「邂逅遭遇」医療者のおもいをリサーチに
2022.11.12(SAT)-11.13(SUN)

国際シンポジウム

国際シンポジウム1

COVID-19禍における免疫研究のフロンティア
座長:
  • 宮田 俊男(早稲田大学)
  • 濱口 杉大(福島県立医科大学)
演者:
  • Georg Holländer, FMedSci(Oxford大学)
  • 宮田 俊男(早稲田大学)
(共催 ノーベルファーマ)
詳細を見る
内容

コロナ禍において、英国における創薬力は目覚ましく、オックスフォード大学は産学官連携の中心にあり、ホランダー教授はその中心人物の1人で、世界の免疫研究の第一人者でもあります。
日本の創薬力の復活のヒントを探ります。

Georg Holländer, FMedSci
The immune system determines the relationship between our health and our natural environment as it detects, eliminates and prevents harm to our health by biological threats. An overview of the system will be presented and related to SARSC-CoV-2 infections.
宮田 俊男
Zinc and immunity
Zinc, an essential micronutrient in the human body, is key trace element in growth and development, immune function, taste, wound healing, protein synthesis, and maintenance of skin and hair. Impact of zinc on immune function and COVID-19 infections will be presented.

国際シンポジウム2

Multimorbidityをはかる:
新時代の測定法
座長:
  • 山本 洋介(京都大学)
演者:
  • John E. Ware Jr., PhD(Harvard大学、Tufts大学)
  • 福原 俊一(京都大学)
(共催 Reckitt)
詳細を見る
内容

現在わが国の医療費の半分以上を消費している高齢者においては、複数の慢性疾患を併存している状態 (multi-morbidity) は当たり前のことになっている。高齢者を対象とする臨床研究においても、このmulti-morbidityを無視して科学的な分析は実施できない。これらを科学的に測定し、適切に調整しなければならない。multi-morbidityを測定する数少ない指標の一つであるCharlson Indexは、死亡をアウトカムとして重みづけをvalidateされたものであり、レガシー的な指標になりつつある。John Wareと福原は、このmulti-morbidityを測る画期的な指標QDISに関して長年にわたり共同で作業を行ってきた。今回のシンポジウムでは、その開発検証の過程や結果とともに、様々な慢性疾患に活用して得た成果も報告する。

Measuring multimorbidity: New approach for the next generation

Over a quarter of US adults (27.2%) have multiple chronic conditions (MCC) that adversely impact health status, functioning, or health-related quality of life (QOL). MCC are most prevalent among women, those over age 65, and those living in rural areas. One-third have three or more MCC. Because MCC are predictive of mortality, disability, response to treatment, healthcare utilization, expenditures, and declines in QOL (particularly physical QOL), accounting for differences in MCC is essential for evaluating health outcomes and clinical effectiveness. MCC data can also enhance the staging of individual patient complexity, aid in treatment planning, and improve the application of quality-of-care guidelines. Therefore, if we improve our estimates of the impact of MCC we can improve comparative effectiveness research. With better measurement of the impact of MCC, we can improve patient care by identifying individuals more or less likely to benefit from specific treatments and services.

Among the first MCC measures to incorporate both the number of conditions and the differences in their impact, the Charlson Comorbidity Index (CCI) has been the most frequently and extensively studied. While the CCI is useful for some case-mix adjustment purposes, it has been criticized because it relies on mortality weighting, it omits some common medical conditions, and it assigns the same weight to everyone with a given condition.

In this symposium, we introduce a recently developed disease-specific QOL measure, the 7-item Disease-specific Impact Scale (QDIS-7). The QDIS-7 reliably measures the QOL impact attributed to a specific disease or its treatments. Unlike conventional disease-specific QOL scales, the QDIS-7 is standardized across different conditions, which enables estimation of MCC’s aggregate QOL impact. We also systematically compare predictive validity for older and newer methods for aggregating the impact of MCC. We give examples of practical approaches using QDIS to achieve greater measurement efficiency, compare disease-specific QOL burden across MCC’s aggregate their QOL impact and determine when computerized adaptive assessment is likely to identify clinically important differences in disease-specific QOL

