Japanese cancer patients will soon have rapid access to any drug that has been approved anywhere in the world, regardless of whether or not it has been approved in Japan.
The move, being implemented by the office of the Japanese prime minister, is intended to combat delays in drug access and avert a perceived crisis in cancer care.
However, in a comment published in the March issue of the Lancet Oncology, a group of Japanese cancer experts claims that the changes, which come into effect next year, are not only unnecessary, but could have the severe consequences for Japanese healthcare.
The critics list several "sources of concerns" related to the upcoming system, and predict that the changes will mean that "companies will be reluctant to develop new drugs [and] will be able to make profits without investing money in drug development."
With the implementation of the system, known as "regulatory reform enforcement planning," in 2016, a patient can be prescribed a drug approved elsewhere in the world during the 6-week period after assessment by a team of physicians or surgeons. Patients will have to pay for the drug themselves, but routine clinical care will be covered by the universal healthcare system.
The changes are a response to what has been described as a crisis in cancer treatment in Japan. Patients have been frustrated by delays in marketing approval for oncology drugs that have been available in other countries for up to several years.
Politicians and the media have hailed the system as a guarantee that patients will have access to novel drugs more quickly, and at potentially lower costs.
To investigate the veracity of these claims, and to test the presumptions that underly the changes, Yasuhiro Fujiwara, MD, PhD, director-general of the Strategic Planning Bureau at the National Cancer Center in Tokyo, and his colleagues surveyed the status of unapproved oncology drugs in March 2014.
They found that, of 41 drugs not approved by the Japanese Pharmaceuticals and Medical Devices Agency (PMDA), 14 were approved by both the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA), 21 were approved by one or the other agency, four were rejected by either the FDA or the EMA, and two drug applications had been withdrawn by the pharmaceutical company.
Twelve of the 41 drugs were being used off-label in Japan.
Of the 41 drugs, 16 were for hematologic malignancies and seven were for skin cancers. Urologic malignancies were also well represented on the list.
Interestingly, hematologic malignancies and skin cancers are either rare or have very small patient populations in Japan, Dr Fujiwara told Medscape Medical News.
The majority of cancer patients in Japan have breast, lung, or colon cancer, and there is currently no lag in drug availability for these diseases, he explained.
For 24 of the drugs, the average monthly cost is more than ¥1,000,000 (US$8340), according to the authors. "Given that patients with cancer typically undergo several courses of treatment, many patients cannot afford such drugs without some type of support," they write.
"I think the politicians are misunderstanding the situation," Dr Fujiwara said. "They don't get precise information about the approval and development status of each unapproved drug."
"They think if they introduce a new kind of system, they will change the present situation. But I don't think so," he added.
Dr Fujiwara emphasized that the delay in the approval of drugs by the PMDA is, in fact, "very, very small right now," with a timeframe similar to that seen for the FDA and less than that seen for the EMA.
"The problem exists in the developmental phase," he explained. It takes much more time to start clinical trials in Japan than in the United States or European Union.
"Usually, overseas companies start clinical trials in the United States first," he noted. Second, they start in Europe, and third, they start in Japan.
"That causes a submission delay in Japan," he noted, and "is the main cause of the drug lag we encounter right now in Japan."
Instead of the new system, Dr Fujiwara believes that drugs that have been approved elsewhere should be made available under a conditional-approval system, similar to the ones that operate in the United States and the European Union.
This would mean that unapproved or off-label drugs are used in a clinical trial setting first, and then, after 1 or 2 years, the manufacturers could apply for standard approval, as they do now.
"The conditional-approvals system is very important," Dr Fujiwara said. With it, drugs will be available under some conditions before safety and efficacy data are mature. The other benefit is that it keeps drug companies "onside," because they will have to supply the drugs and cooperate with the researchers conducting the clinical trial.
This eliminates a potential pitfall of the new system, in which it could be in a pharmaceutical company's interest to stay outside the Japanese approval system and rely on approval in the United States or European Union.
Another potential issue associated with the changes is that because it is patient-based, it will be difficult to collate population-wide data. Consequently, any attempts to assess the safety and efficacy of the unapproved drugs will be hampered, Dr Fujiwara pointed out.
Finally, the changes highlight the fact that, in the Japanese healthcare system, all drugs that have been approved for use are automatically reimbursed. Dr Fujiwara explained that the government is considering changing the system to one similar to that in the United Kingdom, by introducing a body like the National Institute of Health and Care Excellence (NICE).
Although having a regulatory body conduct cost/benefit analyses could help to rationalize healthcare spending, Dr Fujiwara and his colleagues fear that it could end up pitting the government against the regulator.
This could eventually lead to the establishment of a body similar the Cancer Drugs Fund, which would allow patient access to approved drugs that are deemed too expensive or insufficiently effective. But that could create its own set of ethical dilemmas and contradictions, as reported by Medscape Medical News.
"This would undoubtedly tarnish the reputation of Japan's universal healthcare system and its sustainability, because private health insurance coverage would prevail over public coverage," the authors conclude.
The authors have connections with numerous pharmaceutical companies, and received grants from the Japanese Ministry of Health Labour and Welfare, as detailed in the publication.
Lancet Oncol. 2015;16:251-252. Abstract
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Cite this: Solution for Japanese Cancer Crisis 'Counterproductive' - Medscape - Apr 01, 2015.