1.  EXECUTIVE SUMMARY

  1.1  Cancerbackup is the leading national charity providing information, understanding and support to people affected by cancer. The charity's specialist cancer nurses answer more than 60,000 enquiries a year from patients and carers on all aspects of cancer and its treatment. Cancerbackup's services include a telephone helpline, a wide range of booklets and factsheets, two award-winning websites and a network of local information centres. In addition to providing information and support, Cancerbackup works to promote patient-centred services and equitable access to high quality treatment, information and support for everyone affected by cancer.

  1.2  Cancerbackup has long supported the principle of a body such as NICE. In a complex health world there is a need for an independent body to assess the cost effectiveness of healthcare. During the past year NICE has introduced the new Single Technology Appraisal process, a new implementation team and Government has issued the Best Practice Guidelines, updating and clarifying Health Service Circular 1999/176. Cancerbackup warmly welcomes these moves and believes that many more patients will benefit as a result.

  1.3  However, it remains the case that the UK lags behind much of the rest of Europe: 65[65] we spend less than other countries on cancer treatments, and we are slower to provide them on the NHS. These delays and barriers to treatment mean that for many people with cancer treatments are not available in time to help them.

  1.4  Since the inception of NICE Cancerbackup has campaigned to develop the NICE process and improve the implementation of guidance produced. We strongly believe that as a patient organisation our role in relation to NICE is three-pronged:

—    to ensure the views of people living with cancer are represented in NICE technology appraisals and guideline development;

—    to ensure that the processes NICE undertakes consider the needs and views of all people living with cancer their families and friends fully; and

—    to ensure that NICE guidance on cancer is implemented across the NHS.

2.   WHY NICE'S DECISIONS ARE INCREASINGLY BEING CHALLENGED?

  2.1  Throughout the cancer community there is a widespread sense of difficulty in accessing treatments for people with cancer. We acknowledge that not all cancer treatments will be approved, or even assessed, by NICE. However there is often a consensus amongst clinicians, patients and patient groups that specific treatments are particularly important for people with cancer and should be approved. It is in these instances that Cancerbackup would challenge a NICE decision.

  2.2  Cancer treatments face specific issues which are not common to other disease areas.

—    Treatments are frequently developed to tackle late stage cancers before they are developed for early stages of the same disease. In these late indications treatments often give small, incremental survival and/or quality of life benefits to patients. Whilst these benefits are hugely valuable to people with cancer, many of these treatments prove non cost-effective under the NICE Technology Appraisal processes.

—    Quality of life issues can be specific to cancer treatments and not score highly within the NICE process. The impact of issues such as the oral administration of treatment in place of IV administration, chemotherapy induced fatigue and hair loss of patients undergoing cancer treatment and often in their final weeks of life are frequently undervalued.

—    Cancer treatments are becoming increasingly targeted and are therefore effective for smaller groups of patients. However the development costs for such treatments remain the same as, and may in fact be higher than, those for disease areas with larger patient numbers. The adherence by NICE to a strict QALY limit means that these treatments are seen as not cost effective, despite the obvious benefits to people with cancer and the low overall NHS cost.

  2.3  Whilst we understand the argument that in order to ensure equity across healthcare all treatments should be considered by the same process we feel that there is a strong case for a cancer specific work strand within NICE. See paragraph 4.6 for more information.

3.   WHETHER PUBLIC CONFIDENCE IN THE INSTITUTE IS WANING, AND IF SO WHY?

  3.1  It is not public confidence in NICE that is waning so much as confidence in the ability of the NHS as a whole to provide gold standard and internationally comparable cancer care. When NICE appears to get a decision "wrong" (as mentioned above in paragraph 2.1) this undermines confidence in the NHS. Public confidence in the NHS is damaged further when treatments are available in the EU and even Scotland but not in England and Wales.

  3.2  A recent MORI poll[66] carried out for Cancerbackup showed that nine in 10 British adults agreed that all groups in society should have equal access to cancer care, regardless of their age, gender, or walk of life; proving that the public values equity in cancer care. Importantly this statistic did not vary significantly across age groups.

  3.3  Variations in the availability of treatments around the UK creates uncertainty about the level of cancer care the NHS would provide. The same poll also found that only seven in 10 British adults were confident that the NHS would provide good cancer care if they had cancer compared to nine in 10 people who have had cancer in the past five years.

  3.4  A number of factors could support public confidence in NICE and NHS cancer care. In particular, promoting greater understanding of the role of NICE, changes to the evaluation process and above all a greater focus within the NHS on the implementation of guidance would all benefit the Institute's public standing.

