Table 4.
Evidence level classifications in guidance issued in Japan, the United States, and Europe
| Criterion | Draft revision | Criterion details | Example of measures based on evidence level: draft revision |
|---|---|---|---|
| Response-related evidence level classifications in Japan*1 | |||
| There is a drug approved in Japan for a specific tumor type | A | There is a drug targeting a specific biomarker approved in Japan for a specific tumor type | If there are drugs approved in Japan, administration of the drug is recommended also based on the results obtained with the companion diagnostic, but in accordance with the various guidelines. Otherwise, the use of evaluation treatments, such as clinical trials, advanced medical care, and the off-label use of a drug listed on the National Health Insurance drug price list, or the use of the system for providing medical expenses combined with treatment outside insurance coverage, such as patient-proposal healthcare services, is recommended, but in accordance with the various guidelines |
| There is a drug approved by the FDA for a specific tumor type | A | There is a drug targeting a specific biomarker approved by the FDA for a specific tumor type | |
| A specific tumor type is included in the guidelines | A | The guidelines include the use of a drug targeting a specific biomarker for a specific tumor type | |
| There have been clinical studies and meta-analyses of high statistical reliability and is a consensus among experts for a specific tumor type | B | Regarding the use of a drug targeting a specific biomarker, there have been supportive data from clinical studies and meta-analyses of high statistical reliability and is a consensus among experts for a specific tumor type | Because there is a scientific basis, the use of evaluation treatments, such as clinical trials, advanced medical care, and the off-label use of a drug listed on the National Health Insurance drug price list, or the use of the system for providing medical expenses combined with treatment outside insurance coverage, such as patient-proposal healthcare services, should be considered |
| There is a drug approved in Japan or by the FDA for another tumor type | C | There is a drug targeting a specific biomarker approved in Japan or by the FDA for another tumor type | Because there is a scientific basis, the use of evaluation treatments, such as clinical trials, advanced medical care, and the off-label use of a drug listed on the National Health Insurance drug price list, or the use of the system for providing medical expenses combined with treatment outside insurance coverage, such as patient-proposal healthcare services, should be considered |
| There have been clinical studies and meta-analyses of high statistical reliability and is a consensus among experts for another tumor type | C | Regarding the use of a drug targeting a specific biomarker, there have been supportive data from clinical studies and meta-analyses of high statistical reliability and is a consensus among experts for another tumor type | |
| Usefulness has been shown in a small clinical study in any tumor type | C | Regarding the use of a drug targeting a specific biomarker, usefulness has been shown in a small clinical study in any tumor type | |
| Usefulness has been shown in case reports in any tumor type | D | Regarding the use of a drug targeting a specific biomarker, usefulness has been shown in case reports in any tumor type | Although a scientific basis is weak, the use of evaluation treatments, such as clinical trials, advanced medical care, and the off-label use of a drug listed on the National Health Insurance drug price list, or the use of the system for providing medical expenses combined with treatment outside insurance coverage, such as patient-proposal healthcare services, should be considered based on the consensus of the expert panel |
| Usefulness has been reported in preclinical studies (in vitro or in vivo) | E | Regarding the use of a drug targeting a specific biomarker, usefulness has been shown in preclinical studies (in vitro or in vivo) in any tumor type | Although there is some scientific basis, the use of the drug cannot be aggressively recommended because it has not been administered to humans. However, if the marker is being examined in a clinical trial, enrollment in the trial should be considered based on the consensus of the expert panel. Because the evidence level is expected to increase in the near future, the information is registered with C-CAT |
| Known to be related to cancerous changes | F | A specific biomarker is known to be related to cancerous changes | Although there is no scientific basis related to treatment selection at this point, if the marker is being examined in a clinical trial, enrollment in the trial is considered based on the consensus of the expert panel. To enhance treatment options by promoting an improvement in the evidence level through the accumulation and utilization of information, the information is registered with C-CAT |
| Known to be related to drug resistance | R | A specific biomarker is known to be related to drug resistance | Treatment selection is determined based on the consensus of the expert panel |
| Index summary | Number | Index details |
|---|---|---|
| Indices of drug accessibility | ||
| There is a drug approved in Japan for a specific tumor type | 1 | There is a drug targeting a specific biomarker approved in Japan for a specific tumor type |
| There have been clinical studies in Japan for a specific tumor type | 2 | There have been clinical studies in Japan in which a specific biomarker serves as an inclusion criterion for a specific tumor type |
| There is a drug approved in Japan for another tumor type (off-label use) | 3 | There is a drug targeting a specific biomarker approved in Japan for another tumor type |
| There have been clinical studies overseas for a specific tumor type | 4 | There have been clinical studies overseas in which a specific biomarker serves as an inclusion criterion for a specific tumor type |
| There is a drug approved by the FDA for any tumor type | 5 | There is a drug targeting a specific biomarker approved by the FDA for any tumor type |
| None of the above | 6 | None of the above apply |
NB the numbers do not indicate an order of precedence