AMED: Mission and Perspectives

Transcription

1 AMED: Mission and Perspectives Makoto Suematsu, MD, PhD President Japan Agency for Medical Research and Development (AMED) MHLW/PMDA International Memorial Symposium

2 Our goal is to fast-track medical R&D that directly benefits people not only by extending lifespans but also by improving quality of life: supporting life as biological existance, diary living, and lifespans Coverage: basic researches, TR and clinical researches, including fostering young researchers and returning outcomes of medical R/D for people A success of AMED (Japan Agency for Medical Research and Development) highly depends on alliance and cooperativity of three ministries (METI, MEXT, MHLW) To this end, we need to challenge for an art of probability.. METI MEXT MHLW AMED (1.4 billion US$/FY2015) Not only budgets but also officers and scientists with new mindsets 3 must not rules in AMED organization 1

3 Council of Research and Management Advisory Board Project Section President Executive Director Support Section Management Section Auditor Auditor Organization of AMED Office of Audit Department of Planning and Management Department of General Affairs Department of Financial Affairs Department of Research Integrity and Legal Affairs Permanent Staff Fixed-term Staff Department of Intellectual Property Department of Research Promotion Department of Industrial-Academic Collaboration 102 Approx. 200 Department of International Affairs Department of Research Infrastructure Department of Clinical Research and Trials Department of Innovative Drug Discovery and Development 5

4 Longitude/cross-sectional collaboration between Department of Research Promotion Matrix structure for accelerating Medical R & D Drug Research Regenerative Medicine Research Cancer Research 7 Research Projects Neurological. Psychiatric and Brain Research Support for practical application such as industrial-academic collaboration Rare/Intractable Disease Research Infectious Disease Research Emerging Research Promotion of strategic international researches Support for accommodating R&D platforms such as BioBank, etc. Support for high-quality clinical studies/clinical trials Support through the Drug Discovery Support Network for realizing academia drug discovery 3

5 Undiagnosed Disease Program in NIH diagnose (and treat) those undiagnosed ( from 2008, Dr William Gahl, NIH) Undiagnosed Disease Patients Medical Consultation Home doctors Collection of data and examination Regional Core Hospitals Analyses by physicians with different specialties Genome analysis center Phenotyping Known ones? Return of the Research Findings to Patients Individually Strong team of physician scientists Local Specialists of Clinical Genetics The Japan s National Liaison Council for Clinical Sections of Medical Genetics etc. Genetic counselling Judge treatable or not? Diagnose known diseases Assessment by specialist of clinical genetics G and M data NGS (ISO, CLIA etc) metabolomics Data banking 4

6 Johnson et al Brain 2012: 135; Mutation of riboflabin receptor causes ALS-like severe phenotypes in a newborn baby. AMED will start Joining in IRDiRC in this autumn Organizing IRUD (Initiative for Rare and undiagnosed diseases) under collaboration with NIH-UDP Program 9

7 Dr. William Gahl, MD, PhD Founder of undiagnosed Patients Program, NIH Not sequencers but real physicians Phenotype is everything 10

8 In genome medicine, likely pathogenic variant now may turn out likely benign 5 years later; Necessity for real-time data sharing of clinical genetics through DB N Engl J Med Jun 4;372(23):

9 AMED has launched Initiative for Rare and Undiagnosed Diseases (IRUD) フォローアップ 診断困難な患者さん 受診 診断説明カウンセリング IRUD 診断連携 IRUD 診断委員会は地域の拠点病院 等に配置し 全国配備を目指す 地域での密接な連携 IRUD に参加する拠点病院 ( 総合病院 ) 紹介受診 エントリーシート ( 臨床 検査データ ) IRUD 解析コンソーシアム IRUD 解析センター 依頼された検体についてエクソーム解析等の遺伝学的検査を行う 遺伝学的検査以外の検査や遺伝子変異の機能解析も検討する 解析結果 検査依頼 IRUD 診断委員会 幅広い診療科の医師で構成 臨床遺伝専門医を中心として臨床カンファレンスを開催 地域の臨床医と積極的に連携し地域をあげて取り組む データ IRUD データネットワーク AMED データセンター 登録システムの構築 運営 データベースの構築 運営 国際ネットワークとの連携 12

