访问全球数据解决罕见疾病：健康经济学价值框架英文版（28页）.pdf

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1、White Paper Global Data Access for Solving Rare Disease A Health Economics Value Framework February 2020 World Economic Forum 91-93 route de la Capite CH-1223 Cologny/Geneva Switzerland Tel.: +41 (0)22 869 1212 Fax: +41 (0)22 786 2744 Email: contactweforum.org www.weforum.org 2020 World Economic For

2、um. All rights reserved. No part of this publication may be reproduced or transmitted in any form or by any means, including photocopying and recording, or by any information storage and retrieval system. 3Global Data Access for Solving Rare Disease: A Health Economics Value Framework Contents 4 5 6

3、 9 10 18 22 24 25 Foreword Executive summary Introduction How to examine the benefits of rare-disease data federations Impact of federated data systems on known benefits Gap analysis Conclusion Contributors Endnotes 4Global Data Access for Solving Rare Disease: A Health Economics Value Framework For

4、eword The need to reframe our approach and response to rare disease is urgent. Approximately 10% of the global population or 475 million people are affected by a rare condition,1 with an estimated 15.2 million individuals expected to have clinical genomic testing for a rare condition within the next

5、 five years.2 The number of countries with national initiatives to sequence patients with rare diseases, as well as healthy individuals, is growing. This genomic data, coupled with phenotypic and clinical data, represents a treasure trove of information critical for shortening the diagnostic odyssey

6、 faced by rare-disease patients and for powering research and innovation in diagnostics and therapeutics. We believe that federated data systems offer a promising approach, providing researchers and clinicians with access to global rare-disease datasets while allowing local institutions to protect s