On March 23, 2021 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it has obtained approval from the Ministry of Health, Labour and Welfare (MHLW) for FoundationOne Liquid CDx Cancer Genomic Profile as the liquid biopsy (LB) test that provides comprehensive genomic profiling (CGP) for solid tumors on March 22, 2021 (Press release, Chugai, MAR 23, 2021, View Source [SID1234577004]). The test was also approved for use as a companion diagnostic (CDx) for certain approved targeted therapies in Japan, making it the first MHLW-approved blood-based test with both CDx and solid tumor CGP indications.

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"The approval of FoundationOne Liquid CDx Cancer Genomic Profile, a blood-based CGP option, has a significant meaning for Chugai that aims to advance personalized healthcare based on the genomic profile of a patient’s tumor," said Chugai’s President and CEO, Dr. Osamu Okuda. "In cancer treatment that continues to evolve day-to-day, it enables us to support a wider range of patients to help inform treatment decisions aligning the patient’s status and stage of treatment by utilizing both tissue-based and blood-based tests. We are committed to preparing for the launch of the test as soon as possible and also will continue to expand companion diagnostic functions with the aim of maximizing the value provided to patients."

Developed by Foundation Medicine Inc. based in Cambridge, USA, FoundationOne Liquid CDx Cancer Genomic Profile is a next-generation sequencing based in vitro diagnostic device using blood samples from advanced cancer patients with solid tumors. It identifies genomic alterations in 324 cancer-related genes through detection of blood circulating tumor DNA (ctDNA) in blood. FoundationOne Liquid CDx Cancer Genomic Profile provides an integrated test report informing alterations matched to MHLW-approved targeted therapies.

As a leading company in the field of oncology, Chugai is committed to realize advanced personalized healthcare in oncology and contributing to patients and healthcare professionals through improving access to CGP.

Approval information

Brand name FoundationOne Liquid CDx Cancer Genomic Profile
Japanese medical device nomenclature (JMDN)
Software for gene variants analysis (for cancer genome profiling)
Software for analysis of somatic cell gene variants (for eligibility identification of antineoplastic agents)
Intended uses or indications
The Product is used for comprehensive genomic profiling of blood samples in patients with solid tumors.
The Product is used for detecting gene mutations and other alterations to support the assessment of drug indications listed in the table below.
Alterations Cancer type Relevant drugs
Activated EGFR alterations Non-small cell lung cancer (NSCLC) afatinib dimaleate, erlotinib hydrochloride, gefitinib, osimertinib mesilate
EGFR exon 20 T790M alterations osimertinib mesilate
ALK fusion genes alectinib hydrochloride, crizotinib, ceritinib
ROS1 fusion genes entrectinib
NTRK1/2/3 fusion gene Solid tumors entrectinib
Conditions for approval
The necessary measures must be taken to ensure that the product is used by a physician with adequate knowledge and experience of cancer genomic medicine at a medical institution with a cancer genome profiling-based medical system pursuant to the "Guidelines for the Development of Core Hospitals and Other Facilities for Cancer Genomic Medicine," and in compliance with the scope and timing of testing stipulated in the most recent guidelines, etc., of relevant academic societies.
Appropriate procedures and controls to protect personal information and up-to-date security and privacy protection measures to prevent unauthorized access must be implemented for blood samples sent to the laboratory and for information obtained from these specimens.
Quality control of input data must be performed as described in the Remarks column of the attached Application Form. Any changes to the quality control of input data as described in the Remarks column of the Application Form (excluding minor changes specified by Order of the MHLW in Article 23-2-5, paragraph (15) of the Act on Securing Quality, Efficacy and Safety of Products Including Pharmaceuticals and Medical Devices ["the Act"]) must be approved by the MHLW Minister pursuant to Article 23-2-5, paragraph (15) of the Act. Note that this approval applies mutatis mutandis to the provisions of Article 23-2-5 paragraph (17), Article 23-2-6, and Article 23-2-7 of the Act.
About FoundationOne Liquid CDx Cancer Genomic Profile
Developed by Foundation Medicine Inc. based in Cambridge, USA, FoundationOne Liquid CDx Cancer Genomic Profile is a next-generation sequencing based in vitro diagnostic device using blood samples for advanced cancer patients with solid tumors. It is intended to identify in 324 cancer-related genes through detection of blood circulating tumor DNA (ctDNA). The test is approved by the MHLW for use in cancer genome profiling to report substitutions, insertion and deletion alterations, and select gene rearrangements for short variants in 324 genes. It is also indicated for use as a companion diagnostic to identify patients who may benefit from treatment with specific targeted therapies (listed in Table above of Intended uses or indications). For the latest information about the product, including companion diagnostic indications, please refer to the prescribing information.
Trademarks used or mentioned in this release are protected by laws.

