Cancer patients will miss out on exciting new treatments after NICE passed up a golden opportunity to ensure its evaluation methods support approval of the most innovative, potentially game-changing drugs.
The Institute of Cancer Research, London, said NICE's proposed reforms to its methods for assessing drugs for the NHS fell short of what was needed and in one important respect could make access to new cancer drugs worse.
Experts at the ICR said too little had been done to favour drugs with innovative mechanisms of action, speed up approval of new drugs, or remove barriers to drug approval for rare diseases such as children's cancer.
They warned that 'minor tweaks' proposed in some areas would not be enough to incentivise pharmaceutical companies to take the risks needed to create truly innovative new treatments – and that patients with cancers of unmet need would continue to miss out.
Denied new treatments
The ICR also believes it is essential that NICE properly trials one major change it is proposing – to uprate drugs according to the severity of the disease they treat, but remove criteria favouring drugs where patients are at the end of life. Otherwise, there is a risk that cancer patients at the end of their lives could be denied new treatments that might extend their time with loved ones.
The ICR is welcoming some of the changes NICE has proposed – especially its recommendation that review panels should accept more uncertainty in data when considering new medicines. But it wants to see further consideration and movement in several important areas:
- Greater recognition of the barriers for rare diseases.
Generating the statistical evidence needed for approval is challenging for drugs treating rare diseases. NICE takes into account this barrier for extremely rare diseases, but the ICR believes it should be doing so for a much wider group of rare diseases, including cancers in children.
- Risks of removing the end-of-life modifier
NICE's end-of-life criteria have enabled many thousands of cancer patients to access life-extending drugs on the NHS. The ICR is urging NICE to carry out a pilot of the impact of switching end-of-life criteria for a disease severity modifier, to ensure there is no unintended consequence for cancer patients.
- Prioritising innovative medicines
It is positive that NICE will now apply greater flexibilities when considering uncertainty in the evidence base for a new treatment. But NICE should be particularly willing to accept uncertainty in data, or a higher price, when evaluating treatments that are truly innovative in their mechanism of action. Innovative new drugs and combinations are essential to overcome cancer evolution and drug resistance.
- Using surrogate measures of survival
NICE has made some welcome steps in recognising the importance of wider sources of data, rather than simply relying on overall survival data from clinical trials. But it is disappointing that NICE will not seek to establish 'surrogate' measures of overall survival, which can help to predict whether a new treatment is likely to help patients live longer. Surrogate measures can help approve cancer drugs more quickly, based on earlier trial data.
'NICE has missed a golden opportunity'
Professor Kristian Helin, Chief Executive of The Institute of Cancer Research, London, said:
"We believe NICE has missed a golden opportunity to improve the lives of people with cancer by making it easier to access exciting new treatments on the NHS.
"The medical research community has seen NICE's review of its evaluation methods as a hugely important opportunity to ensure it keeps pace with the science and removes the barriers patients face in gaining access to new drugs.
"We are disappointed that in many areas NICE is making minor tweaks rather than bold reforms. We want to see an appraisal system that incentivises the discovery and development of the most cutting-edge new treatments and technologies, and provides hope for patients with cancers that are rare or especially hard to treat.
"We believe that NICE should be favouring the most scientifically innovative drugs, and levelling the playing field by making it easier for children with cancer, and other patients with rare diseases, to gain access to new treatments. And NICE should be establishing early measures that help to predict longer survival, so it can make innovative new treatments available to patients more quickly.
"We are also concerned that the removal of the end-of-life modifier for life-extending treatments will stop some patients with advanced cancer benefiting from the latest scientific advances. We would urge NICE to carry out a full evaluation of the potential impact of replacing the end-of-life modifier with a disease severity modifier before implementing this change, to ensure it does not have unintended consequences or disadvantage certain groups of patients."