There are several controversies in the assessment of the benefit, value and sustainability of cancer drugs | European Journal of Cancer
First, there is a surprising disagreement on the impact of drugs on the overall outcome of cancer treatment.
Second, raising the bar of efficacy in clinical trials is claimed by many, but at the same time, being conservative appears justified as well, given the overall benefit of multiple incremental gains, typically observed in advanced solid tumours.
Third, sustainability of cancer drug cost is a prohibitive challenge, but no major successful action has been taken so far.
The reason for these controversies can be understood using concepts borrowed from psychological and cognitive sciences: each stakeholder has different perspectives generated by different availability biases; this implies different priorities, leading people to think, choose and act differently. Providing an ‘objective’ assessment of the benefit and value of a cancer drug makes sense only if the perspective adopted is clearly identified.
The American Society of Clinical Oncology (ASCO) scale fits the patients’ perspective by helping individual patients to choose the most valuable therapeutic option for their condition. Conversely, the European Society for Medical Oncology (ESMO) scale has a public health perspective: ranking the clinical benefit of oncologic drugs to identify those agents that should be available in every EU country. Because this scale is being adopted for reimbursement purposes in several European regions, the current major methodological problems of the scale should urgently be corrected to avoid unwanted societal consequences.
The NHS in England will soon be able to routinely fund the use of trastuzumab emtansine for people with certain categories of breast cancer, the National Institute for Health and Care Excellence (NICE) has announced in new draft guidance | OnMedica
NICE’s decision means that, by late summer, more than a thousand women and men could benefit from the drug.
HER2-positive breast cancer accounts for about a fifth of the roughly 41,500 women and 300 men who are diagnosed with breast cancer each year in England, but HER2-positive tumours are typically more aggressive than other types of breast cancer. The targeted treatment trastuzumab (Herceptin) is only effective for this type of breast cancer. Trastuzumab emtansine (Kadcyla) is licensed for the treatment of locally advanced or metastatic HER2-positive breast cancer, after trastuzumab and a taxane, taken either in combination or separately.
Currently, trastuzumab emtansine – which at full list price costs about £90,000 per patient – is only available on the NHS through the Cancer Drugs Fund (CDF). But NICE revealed yesterday afternoon that the drug’s manufacturer Roche had agreed a new commercial access arrangement with NHS England. When NICE factored this confidential agreement into a new clinical and cost-effectiveness analysis, also applying end-of-life criteria, it concluded that it can now recommend the drug as cost effective for routine use on the NHS.
Objectives: The aim of this study was to estimate lowest possible treatment costs for four novel cancer drugs, hypothesising that generic manufacturing could significantly reduce treatment costs.
Conclusions: Our findings demonstrate that affordable drug treatment costs are possible for novel cancer drugs, suggesting that new therapeutic options can be made available to patients and doctors worldwide. Assessing treatment cost estimations alongside cost-effectiveness evaluations is an important area of future research.
Salas-Vega, S. & Mossialos, E. LSE Health & Social Care Blog. Published online: 5 January 2017
To shed light on the clinical risks and benefits from new cancer medicines, we took a narrative synthesis approach to review regulatory assessments of the impact on overall survival, quality of life, and safety from all cancer medicines newly licensed in the US and EU between 2003-2013. For this, two researchers evaluated appraisals from English (National Institute of Health and Care Excellence, NICE), French (Haute Autorité de Santé, HAS) and Australian (Pharmaceutical Benefits Advisory Committee, PBAC) health technology assessment (HTA) agencies that were published through May 2015.
We find that while most new cancer drugs approved between 2003 and 2013 extended overall survival or improved the quality of life of cancer patients, their clinical benefits vary widely. Improvements in overall survival and quality of life also often come at the cost of safety, and there are reasons to question whether claims of clinical benefits have been matched by those observed in real-world settings.
The National Institute for Health and Care Excellence has issued draft guidance recommending that two drugs are removed from the Cancer Drugs Fund because they are not cost effective. The drugs, everolimus (Afinitor made by Novartis) for breast cancer and ibrutinib (Imbruvica, Janssen) for mantle cell lymphoma are being appraised under the new CDF system. The ultimate decision on whether the drugs will be removed will rest with NHS England
Under the new CDF system as a gateway fund for new cancer drugs, NICE recommends drugs are funded through the CDF if there is not enough information to make an immediate decision on whether they should be available on the NHS to enable patients to access the drugs while the company generates the supporting information required.