On March 23, 2021 EORTC reported that GLIOBLASTOMAS represent almost 50% of malignant primary brain tumors in adults, and range among the most lethal cancer types (Press release, EORTC, MAR 23, 2021, https://www.eortc.org/blog/2021/03/23/surviving-glioblastoma-eortc-brain-tumour-group-explores-the-factors-that-contribute-to-long-term-survival/ [SID1234577003]). Approximately three in 100,000 people a year are diagnosed with a glioblastoma. These tumors mainly affect patients in later life, but can occur in younger adults and even in children. Due to the aggressive nature of this tumor, which invades the brain by infiltration and destroys healthy brain tissue, glioblastomas lead to a significantly reduced life span with sometimes considerable loss of quality of life. Almost half of the patients die in the first year of diagnosis, despite the use of a broad therapeutical approach including brain surgery, radiotherapy and sometimes several courses of chemotherapy. Yet, a small percentage of up to 5% of all patients suffering from glioblastoma may survive for more than five years. These patients are referred to as long-term survivors.

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The reasons leading to this survival benefit in this heterogeneous patient group have not been fully identified so far. These patients are now the focus of EORTC 1419, also known as ETERNITY, a large international comprehensive study, assessing potential clinical and biological factors of this long-term survival that may contribute to improved survival in glioblastoma patients in general. Considering the rareness of glioblastoma long-term survival, a multicenter approach, involving a large number of clinical centers collecting patient data, is required. This EORTC study was made possible by a generous grant of US $2,000,000 by the Brain Tumor Funders’ Collaborative (BTFC). The BTFC is a strategic partnership of five private philanthropic and advocacy organisations in the US and Canada: the American Brain Tumor Association, Brain Tumour Foundation of Canada, Children’s Brain Tumor Foundation, James S. McDonnell Foundation, and the Sontag Foundation. EORTC 1419 comprises 33 leading centers for neuro-oncology worldwide, including centers in Europe, Australia and the US.

The participating medical researchers collect extensive clinical data from over 400 patients who survived their disease for more than five years. They record additional information including patient histories and health-related data, and perform extensive neurocognitive assessments to allow for a better understanding of the implications of the disease as well as the therapies in affected patients. Tumor tissue and blood samples from these patients will be collected to study the genetic and immunologic features of glioblastomas. Moreover, an analysis of all neuroimaging studies available from the selected patient group will be performed, assessing tumor growth patterns and development by different imaging tools. All the acquired information will later be compared to the data set of a reference cohort of glioblastoma patients who have not become long-term survivors. The collected clinical data will be inserted in a large database for further comparative analysis. In parallel, all available tissue samples will be catalogued in a central biobank in Germany, and sorted for the planned molecular and genetic investigations. The knowledge gained from this study will allow for a better understanding of the disease, and should help develop better treatment strategies for all glioblastoma patients in the future (contact: [email protected], [email protected]).

