Breast Cancer Now has today called on the Department of Health to address the “intolerable trend” of new breast cancer drugs being rejected for use on the NHS by conducting a full review of the National Institute for Health and Care Excellence’s (NICE) drug appraisal process.

Monday 5 September 2016      Campaigns and policy
Current drug options for breast cancer in England may never be improved if appraisal process isn’t fixed

New Access to Medicines Summit report identifies four key issues preventing breast cancer patients from accessing innovative new treatments in England

The call follows the publication of the charity’s Access to Medicines Summit Report, which identifies four key barriers – inherent to the current NICE appraisal system – that if not addressed could see the best new breast cancer medicines continue to be rejected for use in England.

The system for assessing new and more targeted cancer drugs for NHS use in England has not been working for breast cancer patients in particular for some time now. Each of the last ten indications for breast cancer1 have been rejected by NICE for routine use on the NHS because they have not been deemed cost-effective.

The establishment of the Cancer Drugs Fund (CDF) in 2011 provided a temporary solution to ensure patient access to medicines while wider reform of the appraisal process was undertaken, enabling cancer patients in England to access targeted and life-extending treatments such as Kadcyla.

But this promised reform has unfortunately not been forthcoming, and with NICE now re-evaluating the breast cancer drugs that have previously been on the CDF – using the same methodology that saw the drugs rejected initially – NHS breast cancer patients could soon be denied access to a number of clinically-effective treatments within the coming year.

In the current system, NICE uses the cost per ‘quality adjusted life year’ (QALY) to compare different drugs and other technologies for different medical conditions. The QALY is a measure of the state of health of a person, in terms of length of life, and the quality of life – with one QALY being equal to 1 year of life in perfect health. NICE’s ‘threshold’, over which treatments are not recommended for use in the NHS, is normally £30,000 per QALY.

However, this process has failed to keep pace with innovations in breast cancer treatment, with not a single breast cancer drug being approved via NICE’s technology appraisal programme since 2007, causing breast cancer treatment in England to begin to lag behind the standards set by other countries2.

To this end, Breast Cancer Now held an Access to Medicines Summit in January 2016, bringing together a range of experts on issues relating to access to drugs to enable new ideas and possible solutions to be proposed on how current practice could be improved for the benefit of patients.

Participants at the Summit included representatives from NHS England and NICE, the Association of British Pharmaceutical Industry (ABPI), Roche, Pfizer, Eisai Europe, academia (including from UCL School of Pharmacy), charity and patient representative organisations – including Prostate Cancer UK, Breast Cancer Now, Myeloma UK and Pancreatic Cancer UK – as well as an independent economist and policy expert.

Participants highlighted the need for a review of the processes underpinning the drug appraisal system to ensure that the system is still working for the benefit of patients and the NHS. Using the ideas, suggestions and discussions recorded (under Chatham House rules) at the Summit, Breast Cancer Now have today published a report exploring the areas where the current system could be improved.

The Breast Cancer Now Access to Medicines Summit Report identifies four key points in the drug appraisal process which would benefit from increased flexibility and could help stop breast cancer patients in England missing out on the best new treatments coming through:

1. Branded and generic comparators

  • NICE currently calculates cost-effectiveness by working out the additional benefit of a potential new treatment compared to the existing treatment for that condition – and then uses the price difference between the two to see whether the benefit justifies the cost.
  • But, the price difference between new branded medicines and their generic comparators is often stark (often thousands of pounds per treatment), and no new medicines could therefore hope to compete on price against existing treatments where their comparators are generic medicines.
  • With many new breast cancer treatments having generic comparators, we run the risk of not being able to benefit from further new innovation and research progress and breast cancer patients missing out on new drugs being made available elsewhere.

2. Innovative neo-adjuvant (pre-surgery) treatments

  • We’re currently seeing a move towards more innovative uses of existing medicines, with drugs traditionally given towards the end-of-life now showing great promise much earlier in the patient pathway as first-line treatments.
  • However this is currently posing a problem for NICE in terms of the data that can be collected to assess a drug’s effectiveness, as it would take many years to collect overall survival data from a neo-adjuvant (before surgery) treatment being administered so early in the patient pathway. Overall survival data is the gold standard used by NICE to calculate the QALY value of a new cancer medicine.
  • Progression-free-survival (the time it takes before a patient’s cancer progresses) is often an earlier indication of the effectiveness of a new drug, however this is not necessarily equivalent to overall survival benefit and NICE currently has no agreed standard way of translating progression-free survival into predicted overall survival, meaning that a treatment with only PFS receives a very small QALY gain. Sometimes, when a treatment is shown to increase the time it takes for disease to progress, the cost-effectiveness can become worse.
  • If NICE continues to require overall survival data, this will take decades to collect and patients may miss out on these new uses of existing treatments for many years – clarity is therefore required around what other data they might accept in assessing more innovative drugs.

3. The value of quality of life

  • Similarly, participants raised the issue of the value of overall survival data compared to quality of life for patients in the drug appraisal process.
  • A recent US study found that a majority of patients valued quality of life more than extra length of life, while European patients valued overall survival more. While many patients have told Breast Cancer Now that they value quality of life as much as extra life, it is crucial that more robust research is carried out into the attitudes of UK metastatic patients to find out whether the weightings currently being applied by NICE actually reflect the views of those people who are most affected by these decisions.

4. ‘End of life’ criteria

  • The one significant change made to the NICE appraisal methodology since its establishment in 1999 was the introduction of the End of Life (EoL) criteria (in 2009), which allows a higher cost per QALY to be accepted for certain medicines that fall within the criteria: where medicines extend life by more than three months and are applicable to patient populations where the life expectancy is less than 24 months on average.
  • These two criteria cause significant inequalities in access for patients of different conditions. Some types of metastatic breast cancer, for example, have a life expectancy of just over two years which falls just outside of the EoL criteria. This is an arbitrarily-set cut-off point, and the End of Life criteria in Scotland, for example, goes up to 3 years, meaning patients in England are likely to miss out on some new treatments being made available north of the border.

With today’s publication, the charity is now calling on the Department of Health to address the overriding need identified by the Summit and conduct a wide-ranging review of the role of NICE and elements of the QALY process in drug appraisal, and to identify opportunities to improve the system going forward.

Baroness Delyth Morgan, Chief Executive at Breast Cancer Now, said:

“The last ten appraisals of breast cancer drugs by NICE have seen ten rejections and this intolerable trend is leaving patients in England behind.

“With the current barriers, it is hard to see how any new and improved breast cancer treatments would reach patients in England, despite the research progress being made.

“That said, we were highly encouraged by discussions at the Summit, which helped demonstrate the range of ideas and possibilities to fix this broken system. It has become crystal clear that we need the Department of Health to review the role of NICE and elements of the QALY process within the drug appraisal system.

“We must see industry price their drugs more responsibly, but much more flexibility is also needed in the way that modern and targeted drugs are assessed. 9,500 women in England are still losing their lives to breast cancer each year, and we must ensure that new innovations have the best chance of being made available at the best price for the NHS.”

Read the full Access to Medicines Summit report 2016

1 Afinitor has been assessed twice for the same indication in 2013 and 2016 with slightly new evidence.

2 International comparisons of Health Technology Assessment: A report from Breast Cancer Now and Prostate Cancer UK, published August 2016.