Summit on Access and Affordability in Cell and Gene Therapies

Join the ISSCR in Los Angeles, California in collaboration with the California Institute for Regenerative Medicine (CIRM) and UCLA Broad Stem Cell Research Center.

Program | Sponsors

Program Description: As cell and gene therapies move from experimental treatments to approved interventions, the healthcare community faces a critical challenge: ensuring these groundbreaking therapies reach patients who need them most.

This summit brings together senior leaders in research, clinical practice, market access, policy, and patient advocacy to address the urgent need for sustainable pricing models, scalable manufacturing approaches, regulatory strategies, and innovative financing mechanisms. Through collaborative dialogue, we aim to balance continued innovation with the fundamental principle that medical breakthroughs should benefit all patients, regardless of economic circumstances or geographic location.

Summary of Sessions

Strategies for Reducing Risks and Addressing Commercial Insurer Participation
Emerging Policy for New Cell and Gene Therapy Affordability Models
Manufacturing and Regulatory Innovations

Registration is by invitation. Invited attendees will receive an email communication outlining the registration process. Use this form to indicate your interest.

Champion Sponsors:

Program

9:00 AM – 10:00 AM Welcome and Keynote Session

Dana P. Goldman is University Professor of Public Policy, Pharmacy, and Economics at the University of Southern California and the Founding Director of the USC Schaeffer Institute for Public Policy & Government Service. The Institute houses the Schaeffer Center for Health Policy & Economics, a program now ranked 3rd globally in health economics. He is a special advisor to the Provost and previously served as Dean of the USC Price School of Public Policy. Dr. Goldman’s research spans economics, medicine, and public policy. He is the author of more than 400 articles with 25,000+ citations. He pioneered a “Netflix model” for patient treatment and has been awarded more than $120 million in research support.  He is an elected member of the National Academy of Medicine, the National Academy of Social Insurance, and the National Academy of Public Administration.  He is a co-founder of EntityRisk and Precision Health Economics and a scientific advisor to several companies.  He is a research associate at the National Bureau for Economic Research. He graduated from Cornell University summa cum laude and earned a Ph.D. in economics from Stanford University.

10:30 AM – 12:00 PM Panel 1: Strategies for Reducing Risks and Addressing Commercial Insurer Participation

Dave works as an advisor and consultant in the pharmacy, health care delivery and health insurance industries. He has over 50 years of experience in the health care field and has served as the CEO of several health care service and technology companies. In 2018 he co-founded Emerging Therapy Solutions (ETS) to provide the payer industry with services to manage patients in need of complex and costly treatments such as bone marrow transplants, solid organ transplants, cell therapies, and gene therapies. As CEO, Dave was responsible for all strategic planning and development, operations, and financial performance until his retirement at the end of 2023.
Prior to founding ETS, Dave served as CEO at Medication Management Systems, NovoLogix, RxHub, and United Resource Networks.
Earlier in his career he worked in executive positions in the development and management of HMOs and PBMs with American MedCenters, Partners National Health Plans and Clinical Pharmaceuticals. He started his career as a hospital pharmacist and holds a PhD in Pharmacy Administration from the University of Minnesota, and a Bachelor of Science in Pharmacy from Ohio State University.
Harlan Levine, M.D., is president of health innovation and policy for City of Hope. He also serves as the chairman of the board of AccessHope, a spinout company from City of Hope that is focused on serving the employer market and making leading-edge cancer care available to all regardless of geographical location.
Prior to City of Hope, Dr. Levine held executive roles at UnitedHealth Group, Towers Watson and Anthem Inc. He currently serves as a board member for BioScienceLA, Reimagine Health, Active Life Scientific and Vida Health, and in an advisory capacity for Quantum Health and Laguna Health. In addition, he is a member of the Accessibility and Affordability Working Group for California Institute for Regenerative Medicine and a senior fellow for Health Evolution.
He is certified by the American Board of Internal Medicine and licensed by the Medical Board of California. He earned his undergraduate degree from Harvard College and his medical degree from the University of California, San Francisco. He interned at Mount Zion Hospital and Medical Center in San Francisco and completed his residency in internal medicine at Cedars-Sinai Health System in Los Angeles.
Anup Malani is the Lee and Brena Freeman Professor at the University of Chicago Law School, currently on leave to serve as the Chief Economist for the Centers for Medicare and Medicaid Services in Washington, D.C. His research spans health economics and policy, law and economics, and development economics. He has taught courses in health law and policy, health economics, microeconomics, law and economics, food and drug law, securities law, bankruptcy, corporate, contract, and insurance law. Malani also holds a courtesy appointment at the Pritzker School of Medicine and is a Research Associate at the National Bureau of Economic Research. He is the founding Faculty Director of the International Innovation Corps at the Harris School of Public Policy. An elected member of the American Academy of Arts and Sciences and a member of the COVID Crisis Group, Malani is a two-time recipient of the Emergent Ventures Prize. He clerked for Justice Sandra Day O’Connor on the U.S. Supreme Court and Judge Stephen Williams on the D.C. Circuit. Malani holds a J.D. and Ph.D. in economics from the University of Chicago.
Dr. LaShawn McIver is a proven public health leader with more than 20 years of experience advancing health equity, improving health outcomes, and promoting access to quality care. In July 2023, she joined AHIP as Senior Vice President and Chief Health Equity Officer, where she leads AHIP’s industrywide Health Equity Strategic Roadmap and provides strategic direction for member-driven equity initiatives. Previously, Dr. McIver served as Director of the Centers for Medicare & Medicaid Services (CMS) Office of Minority Health and a member of the Federal Senior Executive Service, guiding CMS’s health equity strategy, co-leading the National Rural Health and Quality Strategies, and shaping the HHS Social Determinants of Health framework. She also served on the White House Interagency Policy Councils on Maternal Health and Social Determinants of Health. Earlier, as Senior Vice President for Government Affairs and Advocacy at the American Diabetes Association, she led national efforts to expand diabetes research, prevention, and equitable access to care. Dr. McIver holds an MD in International Health and Medicine and an MPH from Johns Hopkins University.