On March 23, 2021 Taiho Pharmaceutical Co., Ltd. (hereinafter "Taiho") reported that it has submitted to the Japanese Ministry of Health, Labour and Welfare a new drug application for an NK1 receptor antagonist antiemetic drug (development code: Pro-NETU; generic name: fosnetupitant chloride hydrochloride; hereinafter "fosnetupitant") for gastrointestinal symptoms (nausea and vomiting) associated with cancer chemotherapy (Press release, Taiho, MAR 23, 2021, View Source [SID1234577002]).

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Fosnetupitant is an NK1 receptor antagonist antiemetic drug developed for the prevention of chemotherapy-induced nausea and vomiting. It is a phosphorylated pro-drug preparation (injection) of netupitant, the active component. The prevention of chemotherapy-induced nausea and vomiting is considered important in clinical practice. Taiho acquired exclusive development and marketing rights for fosnetupitant in Japan under a license agreement signed in April 2011 with Helsinn Healthcare SA and has conducted clinical trials in Japan since then.

The new drug application is based on the results of a Phase III clinical trial comparing the efficacy and safety of fosnetupitant and fosaprepitant (NK1 receptor antagonist) in patients receiving highly emetogenic chemotherapy. In the trial, either fosnetupitant (235 mg) or fosaprepitant (150 mg) was administered in a single I.V. dose prior to administration of the chemotherapeutic agent, in combination with palonosetron (5-HT3 receptor antagonist) and dexamethasone. Details of the trial results will be presented at a future medical conference.

Within Taiho’s mainstay field of oncology, the company is also focusing on cancer supportive care. Under a former distribution and license agreement effective as of January 2004 with Helsinn Healthcare SA, Taiho has been marketing the 5-HT3 receptor antagonist Aloxi (generic name: palonosetron hydrochloride) in Japan since April 2010.

Taiho will work to secure approval of fosnetupitant, aiming to deliver the agent as soon as possible as a new treatment which can contribute to improving quality of life for patients, thereby contributing to comprehensive care in cancer.

On March 23, 2021 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") and MSD K.K. (Headquarters: Tokyo, President: Kyle Tattle, "MSD"), a subsidiary of Merck & Co., Inc., Kenilworth, N.J., U.S.A., reported that LENVIMA (generic name: lenvatinib mesylate), the multiple receptor tyrosine kinase inhibitor discovered by Eisai, has been approved in Japan for the additional indication of treatment of unresectable thymic carcinoma (Press release, Eisai, MAR 23, 2021, View Source [SID1234577001]). This marks the first approval for LENVIMA for unresectable thymic carcinoma in Japan.

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The approval was based on the results of an open-label, single-arm, multicenter, investigator-initiated clinical phase II study (REMORA study) conducted in 8 centers across Japan including the National Cancer Center Hospital, evaluating LENVIMA as a single agent in 42 patients with thymic carcinoma previously treated with at least one platinum-based regimen.

LENVIMA met the study’s primary endpoint of objective response rate (ORR) as assessed by independent imaging review, demonstrating an ORR of 38.1% (90% confidence interval (CI): 25.6-52.0). The lower value of the CI exceeded the pre-specified statistical criteria with a threshold ORR of 10%. The most common three treatment-related adverse events were hypertension (88.1%), proteinuria (71.4%), and palmar-plantar erythrodysesthesia syndrome (69.0%), which is consistent with the safety profile observed in the previously approved indications.

Thymic carcinoma is an extremely rare disease with low prevalence. It is estimated that there are about 140 patients in Japan. For unresectable thymic carcinoma, platinum-based first-line therapy is recommended. However, since the standard treatment has not yet been established for second-line or later therapy, this is an area of high unmet medical need. In June 2020, LENVIMA received orphan drug designation in Japan for unresectable thymic carcinoma.

Eisai and MSD have been collaborating through the provision of information on LENVIMA in Japan since October 2018, and will work together to expedite the maximization of LENVIMA’s contribution to patients with cancer.