10 IRUD constitutes a network consisting of multiple hospitals to save patients with RUD with equal opportunities all over Japan IRUD Core Hospitals IRUD Clinical Expert Committee 疾患エキスパートによる専門分科会 IRUD 診断委員会による依頼を受け 専門的な症例検討を追加する 必要に応じて IRUD に参加する拠点病院で直接診療 IRUD exerts initiative for all-japan network 症例検討依頼 IRUD Diagnostic Committee 幅広い診療科の医師で構成 臨床遺伝専門医を中心として臨床カンファレンスを開催 地域の臨床医と積極的に連携し 地域をあげて取り組む 検討結果フィードバック 13

11 AMED has participated in IRDiRC to share IRUD data for saving patients over the world Japan has joined in IRDiRC in July 30, 2015 data sharing for patients Machine-readable consent microattribution 14

12 Discovery Innovation Screening Consortium: DISC Purpose: Enrich All Japan screening library by integrating unique compounds of pharmaceutical companies for use in HTS by the Drug Discovery Support Network AIST(N 2 PC) Natural products Univ. of Tokyo Low MW compounds Compound storage, assay plate preparation, HTS implementation and cherry picking to be outsourced to industry All Japan Screening Library Tool compound discovery Lead discovery HTS by RIKEN Already established: AIST, NIBIOHN, Univ.Tokyo NIBIOHN mab-phage/aptamer AMED/iD3 Low MW compounds pharma-companies Highly patentable, unique structures XCo. Ltd Storage assay-plate preparation YCo. Ltd HTS Pharma DISC Pharma E Impact of DISC Whole: create the first non-competitive collaborative space in Japan and improve overall pharma R&D productivity Academics: increase the chance to find quality lead and tool compounds for their own newly discovered targets Industry:maximize potential value of their own compounds through increased chance to be screened out against new targets discovered by universities, etc. Public-Private Partnership to take advantage of unique compounds & HTS technology of pharmaindustry for more effective translational research id3 to select targets & cover HTS cost Pharma to provide access to premium compounds and HTS technology/equipment Pharma D Pharma C Pharma A Pharma B 15

13 Strengthening AMED/PMDA Partnership (August 2015) To accelerate the creation of innovative medical products Utilizing AMED/PMDA s knowledge and experience mutually at the operation of our tasks Connecting project adoption by AMED with Pharmaceutical Affairs Consultation on R&D Strategy by PMDA PMDA s cooperation at the project evaluation by AMED PMDA s support for AMED s activities to improve and refine the clinical research/trial infrastructure (e.g. dispatch of PMDA staffs to training courses) Information sharing (e.g. AMED; medical R&D trends, state of projects, PMDA; trend of the regulation on medical products, state of the Consultation and clinical trials) Copyright 2015 Japan Agency for Medical Research and Development. All Rights Reserved. 16

14 From subjects in HIROs Meeting (Ottawa, June 2, 2015) (Heads of International Research Organizations) 1.Global cooperation for fighting against infectious diseases (Ebora fever, Mycobacterium with multidrug resistance, etc. 2.Precision Medicine 3.Genome editing; bright and dark sides 4.Attrition data filing 5.Fighting against rare and undiagnozed diseases through global data sharing (GA4GH, IRDiRC) HIROs members will get together in AMED next year Copyright 2015 Japan Agency for Medical Research and Development. All Rights Reserved. 17

15 AMED Mission 1 Global Cooperation for Controlling Diseases: 1Infectious diseases 2Regenerative medicine 3Neuropsychiatric diseases 4Rare diseases (and undiagnosed diseases) and R/D for orphan drugs 2 Empowerment of Medical R&D in Japan 1 Opening resources of drug libraries 2 Accelerating nation-wide registry for optimizing cancer Tx 3 Establishing infrastructure of clinical genetics (CLIA lab, etc) 4 Fostering young physician scientists for medical R/D 5 Utilization of private-public partnership to stimulate medical R/D 3 Renew selection processes and improve flexibility of budget systems 4 Clinical database (under supervision by Cabinet Secretariat, Office of Healthcare Policy) 14