CGT Overview

Cell and Gene Therapies (CGT) Hub

UPDATED!!   Leading employers acknowledge that a value proposition is necessary when designing specialty drug benefits. This means offering high-quality, competitive benefits while acknowledging the need for shared responsibility between employees and the company. The following areas will support employers' efforts to build or enhance their strategies for managing specialty drugs.

Cell & Gene Therapy (CGT) Resource Hub

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This page is designed to help MBGH employer members quickly locate credible resources on cell and gene therapy fundamentals, financing and coverage options, financial risk management, access and provider networks, and the fast-moving future pipeline. The emphasis is on practical employer readiness: understanding the market, asking better questions of vendors, and building a more durable benefits strategy.

 

Start with the fundamentals Jump to future trends

How to use this hub

Start with the foundational section if your team needs a common baseline. Move next to coverage and financing if you are reviewing benefit strategy or renewal assumptions. Use the access and network section when evaluating centers, navigation pathways, genetic counseling, or specialty pharmacy support.

Employer readiness actions

  • Review current medical and pharmacy benefit language for cell and gene therapy coverage, exclusions, and site-of-care rules.
  • Ask medical carriers, PBMs, specialty pharmacy partners, and stop-loss carriers for their current CGT strategy and 2026-2027 roadmap.
  • Map internal decision rights for clinical review, catastrophic claim response, member communications, and exception management.
  • Identify which centers of excellence, genetic counseling services, and navigation supports are available to members today.
  • Track pipeline updates at least quarterly and refresh prevalence/risk analyses for conditions with near-term launches.

Section

Understanding Cell & Gene Therapy

Foundational explainers, approved-product references, patient education tools, and employer-oriented overviews that help benefit leaders build a working knowledge of cell and gene therapy.

FDA

Approved Cellular and Gene Therapy Products

Official FDA list of approved cellular and gene therapy products, useful for tracking the commercial market and covered therapies.

FDAApproved productsMarket overview
Open resource

FDA

Cellular & Gene Therapy Products

FDA overview of how cellular and gene therapy products are regulated, including core definitions and oversight.

FDARegulationDefinitions
Open resource

ASGCT

Gene & Cell Therapy Patient Education Portal

Trusted explainers, videos, webinars, and beginner-friendly education for patients, caregivers, and non-specialists.

EducationPatient-friendlyBasics
Open resource

ASGCT

ASGCT Patient Education Program

Overview of ASGCT’s Gene Therapy 101 and disease-specific educational materials.

EducationGene Therapy 101Explainers
Open resource

NCATS

Gene-Targeted Therapies

NIH/NCATS explanation of gene-targeted therapies, platform development, and translational work in rare disease.

NIHRare diseaseScience overview
Open resource

NCATS Toolkit

Genetic Therapies Toolkit

Plain-language definitions of gene therapy, genome editing, RNA-based therapies, and cell-based gene therapy.

ToolkitDefinitionsReadiness
Open resource

NORD

Gene Therapy Resources

Curated rare disease education resources, videos, and on-demand webinars on gene therapy and genome editing.

Rare diseaseEducationAdvocacy
Open resource

NORD

Gene Therapy FAQs

Frequently asked questions on what gene therapy is, whether it is curative, safety questions, and eligibility considerations.

FAQPatient questionsBasics
Open resource

NORD Video Library

Gene Therapy: Your Questions Answered

Short video resource answering common questions patients and families ask about gene therapy.

VideoPatient educationFAQ
Open resource

Global Genes

A Guide to Gene Therapy

Downloadable toolkit that explains how gene therapy works, current challenges, and questions to ask when evaluating treatment.

ToolkitRare diseaseDownload
Open resource

Global Genes

Understanding Gene Therapy Webinar

Webinar introducing gene therapy and patient engagement in research and trials.

WebinarEducationResearch
Open resource

National Alliance of Healthcare Purchaser Coalitions

Understanding Cell and Gene Therapy and Its Impact on the Workforce

Employer-oriented slide deck covering precision medicine, CGT basics, workforce implications, and emerging financing considerations.

Employer viewWorkforceSlides
Open resource

Section

Coverage & Financing Strategies

Resources focused on benefit design, alternative payment models, multi-state purchasing approaches, outcomes-based arrangements, and policy frameworks that can inform employer financing strategies.

National Alliance of Healthcare Purchaser Coalitions

An Employer Roadmap to Addressing Cell and Gene Therapies

Practical employer roadmap covering population risk, coverage options, non-traditional payment approaches, care delivery, and communication strategies.

