Regenerative medicine products, which are intended to repair or replace damaged cells or tissues in the body, include a range of therapeutic approaches such as cell- and gene-based therapies, engineered tissues, and non-biologic constructs. The current approach to characterizing the quality of a regenerative medicine product and the manufacturing process often involves measuring as many endpoints as possible, but this approach has proved to be inadequate and unsustainable.

The Forum on Regenerative Medicine of the National Academies of Sciences, Engineering, and Medicine convened experts across disciplines for a 2-day virtual public workshop to explore systems thinking approaches and how they may be applied to support the identification of relevant quality attributes that can help in the optimization of manufacturing and streamline regulatory processes for regenerative medicine. A broad array of stakeholders, including data scientists, physical scientists, industry researchers, regulatory officials, clinicians, and patient representatives, discussed new advances in data acquisition, data analysis and theoretical frameworks, and how systems approaches can be applied to the development of regenerative medicine products that can address the unmet needs of patients. This publication summarizes the presentation and discussion of the workshop.

Contents

• The National Academies of SCIENCES • ENGINEERING • MEDICINE
• PLANNING COMMITTEE ON APPLYING SYSTEMS THINKING TO REGENERATIVE MEDICINE
• FORUM ON REGENERATIVE MEDICINE
• Reviewers
• Acknowledgments
• Acronyms and Abbreviations
• 1. Introduction
  • ORGANIZATION OF THE PROCEEDINGS
• 2. Introduction to Systems Thinking Concepts
  • AN INTRODUCTION TO SYSTEMS THINKING
  • APPLYING SYSTEMS THINKING TO THE DEVELOPMENT OF REGENERATIVE MEDICINES
  • SYSTEMS DYNAMICS OF CELL-STATE TRANSITIONS: RELEVANCE FOR REGENERATIVE MEDICINE
  • DISCUSSION
• 3. Exploring the Challenges of Critical Quality Attributes: The Role of Systems Thinking
  • SYSTEMS THINKING AND THE REGULATION OF REGENERATIVE MEDICINE PRODUCTS
  • USING SYSTEMS THINKING APPROACHES FOR CELL THERAPY PRODUCT DEVELOPMENT
  • DISCUSSION
• 4. Challenges Associated with Data Collection, Aggregation, and Sharing
  • TOWARD OPEN SCIENCE IN OMICS ANALYSIS AND DISEASE MODELING
  • USING BIG DATA FOR CLINICAL STRATIFICATION OF PATIENTS
  • DISCUSSION
• 5. Challenges and Opportunities Associated with Systems-Level Analysis and Modeling
  • DEVELOPING ALGORITHMS FOR SINGLE-CELL GENOMICS
  • MODELING DYNAMIC DATA TO IDENTIFY A LATENT SPACE
  • ADOPTING METABOLIC MODELING TOOLS IN BIOPHARMACEUTICAL DRUG DEVELOPMENT
  • DISCUSSION
• 6. Addressing Regenerative Medicine Manufacturing and Supply Chain Challenges with Systems-Level Approaches
  • ARTIFICIAL INTELLIGENCE IN CELL AND GENE THERAPIES
  • MODELING THE MANUFACTURING PROCESS IN REGENERATIVE MEDICINE
  • NOVEL SUPPLY CHAIN AND COST MODELING FOR CELL THERAPIES
  • DISCUSSION
• 7. Exploring Issues of Workforce Development Related to Systems Thinking
  • EDUCATION AND WORKFORCE DEVELOPMENT TO ADVANCE SYSTEMS THINKING
  • REFLECTIONS ON THE WORKSHOP
  • FINAL WORDS ON MODELS AND DATA
• References
• Appendix A. Workshop Agenda
• Appendix B. Speaker Biographical Sketches
• Appendix C. Statement of Task

Suggested citation:

National Academies of Sciences, Engineering, and Medicine. 2021. Applying systems thinking to regenerative medicine: Proceedings of a workshop. Washington, DC: The National Academies Press. https://doi.org/10.17226/26025.

Digital Object Identifier: https://doi.org/10.17226/26025

Additional copies of this publication are available from the National Academies Press, 500 Fifth Street, NW, Keck 360, Washington, DC 20001; (800) 624-6242 or (202) 334-3313; http://www.nap.edu.

Printed in the United States of America