1. About LENVIMA (generic name: lenvatinib mesylate)
LENVIMA, discovered and developed by Eisai, is a kinase inhibitor that inhibits the kinase activities of vascular endothelial growth factor (VEGF) receptors VEGFR1 (FLT1), VEGFR2 (KDR), and VEGFR3 (FLT4). LENVIMA inhibits other kinases that have been implicated in pathogenic angiogenesis, tumor growth, and cancer progression in addition to their normal cellular functions, including fibroblast growth factor (FGF) receptors FGFR1-4, the platelet derived growth factor receptor alpha (PDGFRα), KIT, and RET. In syngeneic mouse tumor models, lenvatinib decreased tumor-associated macrophages, increased activated cytotoxic T cells, and demonstrated greater antitumor activity in combination with an anti-PD-1 monoclonal antibody compared to either treatment alone. Currently, LENVIMA has been approved for monotherapy as a treatment for thyroid cancer in over 70 countries including Japan, in Europe, China and in Asia, and in the United States for radioiodine-refractory differentiated thyroid cancer. In addition, LENVIMA has been approved for monotherapy as a treatment for unresectable hepatocellular carcinoma in over 65 countries including Japan, the United States, in Europe, China and in Asia. It is also approved in combination with everolimus as a treatment for renal cell carcinoma following prior antiangiogenic therapy in over 60 countries, including the United States, in Europe and in Asia. In Europe, the agent was launched under the brand name Kisplyx for renal cell carcinoma. In addition, it is approved in combination with KEYTRUDA as a treatment for advanced endometrial cancer that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR), who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation in over 10 countries including the United States, Canada and Australia. Continued approval for this indication is contingent upon verification and description of clinical benefit in the confirmatory trials.

2. About REMORA study (NCCH1508 study)1
This study is an open-label, single-group, multicenter, investigator initiated clinical phase II study (8 centers across Japan including the National Cancer Center Hospital). Forty-two patients with unresectable advanced or metastatic thymic carcinoma were enrolled who had progressed after at least one prior platinum-based therapy. The primary endpoint is Objective Response Rate (ORR) by independent image review using RECIST1.1, and secondary endpoints include Progression Free Survival (PFS), Disease Control Rate (DCR), Overall Survival (OS) and safety. Lenvatinib was administered at a starting dose of 24 mg once daily in 4-week cycles, and the dose was appropriately reduced according to the patient’s condition until the disease progressed or unacceptable adverse events was observed.

For efficacy analysis2, ORR was 38.1% (90% Confidence Interval (CI): 25.6-52.0) and the best overall response was 38.1% for partial response, 57.1% for stable disease, and 4.8% for disease progression. PFS (median) was 9.3 months (95% CI: 7.7-13.9), DCR was 95.2% (95% CI: 83.8-99.4), and the median OS was not reached (95% CI: 16.1-NR (not reached)) at the data cutoff date (Feb 22, 2019). The major treatment-related adverse events3 (more than 30%) were hypertension (88.1%), proteinuria (71.4%), palmar-plantar erythrodysesthesia syndrome (69.0%), hypothyroidism (64.3%), diarrhea (57.1%), thrombocytopenia (54.8%), decreased appetite (42.9%), weight loss (40.5%), dysphonia (40.5%), increased aspartate aminotransferase (33.3%), malaise (33.3%), and stomatitis (33.3%).

1 Jun Sato, Miyako Satouchi, Shoichi Itoh, Yusuke Okuma, Seiji Niho, Hidenori Mizugaki, Haruyasu Murakami, Yasuhito Fujisaka, Toshiyuki Kozuki, Kenichi Nakamura, Yukari Nagasaka, Mamiko Kawasaki, Tomoaki Yamada, Ryunosuke Machida, Aya Kuchiba, Yuichiro Ohe, Noboru Yamamoto; Lenvatinib in patients with advanced or metastatic thymic carcinoma (REMORA): a multicenter, phase 2 trial. The Lancet Oncology, 2020, Vol.21, No. 6, p843-850

2 Based on the package insert.

3 The adverse event data has been updated from the data in the paper.

3. About the Eisai and Merck & Co., Inc., Kenilworth, N.J., U.S.A. Strategic Collaboration
In March 2018, Eisai and Merck & Co., Inc., Kenilworth, N.J., U.S.A., known as MSD outside the United States and Canada, through an affiliate, entered into a strategic collaboration for the worldwide co-development and co-commercialization of LENVIMA. Under the agreement, the companies will jointly develop, manufacture and commercialize LENVIMA, both as a monotherapy and in combination with Merck & Co., Inc., Kenilworth, N.J., U.S.A.’s anti-PD-1 therapy KEYTRUDA (generic name: pembrolizumab).

