was no guarantee it would be directly re-invested in the NHS and could not be considered a nationally available price reduction that was relevant for use in cost-effectiveness analyses.
Sir Andrew added: "Even allowing for additional flexibility by applying NICE's end-of-life criteria in assessing cost-effectiveness, we were not able to recommend this drug for routine use. Our independent advisory committee applied our position statement on the PPRS scheme and concluded the payment made by the company to the Department of Health as part of this scheme, could not be taken into account in assessing the cost effectiveness of the medicine."
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.
For more information call the NICE press office on 0300 323 0142 or out of hours on 07775 583 813.
Notes to Editors
- Nab-paclitaxel 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.
- When assessing the cost effectiveness of a treatment committees take a number of factors in to account to determine the cost of using the drug to provide a year of the best quality of life available (also known as quality adjusted life year or QALY). For treatments that cost above £30,000 per QALY the Committee will need to identify an increasingly stronger case for supporting the technology as an effective use of NHS resources. One of the factors that can be considered is whether the technology meets the criteria for special consideration as a 'life-extending treatment at the end of life'. If a drug meets the criteria to be considered under NICE's supplementary advice for end of life treatments, then the committee can consider the impact of giving a greater weight to QALYs achieved in the later stages of terminal diseases and the magnitude of the additional weight that would need to be assigned for the cost effectiveness to fall within the normal range of maximum acceptable ICERs. The Committee concluded that the end-of-life criteria could be applied for people whom current treatments were unsuitable, however the weighting that would have to be placed on the QALYs gained would be too high to consider nab-paclitaxel a cost-effective use of NHS resources.
- Side effects of nab-paclitaxel with gemcitabine include a drop in the number of 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.
- The draft guidance is available at /guidance/indevelopment/GID-TAG453.
- The list price of nab paclitaxel is £246 per 100 mg vial (excluding VAT; British national formulary [BNF] edition 67). The company estimated the average cost of a 28 day cycle of treatment of nab-paclitaxel to be £1481 (excluding VAT). Median time on treatment with nab paclitaxel from the CA046 study was 3.4 months, equating to a cost of approximately £5035 per patient (excluding VAT).
- The Committee agreed that the most plausible cost per QALY (Quality Adjusted Life Year) for nab-paclitaxel plus gemcitabine compared with gemcitabine alone, would lie somewhere between £72,500 and the £78,500.
- The company's analyses showed that nab-paclitaxel plus gemcitabine was dominated by FOLFIRINOX and had an ICER of £87,100 per QALY gained compared with gemcitabine plus capecitabine. Although these estimates were subject to considerable uncertainty, the Committee was confident that nab-paclitaxel plus gemcitabine would not be considered a cost effective use of NHS resources compared with these treatments.
The National Institute for Health and Care Excellence (NICE) is the independent body responsible for driving improvement and excellence in the health and social care system. We develop guidance, standards and information on high-quality health and social care. We also advise on ways to promote healthy living and prevent ill health.
Our aim is to help practitioners deliver the best possible care and give people the most effective treatments, which are based on the most up-to-date evidence and provide value for money, in order to reduce inequalities and variation.
Our products and resources are produced for the NHS, local authorities, care providers, charities, and anyone who has a responsibility for commissioning or providing healthcare, public health or social care services.