Long gone are the days when an oncologist could look at the evidence base for a drug, decide on its suitability, and prescribe it. Now, the discussion is commonly about cost and, more specifically, about willingness to pay.
The result? Although more and more targeted oncology drugs are being approved for use on clinical grounds, patients are less and less likely to have access to them because of cost.
"Cancer drugs are becoming increasingly expensive...primarily because a lot of the drugs that are coming through are what we could call a first in class," said Sarah Rawlings, PhD, assistant director of policy and information at Breakthrough Breast Cancer, a British charity.
These new drugs often target only cancer cells, so generally have a milder adverse-effect profile than conventional chemotherapy. However, she noted, the cost can be prohibitive.
This is especially true in publicly funded healthcare systems, such as that in the United Kingdom. Medical costs for all citizens are covered by the National Health Service (NHS), but medical procedures, devices, and drugs have to be approved by the National Institute for Health and Care Excellence (NICE).
"In the past few years, we've seen NICE approve fewer and fewer cancer drugs, and often they state that they are not being approved because they are too expensive for use on the NHS," Dr Rawlings explained.
If a drug is not approved by NICE, the only way a patient can obtain it is to pay for it privately, but because of the high price of these drugs, they are out of reach for most people. This has provoked frustration and outrage among patients and physicians. One temporary answer to this problem is the Cancer Drugs Fund (CDF).
This England-only body was set up in 2010, as reported by Medscape Medical News, to give cancer patients access to drugs not routinely covered by the NHS, either because they have been rejected by NICE for being too expensive or because they have not yet been approved.
Prime Minister David Cameron promised to establish the CDF after meeting Clive Stone, a retired bank manager who campaigned for better access to cancer drugs after being refused access to sunitinib (Sutent) for his kidney cancer. Although NICE eventually agreed to fund sunitinib on the NHS, they would not cover any second-line therapies. It was the campaign for access to these second-line therapies that led to the founding of the CDF.
So far, the CDF has allowed 55,000 patients, in four regional areas, to be prescribed from a national list of 41 drugs for a range of cancers. Data from NHS England indicate that the most commonly accessed drug has been bevacizumab (Avastin), followed by abiraterone (Zytiga), bendamustine, cetuximab (Erbitux), everolimus (Afinitor), eribulin (Halaven), aflibercept (Zaltrap), enzalutamide (Xtandi), pemetrexed (Alimta), sorafenib (Nexavar), axitinib (Inlyta), cabazitaxel (Jevtana), pertuzumab (Perjeta), ruxolitinib (Jafaki), bortezomib (Velcade), pomalidomide (Pomalyst), lapatinib (Tyverb), brentuximab (Adcetris), lenalidomide (Revlimid), and pazopanib (Votrient).
A 'Relief" to Have Access
To give an indication of the impact the CDF, Andrew Protheroe, MD, from the Department of Oncology at the University of Oxford, explained how difficult it had been to access these drugs. "If we go back to what it was like before the national Cancer Drugs Fund came into place, it was more about individual patient reviews, according to your local PCTs [primary care trusts], and that was much, much more difficult," he said.
"The perceived wisdom, certainly around Oxfordshire, was that these [drugs] are for cases of exceptionality, but exceptionality wasn't defined in terms of age or social circumstances, so it was very hard to find a way in," he noted.
"When we suddenly have various studies showing the benefits of some of these drugs but we can't prescribe them," Dr Protheroe explained, "it makes it very, very hard."
"As clinicians, we want to treat our patients to the best of our abilities. To a certain extent, cost doesn't come into it; it's about the clinical benefit of these drugs," he noted.
The introduction of the CDF has, in clinical terms, been positive. "Because of the Cancer Drugs Fund, many patients are able to benefit from these new drugs," Dr Rawlings reported. For clinicians, the impact was no less important. As Dr Protheroe observed, "suddenly to be able to use drugs was a huge benefit for us. A relief, in a way."
A recognized cancer specialist, supported by the clinical cancer lead of the specialist's NHS trust, must apply to prescribe a drug from the CDF approved list. If there is an agreed cohort policy for a particular drug, applications are submitted online and, if the criteria are met, funding will be confirmed. For drugs outside the cohort policies, applications will be assessed by a panel in the region, using a national assessment process.
Dr Protheroe is a regular applicant to the CDF. "I have used it three times in the last week," he said. "For urological cancers, we are quite heavy users because of the drugs abiraterone, enzalutamide, and radium-223 dichloride. I would say I probably use it for the ones that are on the cohort for the CDF four or five times a month."