Phil Cyr, MPH, BlueRidge Life Sciences, USA

1:00 PM – 2:30 PM Panel 2: Emerging Policy for New Cell and Gene Therapy Affordability Models

Darius Lakdawalla is Chief Scientific Officer at the USC Schaeffer Center for Health Policy & Economics and Quintiles Chair of Pharmaceutical Development and Regulatory Innovation at the USC Mann School. He holds faculty appointments at the USC School of Pharmacy and USC Price School of Public Policy, is a research associate at the National Bureau of Economic Research, and is a member of US National Academy of Medicine. Lakdawalla was recognized for transforming the study of value in healthcare, using economic insights to reorient health care value assessment around what matters to patients and their families. He developed Generalized Risk Adjusted Cost-Effectiveness (GRACE), demonstrating that health improvements are worth more to those in poorer health. His research focuses on the economics of health risks, medical innovation, health insurance markets, and healthcare industrial organization. He has testified before congressional committees on pharmaceutical innovation and drug pricing. Darius received his Ph.D. in Economics from the University of Chicago and B.A. in Mathematics and Philosophy from Amherst College. He has received multiple awards including the PhRMA Foundation Value Assessment Challenge Award and ISPOR Excellence in Research Methodology Award.
Purva Rawal, PhD, is an internationally recognized leader in value-based care and health policy, with over two decades of experience spanning government, academia, and the private sector. Currently, she is an advisor to the Duke Margolis Institute for Health Policy and Johns Hopkins University, and on international value-based payment implementation efforts. Most recently, she was the Chief Strategy Officer at the CMS Innovation Center. She led the implementation of a strategic vision grounded in accountability and whole, person-centered care, and oversaw its implementation across the Center.
Prior to her role at CMS, Dr. Rawal spent a decade in policy research and business strategy consulting, advising organizations on value-based payment and health system transformation. She also served as an adjunct assistant professor at Georgetown University, where she taught courses on the politics of health care and mentored students.
Earlier in her career, Dr. Rawal was a senior staff member on the Senate Budget Committee during the development of the Affordable Care Act and was an advisor to Senator Joseph Lieberman (I-CT). She began her policy career as a Christine Mirzayan Science and Technology Policy Fellow at the National Academies.
She is the author of The Affordable Care Act: Examining the Facts and has published in internationally recognized journals including, the Journal of the American Medical Association, the New England Journal of Medicine, and Health Affairs. Dr. Rawal received her B.A. and Ph.D. from Northwestern University.
Dr. S. Monica Soni (she/her) is the Chief Medical Officer at Covered California, leading the organization’s Health Equity and Quality Transformation division. She is working to improve the health of the nearly 2 million Covered California enrollees and the quality and affordability of care for all Californians. She is a board-certified internal medicine physician with more than a decade of experience working in both inpatient and outpatient settings and continues to see patients. She is an Associate Professor within the UCLA Department of Medicine and the Charles R. Drew University Department of Internal Medicine. Prior to joining Covered California, Dr. Soni served as Associate Chief Medical Officer within Evolent Health focused on the delivery of high-quality, cost-effective specialty care for the over 16 million supported Medicaid lives across the United States. Dr. Soni also served as the Director of Specialty Care for the Los Angeles County Department of Health Services, the second-largest municipal health system in the United States. She graduated from Harvard College and Harvard Medical School and completed residency at the University of California, San Francisco.
Mark C. Walters, MD, is the Jordan Family Director of the Blood and Marrow Transplantation Program at UCSF Benioff Children’s Hospital, Oakland and professor and Chief, Hematology division in the Department of Pediatrics at University of California, San Francisco School of Medicine. He is Program Director of the California Institute of Regenerative Medicine alpha clinic at UCSF. He has been active in cooperative clinical transplantation trials and has participated in NIH-supported investigations of hematopoietic cell transplantation for sickle cell disease and thalassemia and in industry-sponsored gene therapy clinical trials for hemoglobin disorders. Currently, research interests are focused on genomic editing and gene addition therapies as a strategy to extend curative therapy in all patients who inherit a clinically significant hemoglobinopathy such as sickle cell disease.
Manar Zaghlula is the Director of Public Impact at the Innovative Genomics Institute (IGI), where he leads a team of specialized program managers and researchers working to align the diverse IGI research portfolio with societal needs and values. Prior to this role, Manar served as the IGI Health Policy Manager, where he worked with key stakeholders and experts to develop innovative pathways for improving cell and gene therapy affordability and access, primarily through policy and regulation.
Before joining IGI, Manar was a CCST Science & Technology Policy Fellow in the California State Legislature. There, he served both as a committee consultant in the Assembly Environmental Safety and Toxic Materials Committee and as a legislative staffer to Assemblymember Dr. Bill Quirk. Manar analyzed numerous environmental policy proposals, guided a diverse bill portfolio through the legislative process, and covered policy areas including health and education policy. Manar holds a B.S. in Biology and Chemistry from the George Washington University and a Ph.D. in Translational Biology and Molecular Medicine from Baylor College of Medicine, where he investigated novel therapeutic entry points for a rare neurogenetic disorder.

