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certain categories of breast cancer, the National Institute for Health and Care Excellence (NICE) has announced in new draft guidance | OnMedica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.