プロフィール
John E. Ware, Jr., PhD
Dr. Ware, is Research Professor in the Department of Medicine, Tufts University School of Medicine, Visiting Professor in the College of Health Solutions, Arizona State University and has lectured on patient reported outcome measurement (PROM) at Harvard T.H. Chan School of Public Health for more than 25 years.
He is an internationally recognized PROM expert and an elected member of the National Academy of Medicine. He led development of outcome measures in the RAND Health Insurance Experiment and was Principal Investigator for the Medical Outcomes Study, where he developed the SF-36® Health Survey, and for the International Quality of Life Assessment Project translations of the SF-36 for use in multinational clinical trials and population health surveys.
In the 1990’s, Dr. Ware was among the first to apply “modern” psychometric methods and computerized adaptive testing (CAT) to generic and disease-specific measures to standardize outcomes and measure population health more efficiently. He founded QualityMetric to develop web-based PROs and served as its CEO and CSO for 10 years. Dr. Ware has published more than 450 peer-reviewed articles. His current work focuses on the integration of disease-specific and generic PROMs using CAT survey logic to make patient screening and outcomes monitoring more practical and useful in clinical research and practice.
Shunichi Fukuhara, MD, DMSc, MACP
Dr. Shunichi Fukuhara is a Professor Emeritus and a former Dean of School of Public Health in the Kyoto University Graduate School of Medicine (2013-16). In 2012, He was appointed to be a Vice-President of Fukushima Medical University. He is also an Adjunct Professor of Johns Hopkins University Blooming School of Public Health.
During 1980-83, he received clinical training at University of California at San Francisco, certified in American Board of Internal Medicine and is currently a MACP. After his clinical training and practice as a cardiologist, he received training in clinical epidemiology at Harvard University and received MSc.
Since 1990, he has participated in the International Quality of Life Assessment (IQOLA) project led by Professor John Ware on behalf of Japan.
In 2000, he founded Kyoto University’s Department of Healthcare Epidemiology. He played a central role in Japanese steering committee of a large-scale multinational outcomes study on end-stage renal disease (DOPPS). He and his group has also conducted various kinds of cohort in communities and registry studies on chronic conditions. Since then he and his department have published more than 500 original articles in international peer-review journals. During his 20 years’ chairpersonship, his department has graduated 111 students (85 received Master and Doctoral degree) and about 70% of them became faculty members at leading medical universities, including 13 Professors and more than 20 Associate Professors.
In 2015, He presided the 7th meeting of the World Health Summit (WHS) in Berlin. In 2016, he was elected as the first president of Society for Clinical Epidemiology.

国際シンポジウム3

リアルワールドデータにおける
臨床エンドポイントの
バリデーションをめぐる話題
座長:
  • 浅尾 啓子(IQVIA)
演者:
  • 田栗 正隆(東京医科大学)
  • Emily W Bratton, MSPH, PhD(IQVIA)リモート登壇
(共催 IQVIA)
詳細を見る
内容
リアルワールドデータにおける臨床エンドポイントのバリデーションをめぐる話題
臨床研究における評価項目(clinical endpoints)は研究目的、研究デザイン、疾患の性質、予測される投与薬剤の効果などの因子を考慮し設定される。しかし、リアルワールドデータ(real world data, RWD)を用いたデータベース研究に臨床試験で用いられる評価項目をそのまま導入することは困難なことが多く、研究間の統一性を損なう要因にも成り得る。RWDに基づき設計された評価項目の定義や、そのために使われたアルゴリズムはその妥当性を従来の標準的指標と比較し検証する必要がある。そのためにバリデーション研究が実施され、例えば専門医の判断とデータベースに基づくアウトカム定義が比較される。バリデーション研究における妥当性の指標としては感度・特異度や陽性的中率などを用いることが推奨されているが、最終的にどの定義を採用すれば良いかの定量的基準はコンセンサスがない状況である。そこでROC曲線を用いた新しい考え方と基準を提案し、Youden Index等の既存の基準との比較や使い分けについて実践的な議論を行う。さらに、いくつかの評価項目においては臨床試験の結果との相関性が既に検証されている。そのような評価項目を用いた研究の結果が行政的判断に影響した先例もあり、今回紹介される予定である。また、学術機関や企業など研究遂行に関わる異なる領域間の連携についても議論を行う。
Validation of Clinical Endpoints in Real World Data
Endpoints in clinical research are defined through careful consideration of factors such as research objectives, research design, disease characteristics, and predicted effects of the administered product. However, the adaptation of clinical trial endpoints to a database study design using real world data (RWD) is often challenging and can result in inconsistencies. Real-world endpoint definitions and algorithms, which are often designed based on RWD, must be validated against the traditional gold standard. For that purpose, validation studies are conducted; for example, database-based outcome definitions are compared with the expert judgments. It is recommended to use sensitivity, specificity, positive predictive value etc. as indicators of validity in validation studies, but there is no consensus on the quantitative criteria for which definition should be adopted in the end. Therefore, we will propose a new way of thinking and criteria using the ROC curve and conduct practical discussions on comparison with existing standards such as the Youden Index. Additionally, there are existing studies using real-world endpoints having strong correlations with clinical trial results that are influencing regulatory decisions. In this symposium, we will review a few relevant examples. In addition, collaborations between different sectors, such as academia and industry, will be described throughout.
プロフィール
Speaker 1
Masataka Taguri, PhD
Department of Health Data Science, Tokyo Medical University