3:00 PM – 4:30 PM Panel 3: Manufacturing and Regulatory Innovations

Sponsored by: Mytos Bio

Amander Clark, PhD, is Professor of Molecular, Cell and Developmental Biology at UCLA and the Founding Director of the UCLA Center for Reproductive Science, Health and Education. Professor Clark is an award-winning scientist and internationally recognized expert in stem cell biology, developmental biology, and reproductive science. Results from the Clark Lab provide the foundation for engineering reproductive cells and tissues from stem cells to develop solutions for infertility and reproductive dysfunction at all stages of life. Her current interests include in vitro gametogenesis, understanding the formation of the ovarian reserve, and preserving ovarian health as a strategy to promote the health span of women and girls. Dr. Clark has authored more than 100 scientific articles with over 20,000 citations. She is regularly invited as a subject matter expert for The New York Times, The Economist, The New Yorker, and Public Radio. From 2023–2024, she served as President of the International Society for Stem Cell Research (ISSCR) and currently serves on the National Academies of Sciences, Engineering, and Medicine Health Sciences Policy Board, advancing equitable, inclusive, and publicly informed science.
I am a seasoned senior business leader with over 20 years of experience in biologics and advanced therapies, who is passionate about the advanced therapy translational. I possess a blend of expertise in regulatory, quality, GMP, scientific, strategic, commercial development and management, with a focus in translating research from the bench through regulatory approval to clinical delivery.
My experience encompasses clinical development strategy, due diligence for investors and regulatory advice. I have provided strategic guidance on GMP and licensing requirements, developed quality management systems, and work closely with national and international regulators. My key skills include natural leadership, effective influencing, negotiation, communication, relationship building, strategic thinking, and problem-solving.
Currently holding an executive position and serving as a Board Director at the Cell and Gene Therapy Catapult, I lead a team dedicated to developing regulatory and translational strategies for advanced therapy products and the ecosystem to support their development and adoption in the UK and beyond.
I have successfully navigated regulatory complexities, developed translational strategies, and managed regulatory issues across various domains. I have played a pivotal role in strategic influencing nationally and internationally and I am currently leading a network of advanced therapy treatment centres in the UK to accelerate patient access to these transformative therapies.
I am fortunate to co-chair the ISSCR Standards Steering Committee and an active member of the and Manufacturing, Clinical Translation, and Regulatory Committee. I am also lucky to have many international connections, including sitting on a number of scientific advisory boards and have, board directorships in national and international organisations, details of which can be provided if required.
Matthew Hewitt, B.A. Ph.D., currently serves as Vice President, CTO Manufacturing Business Division at Charles River Laboratories (CRL) playing a critical role in driving strategic vision as well as leading multiple operational initiatives across CRL’s Advanced Therapeutic CDMO sites, Biologics Testing, and Microbial Solutions global network.
Before joining CRL, he was Head of R&D and Clinical Development for Lonza’s Personalized Medicine Business Unit leading Cocoon platform development, a closed, automated, scalable cell therapy manufacturing solution. In addition, he executed numerous collaborations across academia and industry leveraging the Cocoon.
Prior to Lonza, Matt led the Tumor Immunology and Microenvironment program at Bellicum Pharmaceuticals, focusing on improving cell therapy efficacy in solid tumors. He also led the
Immunology group at the University of Pennsylvania’s Gene Therapy Program, leading and contributing to numerous AAV gene therapy programs.
Matt received his B.A. in Molecular Biology at Goucher College while playing Men’s Lacrosse, PhD in Biophysics and Physiology from the University of Alabama at Birmingham, and completed his postdoctoral fellowship at Johns Hopkins University within the Asthma and Allergy Division.