In addition to ongoing clinical studies evaluating the LENVIMA plus KEYTRUDA combination across several different tumor types, the companies have jointly initiated new clinical studies through the LEAP (LEnvatinib And Pembrolizumab) clinical program and are evaluating the combination in 14 different tumor types (endometrial carcinoma, hepatocellular carcinoma, melanoma, non-small cell lung cancer, renal cell carcinoma, squamous cell carcinoma of the head and neck, urothelial cancer, biliary tract cancer, colorectal cancer, gastric cancer, glioblastoma, ovarian cancer, pancreatic cancer and triple-negative breast cancer) across 20 clinical trials.

On March 23, 2021 Eisai Co., Ltd. (Headquarters: Tokyo, CEO: Haruo Naito, "Eisai") reported that it has obtained manufacturing and marketing approval for the anticancer agent "Remitoro for Intravenous Drip Infusion 300μg" (denileukin diftitox (genetic recombinant)) with the indications of relapsed or refractory Peripheral T-cell Lymphoma (PTCL) and relapsed or refractory Cutaneous T-cell Lymphoma (CTCL), in Japan (Press release, Eisai, MAR 23, 2021, View Source [SID1234577000]). This agent was evaluated by the Ministry of Health, Labour and Welfare (MHLW) as a drug with high medical need at the "Study Group for Unapproved Drugs/Off-Label Drugs for High Medical Need". Hence, Eisai has been working on the development of the agent thereof in Japan, and applied for manufacturing and marketing approval in March 2020, based primarily on data from a Phase II clinical study (Study 205).

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Study 205 is a multicenter, open-label, single-arm Phase II clinical study, conducted in Japan to evaluate the efficacy and safety of the agent in patients with relapsed or refractory PTCL or CTCL.

This study achieved the primary endpoint target and exceeded a prespecified tumor response threshold with statistical significance: the objective response rate (ORR) of PTCL and CTCL patients in total (n=36) was 36.1% (95% confidence interval (CI): 20.8-53.8). The ORRs of each subtype were 41.2% (95%CI: 18.4-67.1) for PTCL (n=17) and 31.6% (95% CI: 12.6-56.6) for CTCL (n=19).

The five most frequent treatment-emergent adverse events observed in this study were increased aspartate aminotransferase (AST) (89.2%), increased alanine aminotransferase (ALT) (86.5%), hypoalbuminaemia (70.3%), lymphopenia (70.3%), and pyrexia (51.4%).

Eisai will conduct a post-marketing special use results survey (all-case surveillance) in all patients who are administered the agent until a pre-determined number of patients has been reached in accordance with an approval condition imposed by the MHLW.

The agent is a fusion protein consisting of interleukin-2 (IL-2) and partial sequence of diphtheria toxin, and specifically binds to the IL-2 receptor on the surface of tumoral lymphocytes. The antitumor effect of denileukin diftitox depends on the intracellular delivery of diphtheria toxin fragment which inhibits protein synthesis and induces cell death. Eisai retains exclusive development and marketing rights for the agent in Japan and Asia.

Eisai aims to make continuous efforts to meet the diversified needs of and increase the benefits provided to patients with cancer, their families, and healthcare professionals, by delivering "Remitoro" as a new treatment option for relapsed or refractory PTCL and relapsed or refractory CTCL in Japan.

