Drug Information Q&A Q52. How is the pharmaceutical industry tackling "unapproved drugs"?
Answer
The Japan Pharmaceutical Manufacturers Association (JPMA) has established an organization to support companies developing "unapproved drugs" and is actively working to make unapproved drugs available in Japan.
Explanation
Unapproved drugs" are drugs that have been approved overseas but not yet approved in Japan.
Even drugs that are approved and used in Japan may not be approved for the uses (indications) that are recognized in other countries. This is called "unapproved indications (off-label use)" and, like unapproved drugs, is a major problem for patients.
Currently, if a patient wishes to use an unapproved drug, he or she must import it on his or her own and bear the related costs.
In addition, if a patient receives treatment with an unapproved drug at a hospital, all drug costs, consultation fees, and laboratory fees that are normally covered by insurance cannot be billed by insurance. In such cases, the patient may have to pay the entire cost of the treatment. Furthermore, if you should experience side effects from using an unapproved drug, the government's Adverse Reactions Relief System (see Q13) will not apply.
Thus, in addition to the problem of not being able to use unapproved drugs and not being able to treat patients, even when they do use them, they face the problem of bearing a large cost burden.
On the other hand, some unapproved drugs have a very limited number of patients, and the long time required to conduct clinical trials and the large amount of research and development costs involved can be too much for a single pharmaceutical company to handle.
Because of the complexity of the various factors involved in the problem of unapproved drugs, it is essential that industry, government, and academia work together to solve the problem.
The Japan Pharmaceutical Manufacturers Association (JPMA) established a project to address these issues, and in 2009 launched the "Center for Unapproved Drugs Development Support". The Center provides professional and technical support and advice, as well as R&D funding, to companies working on the development of unapproved drugs at every stage from research and development to manufacturing and marketing. (As of March 31, 2013, the Center has supported the development of 14 products.)
Meanwhile, in April 2010, the Ministry of Health, Labour and Welfare (MHLW) established the "Review Committee on Unapproved Drugs and Off-label Drugs of High Medical Need" in addition to the introduction on a trial basis of an additional fee to promote the creation of new drugs and the resolution of off-label drug use. The purpose of this study group is to help promote the development of unapproved drugs and off-label indications by pharmaceutical companies by evaluating the medical necessity of unapproved drugs and off-label indications, and by confirming whether they are applicable to applications based on public knowledge* and whether additional tests are necessary to apply for approval. Of the 274 unapproved and off-label drugs for which the committee requested pharmaceutical companies to develop them, 133 have already been approved and marketed, 33 have already been filed for approval, and others are currently under development.
As part of its social mission and contribution to society, the pharmaceutical industry is not limited to the research and development of new drugs, but is also committed to providing drugs that are highly necessary for the treatment of patients.
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As of December 2023Public knowledge application: A system that allows for the approval of indications or effects, etc., for off-label use of an already approved drug without the need to conduct clinical trials, in whole or in part, anew, if the drug is recognized as publicly known in the medical and pharmaceutical sciences on the basis of scientific evidence.
Chart/Column
Initiatives for Unapproved Drugs
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As of December 2023Public knowledge application: A system that allows for the approval of indications or effects of an already approved drug for off-label use without the need to conduct a new clinical trial, in whole or in part, if the drug is recognized as publicly known in terms of medical and pharmaceutical sciences on the basis of scientific rationale.
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