What is Gene Therapy?

US Food and Drug Administration, What Is Gene Therapy? How Does It Work?, accessed 5/31/2019

American Society of Gene + Cell Therapy, Gene Therapy Basics, accessed 5/15/2020

American Society of Gene + Cell Therapy, Different Approaches, accessed 5/15/2020

Global Genes, A Guide to Gene Therapy, Rare Toolkits, accessed 5/31/2019

National Hemophilia Foundation, Resources on Gene Therapy, accessed 4/16/2020

National Organization for Rare Disorders (NORD), Gene Therapy: Your Questions Answered, accessed 5/31/2019

University of Utah, Gene Therapy, Learn.Genetics: Genetic Science Learning Center, accessed 5/31/2019

Value of Gene Therapy to Patients

FoCUS Research Brief: The resource navigation challenges for patients and caregivers (January 2021)
The analysis identified 27 themes from 423 published articles; themes spanned patient-specific and caregiver-specific challenges, finance-specific challenges and health, economic, and social challenges, including age and race.

American Society for Gene and Cell Therapy, Addressing the Value of Gene Therapy: Enhancing Patient Access to Transformative Treatments, accessed 5/31/2019
AGCT paper on the value of gene therapy and its policy recommendations to ensure patient access; contains case studies illustrating the various types of gene therapy value and impact on patients.

University of Utah, Gene Therapy Successes, Learn.Genetics: Genetic Science Learning Center, accessed 5/31/2019
Overview of gene therapy by the University of Utah; includes disease-specific examples and explanations of gene therapy impact

Pipeline Estimates: Durable, Curative Therapies

FoCUS Research Brief: Updated projection of US durable cell and gene therapies product-indication approvals based on December 2019 development pipeline. (July 2020)
Summary of an analysis, at the indication level, of durable cell and gene therapy new product launches in the US through 2030. Note: Research continues to advance and will be summarized and published periodically.

FoCUS White Paper: Tracking the CAR-T revolution:  Analysis of clinical trials of CAR-T and TCR therapies for the treatment of cancer (June 2020)
Summary of an analysis of all available FDA CAR-T/TCR clinical trials for the treatment of cancer with respect to different indications, targeted antigens, products, and originator locations. Note: Research continues to advance and will be summarized and published periodically.

FoCUS Research Brief: Pediatric inpatient costs of select orphan conditions with potential gene therapy launches (March 2019)
Summary of an analysis of the current cost burden associated with managing pediatric orphan conditions; cell and gene therapies may result in significantly decreased disease-related hospitalizations for children with these rare hereditary genetic conditions. Note: Research has continued to advance since this publication.

FoCUS Research Brief: Clinical trials and investment trends for novel CAR-T and TCR therapies (December 2018)
Summary of clinical trials and investment trends for CAR-T/TCR products. Note: Research has continued to advance since this publication.

Precision Financing Solutions


FoCUS White Paper: Warranty Model: A potential precision financing solution for durable cell and gene therapies (October 2020) FoCUS investigated a new warranty solution that provides risk-sharing flexibility between payers and developers by addressing the performance risk challenge, reducing data aggregation complexity, and mitigates MBP exposure

FoCUS White Paper: Precision Financing Solutions for Durable / Potentially Curative Therapies (January 2019)
A summary of the key solutions detailed in the FoCUS working groups with detail on legal, regulatory and operational enablers for each.

FoCUS White Paper: Designing Financial Solutions to Ensure Affordable Access to Cures (August 2018)
An overview of the FoCUS work. Includes a cures characterization framework, financing challenges posed by durable, curative therapies, a synthesis of key product characteristics influencing precision financing solutions, considerations for different stakeholder segments and an overview of key precision financing solutions.

FoCUS Research Brief: Precision Financing Challenges for Solid Tumor Adoptive T-cell Therapies (December 2019) – Provides an overview of the financing and operational challenges facing solid tumor adoptive T-cell therapies, which could raise provider capacity expansion financing issues as well as classical durable therapy financing challenges depending on the scale of launch.