NICE rejected everolimus (in combination with the drug exemestane) for treating HER2-negative, hormone-receptor-positive advanced breast cancer in 2013 and it was then made available through the CDF.
Gallagher, J. BBC News. Published online: 16 May 2016
In a letter to the prime minister, the charities said many drugs would “now struggle to gain approval”. The medicines regulator has rejected this and said drugs would be approved faster than anywhere else in Europe.
The dispute is over planned changes to the Cancer Drugs Fund – a special pot of money just for cancer medicines. It currently pays for innovative drugs that have been deemed too expensive for the NHS as a whole. It includes medicines such as Kadcyla, which initially cost £90,000 per patient, and extends the lives of women with breast cancer by six months on average. However, the fund was a victim of its own success and has been greatly overspent. Its costs have risen to £340m in 2015-16 from an initial annual budget of £200m when it was set up in 2011.
Image shows false-coloured scanning electron micrograph of a single prostate cancer cell grown in culture
The National Institute for Health and Care Excellence (NICE) has approvedthe drug abiraterone for men with advanced prostate cancer, who have not yet had chemotherapy. The drug – developed by UK scientists – can delay the disease’s progression, help to prolong life, and provide a better quality of life with less fatigue and pain. It has been available across the UK for men after chemotherapy since 2012.
Today’s decision – a reversal of NICE’s previous ruling – brings England and Wales in line with Scotland, which approved(link is external) the drug for men pre-chemotherapy last year. NICE was able to rule favourably after the drug’s manufacturers, Janssen, provided new data showing the drug’s cost-effectiveness, and agreed a discount price for the NHS.
Under the discount, the NHS will pay for the first 10 months of treatment. For people who remain on treatment for more than 10 months, Janssen will cover the drug’s cost from the 11th month until the end of treatment.
Until now, the drug was only available to patients in England before chemotherapy through the Cancer Drugs Fund (CDF), and was the second most requested medicine on the fund. Today’s approval means that drug can now also been removed from the Fund – which is the process of being reformed – and paid for routinely by the NHS.
Cancer drugs fund will be a “managed access” fund from July
Under new scheme money due for drugs will be set aside and retained by NHS England if CDF budget is overspent
Pharmaceutical firms and cancer charity criticise changes
NHS England has confirmed that its new Cancer Drugs Fund will go live in July this year, with a provision that would force pharmaceutical companies to help cover the cost of overspends.
As previously reported by HSJ, the CDF will become a “managed access” fund, paying for new drugs for a set period before they are definitively approved or rejected by the National Institute for Health and Care Excellence.
The aim of the reforms is to make the CDF financially sustainable. Its original budget in 2010 was £200m but it has since ballooned to £340m. In December the fund was overspent by £92m for this financial year.
At its board meeting last week, NHS England confirmed that on 1 April the current list of drugs covered by the CDF will be “rolled over”, but the fund will close to new drugs until the start of the revamped scheme in July.
All existing CDF drugs will continue to be funded until NICE has been able to appraise them, and all patients currently receiving drugs will continue to get them, even if NICE eventually decides not to recommend them for continued use.
A total of £1.27bn (€1.7bn; $1.9bn) has so far been spent on the Cancer Drugs Fund in England, but no data exist to show whether it has been spent wisely, the House of Commons Public Accounts Committee says in a new report.
Despite the Department of Health noting when the fund was set up in 2010 that it was important to collect clinical outcomes from the drugs it pays for, few data exist. The department encouraged NHS trusts to collect data but did not require them to do so. When NHS England took over the fund in April 2014 it made data collection mandatory, but the committee still found gaps for 2014-15, with 93% of records having no summary of outcomes.
The health department and NHS England “have not managed the fund effectively” the report concluded.
On the positive side, the committee found that the gap between the United Kingdom and other advanced economies in the uptake of novel cancer treatments had narrowed. In 2009 use of new cancer drugs in the UK was only 45% of the average figure across 13 other countries. But by 2013 it had reached 92%, and NHS England said that it was likely to have improved still further since then. The committee considered that the fund must have contributed to this improvement, even though it applies only in England and not in the rest of the UK.