4.   NICE'S EVALUATION PROCESS, AND WHETHER ANY PARTICULAR GROUPS ARE DISADVANTAGED BY THE PROCESS

  4.1  Cancerbackup participates in NICE to ensure that the views of people living with cancer their families and friends are considered. We recognise that NICE's evaluation processes are rigorous however we are particularly concerned by certain aspects of the technology appraisal process; in particular:

—    how much weight is placed on the views of patients and patient groups;

—    how QALYs are formulated;

—    the formulation of assessment reports by the Evaluation Review Groups (ERGs);

—    how orphan drugs and orphan disease areas are appraised;

—    and how cancer treatments as a whole can best be appraised.

4.2  Patient views

  4.2.1  As a patient organisation there is a concern regarding what evidence we can usefully add to the NICE process and how much this evidence is actually considered. The focus on cost per Quality Adjusted Life Year (QALY), statistical evidence and data from the manufacturers leaves little room for consideration of patient views. For example a recent Final Appraisal Determination stated:

"The Committee noted the clinical and patient experts' views that [X] is a potential breakthrough for patients for whom no other treatment is available",[67] before then issuing a negative decision on the treatment with little, apparent, consideration of the patient perspective.

  4.2.2  The views of people with cancer and patient organisations must be fully considered and weighted within the appraisal process.

4.3  QALY

  4.3.1  The NICE evaluation process focuses heavily on the cost per QALY of new treatments. We are concerned that the figures that make up the QALY do not include, or sufficiently weight, issues that are crucial to people with cancer.

  4.3.2  We would very much encourage the Committee to probe further the assessment of the cost per QALY and question whether it fully considers:

—    the impact of the treatment on quality of life;

—    the value society places on symptom management in the last weeks/months of life;

—    whether QALYs fully consider the costs to the wider NHS—with a long term perspective and across budget silos; and

—    and whether QALYs should include wider societal costs such as the ability of someone with cancer to continue in employment or to care for their children?

4.4  Evaluation Review Groups

  4.4.1  The NICE appraisal process relies heavily upon reports from the Evaluation Review Groups, especially in the case of Single Technology Appraisals (STAs). There are seven ERGs based within universities across the UK.

  4.4.2  We are concerned that the conclusions these groups come to, the methods they use, and often therefore the results of an appraisal, vary between the ERGs. We would very much encourage the Health Select Committee to explore this stage of the process.

4.5  Orphan drugs and diseases

  4.5.1  Cancerbackup is concerned that drugs designated as orphan products by the EMEA are unlikely to receive NICE approval if the same appraisal process and cost effectiveness threshold applies as for other treatments. NICE examined this issue in 2005 with its Citizens Council[68] and published draft recommendations in 2006. [69]NICE decided to divide orphan drugs into two categories:

—    Orphan drugs—conditions with a prevalence of less than five per 10,000 of the population.

—    Ultra-orphan drugs—conditions with a UK prevalence of less than one in 50,000.

  4.5.2  NICE recommended that separate decision rules and a new a higher QALY threshold, up to £300,000 per QALY, would be needed to enable it to assess ultra-orphan drugs.

  4.5.3  Cancerbackup believes that the EMEA definition of orphan drugs should be adopted and that a new process is needed to assess all orphan products. Cancerbackup believes this separate appraisal procedure should also cover treatment for rare cancers where the treatment is suitable for less than 5 per 10,000 of the population but where the manufacturer did not apply for orphan drug status at the time of licensing.

4.6  Cancer Specific Workstream

  4.6.1  Cancerbackup believes that there are strong justifications for a cancer specific workstream within NICE. Assessing cancer treatments raises a very specific set of issues (as laid out in paragraph 2.2 above) and there are a large number of cancer treatments currently going through, or shortly due to start, the licensing process.

  4.6.2  A cancer specific workstream could continue the model of the cancer specific Consideration Panel. It could ensure that those experts carrying out NICE appraisals on treatments for cancer fully understand cancer, the issues for people living with cancer and the specific set of issues associated with this condition. If this is to be successful it would be necessary to allocate sufficient resources to NICE for it to carry out its role successfully.

5.   THE SPEED OF PUBLISHING GUIDANCE

  5.1  Throughout 2005 Cancerbackup campaigned to speed up the NICE technology appraisal process; naming 23 cancer drugs which were being held up in the system as a result of delays inherent in the way technologies were appraised. These delays meant that many cancer patients would be waiting for up to four years for new treatments to become routinely available on the NHS. For many, the delays meant that the treatments would not be available in time to help them.