Mytelka, DS, Cassidy, WM, Kohn, DB, and Trusheim MR,  Managing Uncertainty in Drug Value: Outcomes-Based Contracting Supports Value-Based Pricing, Health Affairs Blog, January 30, 2020, accessed 4/23/2020
Argues that outcomes-based contracting can be important toward achieving the goals of value-based pricing by supporting optimal price-value alignment.

FoCUS Research Brief: Impact of Patient Mobility on Annuity/Performance-based Contracting (June 2018)
Explains the challenges of patient mobility in the context of multi-year payment models and describes potential solutions.

FoCUS Research Brief: Stop-Loss Insurance or Reinsurance for Multiyear Contracts (July 2018)  
Explores how stop-loss insurance or reinsurance might function in the context of a multi-year contract with responsibility spanning more than one payer and suggests potential approaches.

FoCUS White Paper: The Role of Stop-Loss Insurance and Reinsurance in Managing Performance-based Agreements (September 2019) 
Examines reinsurance and stop-loss contracts and how they will be impacted by durable and curative therapies; suggests ways in which these secondary insurance products could evolve in order to be able to accommodate the needs created by these therapies.

Ali, F. et al., Curative Regenerative Medicines: Preparing Health Care Systems for the Coming Wave, In Vivo (November 2016), accessed 6/25/2019 
Provides an overview of the curative regenerative medicine landscape and highlights proposed alternative financial approaches.

Slocomb, T. et al., New Payment And Financing Models For Curative Regenerative Medicines, In Vivo (July 24, 2017), accessed 6/25/2019
Highlights and describes alternative potential financing models.

Duke Margolis Center for Health Policy, Breakthroughs and Barriers: Advancing Value-Based Payment for Transformative Therapies, May 2019, accessed 6/25/2019
Description of alternative payment approaches for outpatient- and inpatient-administered transformative therapies with recommended next steps.

Mahendraratnam, N. et al, Value-Based Arrangements May Be More Prevalent Than Assumed, The American Journal of Managed Care, February 2019, accessed 6/25/2019
Results of a survey of manufacturer experiences with value-based arrangements with an emphasis on outcomes-based arrangements; includes detail on contract design and challenges in implementation.


FoCUS White Paper: Tools for Implementation of Precision Financing Solutions within Medicaid Plans (November 2019)
A toolkit to assist State Medicaid organizations in designing, negotiating and establishing milestone-based contracts, building on experiences of State Medicaid leaders. Includes links to examples of approved SPA applications, a model, value-based supplemental rebate agreement and example worksheets to support negotiation of a Milestone-Based Contract. These are provided with the caveat that users should not construe these examples as advice for State Medicaid decision-makers. Teams should seek independent advice regarding their own states's SPA applications.

Risk Pools/Actuarial Risk Management

FoCUS Research Brief: Impact of Actuarial Risk on Health Plans (April 2018)
Provides a high-level perspective and examples of how treatment incidence and plan size affect the level of actuarial risk for a payer of a hypothetical new therapy; Highlights the importance analyzing retained risk to understand what financial tools may be required to avoid extreme income statement outcomes.

The Orphan Reinsurer and Benefit Manager (ORBM)

FoCUS Research Brief: An Orphan Reinsurer Benefit Manager (ORBM) (May 2018)
An overview of the ORBM concept, service offerings, and financial feasibility and sustainability considerations.

Trusheim, M. et al., Improving Management of Gene and Cell Therapies, Pharmaceutical Executive (Sep. 10, 2018), accessed 6/18/2019
A more detailed overview of the ORBM concept, service offerings and financial feasibility, also including an illustration of the ORBM’s interactions with other healthcare stakeholders.

Policy Barriers and Solutions

Overview Documents

FoCUS, Federal Policy Suggestions for Durable Therapies, (2019)
Two-pager summarizing key FoCUS suggestions to enable precision financing solutionsand patient access to durable therapies.

