England and Canada diverge from US on cancer drug decisions

Regulatory NewsRegulatory News | 25 February 2021 |  By 

Regulatory and health technology assessment agencies in Canada and England are diverging from the US when it comes to oncology drugs. But they often rely on the same surrogate endpoints to make their decisions, according to two new analyses published in JAMA Internal Medicine.
The first study, led by Avi Cherla, MSc, of the London School of Economics and Political Science, revealed that among 68 cancer indications with accelerated approval in the US that also received market approval in the EU, just 45 indications (66.2%) were recommended by the National Institute for Health Care Excellence (NICE) for public coverage through the National Health Service. But about 60% of NICE decisions were based on surrogate endpoints like response rates and progression-free survival, rather than overall survival or quality of life benefits.
Similarly, in an analysis of Canada’s assessment of cancer drugs for coverage, Daniel E. Meyers, MD, of the University of Calgary and colleagues found that just 75% of the submissions for cancer drugs for solid tumors (78 out of 104) received a positive recommendation for coverage by the pan-Canadian Oncology Drug Review (pCODR). During the same period between 2011 and 2020, the US Food and Drug Administration (FDA) approved 163 drugs for adult solid tumors and none of the submissions that were turned down by the pCODR were rejected by the FDA. Canada also relied on surrogate endpoints in reaching its coverage decisions. In half of the positive recommendations for coverage, surrogate endpoints were used. In the other half, the median overall survival gain was 3.7 months.
The studies aim to help compare the impacts of the differing drug approval and coverage systems in the US, England, and Canada. In England and Canada, drugs are assessed for efficacy and safety by one agency, while a second agency evaluates the clinical benefit against the cost effectiveness, and other factors, in determining coverage for the drug. In the US, coverage decisions, which are made by the Centers for Medicare and Medicaid Services and private insurers, are virtually automatic since cancer drugs are a protected class of medications.
“The differences between the three countries raise a key policy question: does the current US system of drug approval and reimbursement speed access to better drugs and lead to better outcomes for patients with cancer?” Vinay Prasad, MD, MPH, of the University of California San Francisco, and Myung S. Kim, MD, of the Oregon Health and Science University, wrote in an invited commentary in JAMA Internal Medicine. “The US system of approval of drugs with uncertain clinical benefit followed by mandated coverage by Medicare without any ability to negotiate on prices ensures access. It is less clear that the US system benefits patients with cancer.”
Prasad and Kim highlighted drawbacks of the current US approach to approving cancer drugs. First, the FDA frequently utilizes surrogate endpoints as the basis for accelerated approval of drugs, but at least one study suggests that using surrogate endpoints instead of overall survival only speeds drug development by about 11 months in a process that typically takes 7 years.
Another drawback of reliance on surrogate endpoints in the US approval process is that it makes it more difficult to perform cost effectiveness studies since there is no information on survival or quality of life, they noted. This has a cascading effect on other countries where cost effectiveness is a key element of coverage determinations. “These choices paradoxically lead other high-income nations to delay or abandon uptake of these medications entirely because of reservations about efficacy and value,” Prasad and Kim wrote. “The lack of information regarding clinical end points, a consequence of the low US regulatory bar, makes it more difficult for other countries to obtain the evidence they need to justify coverage.”
The recent findings also highlight a shortcoming across all three systems, namely that available cancer drugs “are not as good as physicians would hope for patients,” Prasad and Kim wrote. They noted that just 34 of 52 drugs considered by NICE had survival benefits at any point in time and in Canada the median improvement in survival from covered drugs was 3.7 months.


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Tags: Canada, FDA, HTA, NICE, oncology, pCODR, UK, US

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