"The process is, I have to say, very straightforward," he explained. "And it's been a lot better since we have been able to use the Web-based program." Dr Protheroe noted that, for agreed cohort policy drugs, the process is simply one of patient registration followed by an instantaneous answer. "For the individual approvals for slightly more esoteric requests, that is a bit more involved," he said. "But, again, it's quite a straightforward process."
Undermining Concepts of Equality
However, the CDF is not without its detractors. John Appleby, chief economist for health policy at The King's Fund, observed that, fundamentally, the CDF undermines the concept of all patients being equal and creates its own ethical dilemmas.
Others have argued that by establishing a fund for cancer drugs, and not other drugs that are also expensive and could be of benefit to patients with serious disorders, British policymakers are advocating what has been described as "a kind of exceptionalism for cancer therapy," as reported by Medscape Medical News.
On the more prosaic issue of money, the CDF overspent by £30 million in the 2013/14 financial year, and the forecast for 2014/15 is that it will overspend by £40 million on its current annual budget of £200 million. This led the government to announce in August that the budget will be increased to £280 million per year. However, it is estimated that, by the time the CDF's mandate runs out at the end of March 2016, it will have cost more than £1 billion.
"It does look like some of the drugs will come off the fund [to deal with the overspending]," Dr Rawlings reported. "That's a real blow for access to drugs that your doctor may think are particularly useful for you and your particular type of cancer."
This is against a backdrop of campaigners looking to widen the list of approved treatments to encompass, for example, radiotherapies such as the gamma knife. Indeed, Prime Minister Cameron promised Stone personally that the gamma knife would be approved for use on the CDF. That promise has failed to materialize, however.
"I sat on the original clinical reference group that agreed to the gamma knife two years ago, and now people are not getting it," Stone explained. "They made me wait, with tumors growing in my brain. They made me wait 6 weeks for no reason at all."
"I've had to pay two lots of £15,000 to have gamma knife done on my head, because the local PCT said they wouldn't fund it," he explained. "They were sitting on a £2 million surplus at the time.... I now have to keep saving all my pension in case I need it again."
Only in England
Another issue that has been raised about the CDF is that it is confined to England; there is no equivalent body in Scotland, Wales, or Northern Ireland. In November, Malcolm Mason MD, dean of research at the College of Biomedical and Life Sciences, Cardiff University, in Wales, will chair a debate at the National Cancer Research Institute Cancer Conference in Liverpool on the implications of the CDF for the rest of the United Kingdom.
"It would be a far preferable situation if you did have something that was the same nationally," Dr Malcolm said. "We are all increasingly uncomfortable about differences in availability across the United Kingdom. It comes into sharp relief in parts of Wales, where some people on the borders may be going across to have treatment in an English center," he said.
However, he does not necessarily believe that the answer is simply to extend the CDF to the rest of the United Kingdom. "It's not as straightforward as simply saying that the Cancer Drugs Fund is undoubtedly the best way. There are questions about how drugs are selected and prioritized within it, and there are questions about whether the absence of a Cancer Drugs Fund means that there is no way of achieving the same sort of thing for patients."
Extending a Temporary Solution
But what should replace the CDF when its mandate runs out in 2016? And should it be replaced?
Dr Rawlings would like to see the CDF mandate extended to the end of the next government, in 2020, and replaced by a more sustainable solution. However, she believes that finding one will not be straightforward.
"If there was an easy solution to this, we would already have it," she said. "There isn't, and part of the campaign that we have started around this particular issue is to get a commitment that the government, the pharmaceutical industry, and NICE will work together to really bottom out what that long-term solution is. Breakthrough Breast Cancer is incredibly committed to helping as much as we can."
Nevertheless, the CDF has, in its short life, had a huge impact on patients' lives. "The good thing about the Cancer Drugs Fund is that, according to all the figures I've seen, we now have 55,000 patients using drugs on it," Stone said. "That doesn't mean to say that they've saved their lives, but what I say to everybody is, 'if you have that extra time, you don't know what improvements are going to be made'."
"There are a lot of good things happening in cancer now. If we can get it to the stage of it being just a chronic disease for most cancers...that would be an incredible thing," he said.
"I keep thinking of the children, you know. We've got to keep pressing on," Stone explained.
Medscape Medical News © 2014 WebMD, LLC
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Cite this: Cancer Drugs Fund in England: Plugging a Gap in Cancer Care - Medscape - Oct 09, 2014.