Duke-Margolis Center for Health Policy, Overcoming the Legal and Regulatory Hurdles to Value-Based Payment Arrangements for Medical Products, December 2017, accessed 6/25/2019  
Provides an overview of the policy barriers to value-based arrangements and potential solutions, focusing on Medicaid Best Price, FDA Regulation of Manufacturer Communications and Anti-kickback Statute issues.

Cook, F., et al, Ensuring patient access to regenerative and advanced therapies in managed care: How do we get there?, In Vivo (July/August 2018), accessed 6/25/2019 
Provides an overview of policy barriers to alternative financing approaches and proposes solutions.

Specific Federal Policy Issues

FoCUS Research Brief: Incorporation of value-based payment (VBP) agreements into the calculation of Medicaid drug rebates (January, 2019)
Describes the challenge of current Medicaid Best Price calculations to the use of value-based payment agreements and proposes concrete alternative calculations approaches.

Duke Margolis Center for Health Policy, Clarifying Medicaid Best Price Regulations In the Context of Value-Based Payment Arrangements, September 2019, accessed 4/14/2020
Provides an overview of challenges with current Medicaid Best Price regulations for value-based payment models and suggests regulatory clarifications for applying Medicaid Best Price to value-based payment arrangements, including two clarifications to the bundled sales provision and the free goods exception.

FoCUS Research Brief: Impact of FDA Guidelines on Communications Between Developers and Payers on Metrics in Performance-Based Agreements (January, 2020)
Provides an overview of current FDA guidelines on communications between developers and payers on metrics, and highlights challenges for the establishment of performance-based agreements under various scenarios.

State Policy Issues

FoCUS Research Brief: State Insurance Regulations Regarding Benefit Design (Deductible and Co-Pay Waivers (August 2019) 
Describes and proposes solutions to the challenges of state insurance regulations for payers seeking to rapidly modify benefit designs to redue patient copays on durable therapies.

Operational Considerations

FoCUS Research Brief: Roles of Centers of Excellence (COE) Networks (September 2018)
Outlines the rationale for COE networks and the potential advantages and limitations for their use in the context of cellular immunotherapies for cancer. Includes a description of unique aspects of COE selection criteria and other issues relevant to immune effector cell therapies for cancer.

FoCUS Research Brief: Model contracts for innovative oncology therapies (November 2018)
A multi-stakeholder working group defined appropriate considerations when creating a value-based contract for new cancer therapies.

FoCUS Research Brief: Themes in patient and caregiver financial challenges (March 2019)
An overview of a systematic literature review to investigate current understanding of the patient and caregiver experience, including financial and other related issues.

Stakeholder Perspectives

Patient Organization Overviews

Global Genes, A Guide to Gene Therapy, Rare Toolkits, accessed 5/31/2019

National Organization for Rare Disorders (NORD), Gene Therapy: Your Questions Answered, accessed 5/31/2019

Payer Surveys

FoCUS White Paper: Payer Perspectives on Financing and Reimbursement of One-time High-cost Durable Treatments (October 2019)
Synthesizes the results of a survey of 77 payers, representing 153 commercial fully-insured, Medicaid, Medicare Advantage, and self-insured employer plans. Builds on the June 2019 FoCUS Research Brief, providing additional detail and insights on payer interest in considering new financing approaches, the priority and timing of action, and considerations for adoption.

FoCUS Research Brief: Payer Perspectives Survey (June 2019)
Summary of survey of payer awareness of gene therapies, financing needs and expected approaches.

Barlow, J.F. et al., Are Payers Ready, Willing, and Able to Provide Access to New Durable Gene Therapies? (June 2019)
More detailed synthesis of results of survey of payer awareness of gene therapies, financing needs and expected approaches, including implications by payer type.

Ciarametaro, M. et al, Are Payers Ready to Address the Financial Challenges Associated with Gene Therapy?, Health Affairs Blog, June 28, 2018, accessed 6/25/2019
Survey of payer awareness of gene therapies, financing needs and initial expected approaches.

FoCUS Research Brief: Tracking Medicaid Coverage of Durable Cell and Gene Therapies (September 2019) Summary of survey of State Medicaid coverage policies for initial cell and gene therapies on market.