Plans to reform patient access to cancer drugs not approved for routine use in the United Kingdom have been unveiled by the National Health Service (NHS) in England and the health watchdog, the National Institute for Health and Care Excellence (NICE).
Under the proposals, which have been made available for a 12-week consultation, the controversial Cancer Drugs Fund (CDF) will be restructured. This will allow patients to receive novel drugs that cannot yet be approved for a limited period while NICE gathers more data on their cost-effectiveness.
Sir Andrew Dillon, chief executive of NICE, said that the proposed changes to the CDF will make it easier to collect longer-term data on the benefits of drugs.
He said in a release: "Our joint proposals will ensure that the Cancer Drugs Fund is used to provide patients with promising medicines at a cost which reflects the uncertainty about their effectiveness and cost effectiveness and at the same time, generate additional data to help the NHS make a longer term decision on whether and how to use them."
The consultation was published online by NHS England and NICE on November 19 and runs until February 11, 2016. The new scheme resulting from the consultation comes into operation in April 2016.
The CDF was set up in 2010 by the previous government to provide access to cancer drugs not approved by the NICE, which in the United Kingdom are otherwise available only to those who are able to pay for them privately.
Welcomed by physicians, charities, and patient groups, the CDF has allowed more than 72,000 cancer patients to receive drugs that they would otherwise have had difficulty receiving.
However, the fund has courted controversy since its inception, with experts questioning its ethical basis and numerous cost overruns resulting in its budget allocation increasing dramatically.
The CDF's first budget was set at £200 million (US$300 million). However, as previously reported by Medscape Medical News, this grew to £280 million (US$419.9 million) in 2014/15 and to an estimated £340 million (US$509.9 million) in 2015/16.
To deal with the overruns, the list of cancer indications that the CDF was willing to pay for was slashed from 84 to 59 at the start of this year, affecting 21 cancer drugs and removing nine medications.
This was followed by the removal of 16 drugs for 23 indications on November 4 in an effort to stop the cost overrun from reaching an estimated £410 million (US$614.9 million) this year.
Noting that the next 5 years will likely see the launch of a number novel cancer drugs, including some offering huge benefits and others providing few improvements in outcomes but at an enormous cost, Simon Stevens, chief executive of NHS England, said: "The new Cancer Drugs Fund offers a route for sorting out the wheat from the chaff."
For the drug companies that are "willing to price their products affordably while sharing transparent information about 'real world' patient benefit, the new CDF will offer a new fast-track route to NHS funding," he added.
In line with the recent recommendations from the Cancer Taskforce, the new system will see the CDF fully integrated into the NICE appraisal system. It will act as a transitional fund, with entry and exit criteria, for drugs that have insufficient evidence for a routine recommendation.
Under the new process, NICE will issue draft guidance for cancer drugs before market authorization. Final guidance will follow within 90 days of the time authorization is granted. This guidance will recommend that some drugs be used routinely; it will reject other drugs, and it will recommend others for use within the CDF.
For those drugs earmarked for use within the CDF, data will be collected on patient outcomes, typically for up to 24 months, to expand the available evidence base. At that point, NICE will either recommend the drug for routine use or make it available only through individual patient funding requests.
The proposals received a cautious welcome. Baroness Delyth Morgan, chief executive at the UK charity Breast Cancer Now, which was formed through a merger of Breast Cancer Campaign and Breakthrough Breast Cancer, expressed concern that the consultation process is not long enough to allow much-needed reforms to be properly formulated.
She commented in a release: "We've been on standby to feed into this consultation for months now and we're hopeful that these proposals will lead to improvements to a system in desperate need of reform.
"That said, with just 5 months left until the current Fund expires, we remain extremely concerned that there is limited time to review these proposals and consult in a meaningful way."
She continued: "The problems with our system of drug access as it stands are huge. That's why it's vital that there is sufficient time for any proposed changes to be thoroughly assessed before they are put in place.... We simply hope it won't be a case of too little, too late."
The leading charity Cancer Research UK emphasized in its science blog that reforming the entire system for accessing cancer drugs was more important than simply tackling the CDF.
Although acknowledging that "getting it right" will be difficult, Zoe Molyneux, senior policy advisor at Cancer Research UK, suggested that there were four main areas for consideration.
First, the UK-wide Early Access to Medicines Scheme should be expanded. So far, it has allowed patients to receive two drugs before they were licensed. Molyneux notes that such an expansion would require agreements between the government, the NHS, and pharmaceutical companies.
Second, the CDF should become a vehicle for paying for drugs between the time they are licensed and the time NICE makes a decision on their cost-effectiveness.
Third, there should be a fundamental review of the way in which the NICE assesses cancer drugs.
Finally, NICE should be able to offer conditional approvals that allow patients to benefit from promising drugs. This will force companies to collect more data on their drugs while allowing NICE to refuse drugs that are proven not to be cost-effective.
Other reforms should include a national molecular diagnostic service to offer tests for genetic mutations, argues Molyneux, along with increased flexibility over individual funding requests for patients in exceptional circumstances.
Emlyn Samuel, senior policy manager at Cancer Research UK, has welcomed some aspects of the proposals. "The crucial thing is that it looks like the decision-making on what drugs should enter the Cancer Drugs Fund evaluation, and on whether the drugs become routinely available on the NHS, is made by NICE," he told Lancet Oncology.
NHS England/NICE: Consultation on Proposals for a New Cancer Drugs Fund (CDF) Operating Model From 1 April 2016. Published online November 19, 2015. Full text
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Cite this: Controversial UK Cancer Drug Fund Will Be 'Reformed' - Medscape - Dec 03, 2015.