The Science, Policy, and Potential of Cell and Gene Therapies

Key Resources (listed chronologically, beginning with the most recent)

“Payer Perspectives on Financing and Reimbursement of One-Time High-Cost Durable Treatments.” MIT NEWDIGS FoCUS Project. October 11, 2019. Available at

“Gene Therapy in 2019: Milestones and Challenges.” Patient-Centered Outcomes Research Institute. July 2019. Available at

“Rebalancing High Prescription Drug Prices with Innovation Incentives.” Conti, R. and David, S. Health Affairs Blog. July 1, 2019. Available at

“MIT Group Brings Together Stakeholders to Brainstorm How to Pay for Curative Therapies Over Time.” Caffrey, M. American Journal of Managed Care. June 18, 2019. Available at

“Projections from the Existing Pipeline of Cell and Gene Therapies: Launches and Patient Numbers.” MIT NEWDIGS FoCUS Project. October 29, 2018. Available at

“Paying for Therapies That Cure: Innovative Solutions Needed.” Dubois, R. STAT. July 25, 2018. Available at

“FDA Prepares for the Next Generation of Regenerative Medicines.” Woollett, G. Avalere. December 14, 2017. Available at

Additional Resources (listed chronologically, beginning with the most recent)

“Making Life Saving Treatments More Affordable.” Jena, A., Kee, A., Baumgardner, J. et al. Harvard Business Review. October 25, 2019. Available at

“FDA’s Framework for Regulating Regenerative Medicine Will Improve Oversight.” Richardson, L. Pew Charitable Trusts. October 17, 2019. Available at

“Gene Therapies’ Accessibility Problem.” Owens, C. Axios. October 2, 2019. Available at

“Our Take: How Insurers are Scrambling to Cover Multimillion-Dollar Gene Therapies.” The Daily Briefing. Advisory Board. September 9, 2019. Available at

“Breakthrough Therapies, Breakthrough Economics in the Era of Cure.” Farnia, S., Mostovoy, L., Flamm, C. et. al. Cell and Gene Therapy Insights. September 4, 2019. Available at

“Putting the Costs and Benefits of New Gene Therapies into Perspective.” Cohen, J., Chambers, J., Silver, M. et al. Health Affairs Blog. September 4, 2019. Available at

“Beyond the Hype: Gene Therapies Require Advanced Capabilities to Succeed After Approval.” PwC Health Research Institute. September 2019. Available at

“State Medicaid Programs Already Have Considerable Flexibility to Adopt Innovative Payment Models for New High-Cost Prescription Drugs.” Edwin, P. Georgetown University Health Policy Institute Center for Children and Families. July 9, 2019. Available at

“Transforming Next-Gen Therapy Supply Chains into Patient-Connected Value Chains.” Srivastava, S., Pernenkil, L., and Mooraj, H. Cell & Gene. June 20, 2019. Available at

“How Will We Pay for the Coming Generation of Potentially Curative Gene Therapies?” Cassidy, B. STAT. June 12, 2019. Available at

“The Death of Jesse Gelsinger, 20 Years Later.” Rinde, M. Science History Institute. June 4, 2019. Available at

“Estimating the Clinical Pipeline of Cell and Gene Therapies and Their Potential Economic Impact on the US Healthcare System.” Quinn, C., Young, C., Thomas, J., et. al. Value in Health.  June 2019. Available at

“Extending VBP Models into Medicaid Drug Purchasing: Challenges and Opportunities.” Dworkowitz, A., Fiori, A. and Bachrach, D. Health Affairs Blog. May 22, 2019. Available at

“Commentary: We Need to Make Sure New Drug Cures Don’t Widen Income Gap for Poor.” Gottlieb, S. CNBC. May 20, 2019. Available at

“CAR-T Payment Challenges Are Only Beginning.” Owens, C. Axios. April 29, 2019. Available at

“Hospitals Are Saving Lives with CAR-T. Getting Paid is Another Story.” Swetlitz, I. STAT. March 12, 2019. Available at

“The Future of Pharmaceuticals in Medicaid.” Dworkowitz, A., Fiori, A., and Robinson, S. Manatt, Phelps & Phillips, LLP. January 23, 2019. Available at

“A New Report Shows Nearly 300 Cell and Gene Therapies in Development.” Powaleny, A. The Catalyst. Pharmaceutical Research and Manufacturers of America. December 6, 2018. Available at

“Affording Miracles: As Biotech Victories in Gene Therapy Excite, Costs Spur Question for New Ways to Pay.” Knox, R. 90.9 WBUR. June 7, 2018. Available at

“Mass Health Leads the Way Towards Addressing Cell and Gene Therapy Reimbursement.” Barker, T. Medicaid and the Law. Foley Hoag LLP. February 3, 2018. Available at

“Advancing Gene Therapies and Curative Health Care Through Value-Based Payment Reform.” Daniel, G., Leschly, N., Marrazzo, J., et al. Health Affairs Blog. October 30, 2017. Available at

“Does the Hype Exceed Reality for Precision Medicine?” Knowledge@Wharton. University of Pennsylvania. April 27, 2017. Available at