PSTC Japan Safety Biomarker Conference Session 6. Closing Akihiro - - PowerPoint PPT Presentation

PSTC Japan Safety Biomarker Conference Session 6. Closing

Akihiro Ishiguro, PhD – Pharmaceutical and Medical Device Agency John Michael Sauer, PhD – Critical Path Institute

Agenda – Day 1

2

• miRNA represents an important type of biomarkers
• miRNA can serve as safety biomarkers
• Leakage biomarkers
• Mechanistic biomarkers
• miRNA biomarkers also can be used to evaluate disease activity and

prediction

• Analytical validation maybe much difficult than clinical validation

Session Proposed Session Description / Theme Session Chairs 2 Beyond protein biomarkers: small circulating RNA

• miRNA safety biomarkers – Jiri Aubrecht (Takeda)
• miRNA – Tsuyoshi Yokoi (Nagoya Univ.)
• small circulating RNA – Takahiro Ochiya (Tokyo Medical Univ.)

Takayoshi Nishiya (Daiichi Sankyo) Nicholas King (C-Path)

Agenda – Day 1

3

• There are many discovery activities, including genomics, that will lead to the discovery
• f novel biomarkers
• Computational approaches will enable the discovery and application of biomarkers in

the future

• Qualification of in vitro biomarkers can have significant impact on the progression of

drug development

Session Proposed Session Description / Theme Session Chairs 3 Cluster/network analysis of genomics for disease grouping

• New diagnostic examination (biomarkers) and treatment for mental disorders by using of

genomics, epigenetics and metabolomics technologies – Atsushi Takata (Yokohama City Univ.)

• Modeling of the transcriptional response to multidrug treatment for prediction of positive

and negative effects of combinatorial drug therapy – Erik Arner (RIKEN)

• Building the Human Cell Atlas towards Medical Innovations – Jay Shin (RIKEN)
• TGx-DDI – Qualification of a Preclinical Biomarker – Roland Frötschl (BfArM)

Piero Carninci (Riken) Jiri Aubrecht (Takeda)

Agenda – Day 2

4

• Novel safety biomarkers are need to accelerate drug development
• The novel safety biomarkers discussed demonstrated significant improvement over

the currently used biomarkers

• The implementation of biomarkers and diagnostic tools requires significant effort to

ensure the usability of the biomarker

Session Proposed Session Description / Theme Session Chairs 4 Safety testing and disease diagnosis

• GLDH clinical qualification – Jiri Aubrecht (Takeda)
• Validation of drug-induced liver injury biomarkers in Japan – Noriaki Arakawa (NIHS)
• Qualification of Clinical Skeletal Muscle Injury Biomarkers: An Innovative Approach – Tanja Zabka

(Genentech)

• Diagnostic test/tool standardization – Masato Maekawa (Hamamatsu Medical Univ.)

Yoshiro Saito (NIHS) Tanja Zabka (Genentech)

Agenda – Day 2

5

• Qualification is the process of obtaining regulatory approval for the regular use of a

biomarker in drug development

• The PMDA, EMA and FDA all have qualification programs but with different process

and expectations

• The two fundamental aspects of biomarker qualification are Analytical Validation and

Clinical Validation

• Proper analytical validation is required for successful clinical validation
• However, there is no specific regulatory expectations on biomarker assay validation

and more stakeholder education is required

Session Proposed Session Description / Theme Session Chairs 5 Biomarker qualification evidentiary considerations

• Current evidentiary considerations progress across PMDA, FDA, and EMA – Nicholas King (C-Path)
• Case study: clinical and analytical validation of safety biomarkers – John-Michael Sauer (C-Path)
• Survey results and perspectives on analytical validation in Japan – Yoshiro Saito (NIHS)
• Qualification at PMDA – Mineo Matsumoto (PMDA)

Yasuto Otsubo (PMDA) Masanori Hizue (Pfizer Japan)

Thank you Planning Committee

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Chugai Nozomi Fujisawa Naoto Toyota C-Path Nicholas King John-Michael Sauer Daiichi Sankyo Ito Kazumi Takayoshi Nishiya Mitsubishi Takuya Fujita Manami Miyake Toshinobu Shimuzi NIHS Yoshiro Saito Novartis Yumiko Tsujimura Otsuka Yuichi Shimomura Pfizer Masanori Hizue Chieko Muto PMDA Akihiro Ishiguro Yasuto Otsubo Taiki Sato RIKEN Piero Carninci Masaaki Furuno Linda Kostrencic Todd Taylor Takeda Jiri Aubrecht Naomi Kamiguchi Shingo Okubo Yusuke Sudo

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Thank You to Our Co-Sponsors

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Thank You to Our Host

www.c-path.org

Thank you