1. About Remitoro for Intravenous Drip Infusion 300μg (Denileukin Diftitox (Genetic Recombinant))

This agent is a fusion protein consisting of interleukin-2 (IL-2) and partial sequence of diphtheria toxin. The antitumor effect of denileukin diftitox depends specific binding to the IL-2 receptor on the surface of tumoral lymphocytes followed by intracellular delivery and release of diphtheria toxin fragment which inhibits protein synthesis and induces cell death.

Eisai retains exclusive development and marketing rights for the agent in Japan and Asia, and in other regions, Dr. Reddy’s Laboratories Ltd. has development and marketing rights.

2. About Phase II Clinical Study (Study 205)

Study 205 is a multicenter, open-label, single-arm phase II clinical study evaluating the efficacy and safety of denileukin diftitox (genetic recombinant) conducted in Japan for patients with relapsed or refractory Peripheral T-cell Lymphoma (PTCL) or Cutaneous T-cell Lymphoma (CTCL). The patients who participated in this study received a final histopathological definitive diagnosis by the Central Committee for Pathological Diagnosis, which is independent of the clinical study site. The histopathological subtypes of participants consisted of 17 patients with PTCL, 19 patients with CTCL, and 1 patient with other malignant lymphoma. The efficacy of the agent was evaluated in 36 patients with PTCL or CTCL, and the safety was evaluated in 37 patients. The agent was administered by intravenous drip infusion over 60 minutes at a dose of 9μg / kg / day for five consecutive days from day 1 to day 5 to complete a cycle, with one cycle every three weeks and a maximum of up to 8 cycles conducted. In this study, the primary endpoint was objective response rate, and the efficacy of the agent was evaluated on the basis that the lower limit of the confidence interval (CI) was above a predetermined threshold.

3. About Peripheral T-cell Lymphoma (PTCL)

PTCL is a type of T-cell non-Hodgkin’s lymphoma that is classified as an intermediate-grade lymphoma. PTCL is often detected in advanced stages, and has symptoms such as swelling and lumps in the lymph nodes, fever, heavy night sweats, and weight loss. Among PTCLs, Anaplastic Lymphoma Kinase (ALK)-positive anaplastic large cell lymphoma, which occurs in the 20s and 30s, has a favorable prognosis and is curable. However, other types of PTCL often occur around the age of 60, and may have a poor prognosis or be difficult to treat. Therefore, PTCL is still a disease with extremely high-unmet medical need. It is estimated that the number of patients with PTCL in Japan is less than 6,000.1

4. About Cutaneous T-cell Lymphoma (CTCL)

CTCL is a type of non-Hodgkin’s lymphoma of primary cutaneous disease with various other manifestations in additional sites like lymph nodes and peripheral blood. In CTCL, some of the T cells (a type of lymphocyte involved in the immune system) become cancerous, causing skin lesions and reducing the patient’s QOL (Quality of Life) due to pain and pruritus. CTCL is generally a low-grade lymphoma, with initial patch and plaque skin lesions, but it progresses slowly and advances to the tumor stage over several years to over a dozen years. CTCL is still a disease with extremely high unmet medical need because it has a high malignancy when it reaches the tumor stage and has a poor prognosis. It is estimated that the number of patients with CTCL in Japan is less than 4,000.1

5. About Study Group for Unapproved Drugs/Off-Label Drugs for High Medical Need

The Study Group was set up within the Ministry of Health, Labour and Welfare with the purpose of contributing to enhancing the development of drugs and indications that have not been approved in Japan (unapproved drugs/off-label drugs) by pharmaceutical companies. In addition to evaluating the medical needs of unapproved drugs/off-label drugs, their responsibilities include evaluating the applicability of the drug to an Application with Public Knowledge, and the adequacy of additional clinical studies that need to be conducted for filing applications for approval, and so on.

1. Vital Statistics/Patient Survey in 2017 (Statistics and Information Department, Minister’s Secretariat, Ministry of Health, Labour and Welfare, Japan.) (available in Japanese only)