東京大学大学院で保健学の博士号を取得後、渡米しカリフォルニア大学サンフランシスコ校(UCSF)で客員研究員として勤務。帰国後は横浜市立大学医学研究科で教職に復帰し、2020年4年データサイエンス学部教授に就任。情報・システム研究機構統計数理研究所の客員教員としても勤務し、2021年4月同施設の客員教授として就任。2022年4月より現職の東京医科大学医療データサイエンス分野教授に就任し、研究と教育両方において活発な活動を行っている。観察研究データから因果推論を行うための諸手法に関して特に関心を持ち、多数の臨床研究に統計学専門家として貢献しながら方法論に関する研究も積極的に行っている。これまでに日本計量生物学会奨励賞、統計関連学会連合大会優秀報告賞など多数の受賞歴あり。日本臨床疫学会 学術専門委員。

After receiving a Ph.D. in Health Science from the University of Tokyo, he was appointed as a visiting scholar at the University of California, San Francisco (UCSF) in the United States. After returning to Japan, he became a faculty member at the Yokohama City University Graduate School of Medicine and was appointed to a professor in the School of Data Science in 2020. He is also a visiting faculty member at the Institute of Statistical Mathematics of the Research Organization of Information and Systems and was appointed as a visiting professor at the Institute in April 2021. He has been active in both research and teaching since April 2022 as a professor in the field of health data science at Tokyo Medical University. He is particularly interested in methods for performing causal inference from observational research data, and actively conducts research on methodologies while contributing to many clinical studies as a statistician. He has received many awards, including the Honorable Mention of the Biometric Society of Japan and the Excellence Report Award of the Japanese Joint Statistical Meeting.

Speaker 2 リモート登壇
Emily W Bratton, MSPH, PhD
Director, Global Epidemiology
Real World Solutions, IQVIA

North Carolina at Chapel Hill大学より疫学の修士、感染症疫学の博士学位を取得。現在同大学の助教授でもある。10年以上に渡る製薬業界での業績は主にlate-phaseの疫学観察研究チームに所属しながら達成したものであり、現在も疫学グループのリーダーを務めている。現職のIQVIA社Real-World Solutions Global Epidemiologyの部署では、後方視的・前方視的観察研究両方の設計・分析や解釈を担当しながら、さらに無・低介入臨床試験の設計や遂行にも携わっている。製造販売後調査にも豊富な経験を持ち、グロバールチームの関連業務のサポートを行っている。現在重点を置いている領域は、特に周産期・希少疾患分野の患者レジストリを活用した薬剤の安全性や効能に関する研究である。

Dr. Bratton received her MSPH in Epidemiology and PhD in Infectious Disease Epidemiology from the University of North Carolina at Chapel Hill where she now holds an adjunct assistant professor position. Dr. Bratton has been in the pharmaceutical industry over a decade, contributing to late-phase observational epidemiology research teams. As part of Real-World Solutions Global Epidemiology at IQVIA, she is responsible for the development, analysis, and interpretation of observational studies (both retrospective and prospective) and low interventional clinical trials. At IQVIA, she has been involved in the design and execution of non-interventional and low interventional studies and pragmatic trials. She has extensive experience in post-marketing regulatory requirements including protocols, reports, and regulatory response documents. She also is a scientific leader who manages a team of epidemiologists. She provides cross-functional support to global post-marketing teams, and most of her current work focuses on treatment effectiveness and safety, with emphasis on patient registries (notably pregnancy and rare disease registries).

Moderator
Keiko Asao, MD, PhD
Associate Principal, HEOR
Real World Evidence Solutions, IQVIA Solutions Japan K.K.