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A disease is defined as rare if the prevalence is fewer than 200,000 in the United States. It is estimated that there are more than 7,000 rare diseases, which collectively affect 30 million Americans or 10% of the US population. This diverse and complex disease area poses challenges for patients, caregivers, regulators, drug developers, and other stakeholders. This book is proposedto give an overview of the common issues facing rare disease drug developers, summarize challenges specific to clinical development in small populations, discuss drug development strategies in the evolving regulatory environment, explain generation and utilization of different data and evidenceinside and beyond clinical trials, and use recent examples to demonstrate these challenges and the development strategies that respond to the challenges.Key Features:• Rare disease.• Drug development.• Innovative clinical trial design.• Regulatory approval.• Real-world evidence.

This two volume set of LNAI 11061 and LNAI 11062 constitutes the refereed proceedings of the 11th International Conference on Knowledge Science, Engineering and Management, KSEM 2018, held in Changchun, China, in August 2018. The 62 revised full papers and 26 short papers presented were carefully reviewed and selected from 262 submissions. The papers of the first volume are organized in the following topical sections: text mining and document analysis; image and video data analysis; data processing and data mining; recommendation algorithms and systems; probabilistic models and applications; knowledge engineering applications; and knowledge graph and knowledge management. The papers of the second volume are organized in the following topical sections: constraints and satisfiability; formal reasoning and ontologies; deep learning; network knowledge representation and learning; and social knowledge analysis and management.

This two volume set of LNAI 11061 and LNAI 11062 constitutes the refereed proceedings of the 11th International Conference on Knowledge Science, Engineering and Management, KSEM 2018, held in Changchun, China, in August 2018. The 62 revised full papers and 26 short papers presented were carefully reviewed and selected from 262 submissions. The papers of the first volume are organized in the following topical sections: text mining and document analysis; image and video data analysis; data processing and data mining; recommendation algorithms and systems; probabilistic models and applications; knowledge engineering applications; and knowledge graph and knowledge management. The papers of the second volume are organized in the following topical sections: constraints and satisfiability; formal reasoning and ontologies; deep learning; network knowledge representation and learning; and social knowledge analysis and management.