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Published on: August 1, 2019
Performance of Japanese patients in registrational studies
Yasushi Goto1, Sayaka Arakawa1, Masayuki Shirasawa1
1Department of Thoracic Oncology, National Cancer Center Hospital, 5-1-1, Tsukiji, Chuo-ku, Tokyo 104-0045 Japan.
Introduction:
The accelerated development of lung cancer treatments has resulted in a single global study that is sufficient for a new agent and indication to be approved. Not all new treatments predominate globally, and differences in standards of care may influence the efficacy of treatments in the real world.
Methods:
The results from Japanese domestic trials and global trials that included a subset population of Japanese patients were evaluated for 18 genomic targeted agents and immune therapies approved after 2000. The results were collected from drug applications that were reviewed for treatment approval in Japan.
Results:
Japan is one of the first countries to approve and fully reimburse new agents around the world. Alectinib and nivolumab, which were first developed by Japanese pharmaceutical companies, were evaluated in an independent domestic trial, which resulted in their early approval. For most other indications, 1.1-15.8% of the patients who participated in pivotal registration studies were Japanese, and their treatment results were comparable to those of the overall population. Overall survival was less likely to be improved by four agents for which the post-protocol therapy might have been different in Japan than in other countries.
Conclusions:
Overall, a positive result in a global trial was emulated in Japanese patients and led to the approval of a new standard treatment in Japan. Early approvals were attained by either participating in the global registrational study or conducting a domestic phase II study. The higher efficacy of new agents may be an issue in the future, as Japanese patients had early access to the new agent and may receive better treatment after the trial.
Insights
Japanese patients generally show comparable treatment results to global populations in lung cancer trials. Early approvals for new lung cancer agents in Japan are achievable through global studies or domestic trials, ensuring timely access to novel therapies.
Area of Science:
- Oncology
- Clinical Pharmacology
- Drug Development
Background:
- Accelerated development of lung cancer treatments necessitates global studies for approval.
- Real-world treatment efficacy can be influenced by varying standards of care.
- Japan's role in global pharmaceutical research and development.
Purpose of the Study:
- To evaluate the efficacy of genomic targeted agents and immune therapies in Japanese patients.
- To compare outcomes from Japanese domestic trials with global trial data.
- To understand the approval and reimbursement landscape for new lung cancer treatments in Japan.
Main Methods:
- Analysis of 18 genomic targeted agents and immune therapies approved after 2000.
- Inclusion of data from Japanese domestic trials and global trials with Japanese subsets.
- Review of drug applications submitted for treatment approval in Japan.
Main Results:
- Japan frequently grants early approval and full reimbursement for new cancer agents.
- Domestic trials for alectinib and nivolumab led to their early approval.
- Japanese patient participation in global pivotal studies (1.1-15.8%) showed comparable results to the overall population.
- Four agents showed less overall survival improvement, potentially due to differing post-protocol therapies in Japan.
Conclusions:
- Positive global trial results are often replicated in Japanese patients, establishing new treatment standards.
- Early drug approval in Japan is facilitated by participation in global registrational studies or domestic Phase II trials.
- Ensuring equitable access and managing potential efficacy differences for new agents in Japan is crucial for future cancer treatment.
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