  5.2  The new Single Technology Appraisal (STA) process introduced at the end of 2005 has seen a considerable improvement in the time taken for treatments to pass through the NICE appraisal process. The new Consideration Panels for referring treatments to NICE are now looking at treatments and procedures far ahead of licensing and supporting a faster NICE process. We welcome this.

  5.3  However of the eleven cancer treatments which have gone, or are in the process of going, through the NICE STA process six have received negative decisions[70] many due to a lack of evidence or cost effectiveness issues. If NICE is to continue to appraise new cancer treatments quickly we hope it will understand that data is often immature at license stage and cost effectiveness data may be best built up whilst the treatment is used within the NHS. Risk sharing agreements and early access agreements may need to be entered into in order to enable speedy use of new treatments within the NHS.

6.   THE IMPLEMENTATION OF NICE GUIDANCE, BOTH TECHNOLOGY APPRAISALS AND CLINICAL GUIDELINES

  6.1  Cancerbackup is seriously concerned with the continuing problems regarding the implementation of NICE guidance. We believe that much greater emphasis should be given to ensuring NICE guidance is implemented.

  6.2  We know from the recent report[71] by the National Cancer Director that whilst the uptake of cancer drugs which have been approved by NICE is improving it varies by 2.2-3.3 fold across the country. Cancerbackup believes that an annual audit on the implementation of NICE guidance and support for those Trusts with a slow uptake of guidance is needed to reduce problems of postcode prescribing.

  6.3  The Department of Health should set aside a pot of money as an "Innovation Fund". This Fund should be available to fund the use of new cancer treatments which get their licence and/or NICE guidance part way through the financial year. This would ensure that new, innovative, treatments are made widely available across England and Wales as soon as possible. It would also support the implementation of NICE guidance in the first crucial few months.

  6.4  The recent development by NICE of new commissioning tools and databases to aid implementation and the establishment of local representatives to explain guidance are to be welcomed. However the implementation of NICE guidance forms part of the Healthcare Commission's assessment process and whilst Technology Appraisals are part of the Healthcare Commission's Core Standards NICE Guidelines are only Developmental Standards.

  6.5  NICE's remit should be extended to include responsibility to work with the Healthcare Commission to promote and monitor implementation of NICE guidance. If this is to be successful it would be necessary to allocate sufficient resources to NICE for it to carry out its role successfully.

7.  RECOMMENDATIONS

  7.1  NICE needs to promote greater understanding of its role and processes.

  7.2  The views of people with cancer and patient organisations must be fully considered and weighted within the appraisal process.

  7.3  We would very much encourage the Committee to probe further the assessment of the cost per QALY.

  7.4  We would very much encourage the Committee to explore the methods and conclusions of the ERGs.

  7.5  The EMEA definition of orphan drugs should be adopted and a new process set up to assess all orphan products and treatments for rare cancers where the treatment is suitable for less than five per 10,000 of the population but where the manufacturer did not apply for orphan drug status at the time of licensing.

  7.6  A cancer specific workstream should be set up within NICE to consider cancer treatments. If this is to be successful it would be necessary to allocate sufficient resources to NICE for it to carry out its role successfully.

  7.7  To support positive results from the new STA process NICE must understand that clinical data is often immature at license stage and cost effectiveness data may be best built up whilst the treatment is used within the NHS. The use of risk sharing agreements and early access agreements to enable speedy use of new treatments within the NHS should be considered.

  7.8  The Department of Health should set up an "Innovation Fund" to fund the use of new cancer treatments which get their licence and/or NICE guidance part way through the financial year.

  7.9  NICE's remit should be extended to include responsibility to work with the Healthcare Commission to promote and monitor implementation of NICE guidance. If this is to be successful it would be necessary to allocate sufficient resources to NICE for it to carry out its role successfully.

Joanne Rule

Chief Executive, Cancerbackup

March 2007

66   Cancer-A Public priority? Attitudes towards cancer treatment in Britain. Research Study conducted for Cancerbackup, Ipsos MORI, August 2006. Back

67   Final Appraisal Determination, Erlotinib for the treatment of non-small-cell lung cancer, NICE February 2007. Back

68   NICE Citizens Council Report: Ultra Orphan Drugs, November 2004. Back

69   NICE: Appraising Orphan Drugs, March 2006. Back

70   In some cases this is not yet the final decision or is subject to appeal. Back

71   Usage of cancer drugs approved by NICE. Report of a Review undertaken by the National Clinical Director, Gateway number 7124, 20 September 2006. Back