Employer roadmapCoveragePayment models
Open resource

EBRI

Cell and Gene Therapies in Employment-Based Health Insurance

Current employer-market analysis of prevalence, utilization, spending concentration, and financing options in employment-based coverage.

Employer dataFinancingIssue brief
Open resource

EBRI

EBRI Issue Brief PDF

Downloadable PDF version of EBRI’s 2026 issue brief for sharing or internal review.

PDFEmployer data2026
Open resource

ICER and NEWDIGS

Managing the Challenges of Paying for Gene Therapy

White paper on fair pricing, clinical uncertainty, short-term budget impact, and tools such as stop loss, subscription models, and federal carve-out concepts.

White paperPolicyFinancing models
Open resource

CMS

CMS Cell and Gene Therapy Access Model Overview

Overview factsheet explaining the CMS-led outcomes-based model and its structure for gene therapy access.

CMSOutcomes-basedFactsheet
Open resource

CMS

CMS Cell and Gene Therapy Access Model Infographic

High-level visual summary of the Access Model, goals, and operating concept.

CMSInfographicQuick reference
Open resource

CMS

CMS Access Model Frequently Asked Questions

Detailed FAQ on participating states, manufacturer agreements, access policies, provider requirements, and beneficiary support.

FAQCMSImplementation
Open resource

CMS

CMS Expands Access to Lifesaving Gene Therapies Through Innovative State Agreements

Press release describing state participation and outcomes-based rebates under the Medicaid CGT Access Model.

Press releaseMedicaidOutcomes-based
Open resource

CMS

Innovation Insight: CMS Model Delivers Access to Sickle Cell Gene Therapy

Brief update on the model’s state participation and implications for access and affordability.

CMSSickle cellInnovation
Open resource

ICER

Gene Therapies for Sickle Cell Disease: Effectiveness and Value

Evidence report on clinical effectiveness, durability questions, and value assessment for gene therapies in sickle cell disease.

ICERSickle cellValue assessment
Open resource

ICER

Gene Therapy for Hemophilia B and Update on Hemophilia A

Evidence report that includes cost-effectiveness analysis and policy recommendations for payer coverage and contracting.

HemophiliaICERCoverage policy
Open resource

AMCP

CMS Announcement on the Cell and Gene Therapy Access Model

Managed care pharmacy summary of the CMS model and why it matters for payers.

AMCPManaged carePolicy summary
Open resource

AMCP

Navigating Reimbursement and Access Challenges for Novel Cell and Gene Therapies

Educational webinar on reimbursement, coding, billing, and access challenges for novel CGT products.

WebinarReimbursementManaged care
Open resource

AMCP

AMCP Supports Medicaid VBPs for Patients Act

Policy resource on expanding value-based purchasing arrangements for high-cost novel therapies.

LegislationValue-based purchasingPolicy
Open resource

Mercer

Employers’ Top Four Questions About Cell and Gene Therapies

Employer-facing perspective on market outlook, coverage norms, predictive analytics, stop-loss, and carve-out evaluation.

Employer strategyMercerCoverage questions
Open resource

Section

Financial Impact and Cost Management

Employer-focused resources on budget exposure, claims volatility, stop-loss and carve-out considerations, specialty pharmacy management, and tools for understanding total financial impact beyond drug acquisition cost alone.

National Alliance of Healthcare Purchaser Coalitions

Rethinking How Employers Address High-Cost Claims

Employer report highlighting high-cost claims management lessons, including practical considerations for cell and gene therapy exposure.

High-cost claimsEmployer strategyRisk management
Open resource

Lantern

Why Employers Can’t Ignore Cell and Gene Therapies

Employer-oriented discussion of catastrophic risk, risk-management strategies, patient identification, and provider selection.

Employer strategyRisk managementCare navigation
Open resource

CVS Health

Gene Therapies and Genetically Modified Cellular Therapies

White paper on financial exposure, specialty pharmacy sourcing, provider networks, and practical cost-management levers.

White paperSpecialty pharmacyNetwork strategy
Open resource

SpecialtyRx Toolkit

SpecialtyRx Toolkit

Broader specialty pharmacy toolkit covering biopharmaceuticals, gene therapies, plan performance, and cost-control strategies.

ToolkitSpecialty pharmacyPlan management
Open resource

Innovative Rx Strategies

The Gene and Cell Therapy Landscape

Briefing for plan sponsors on pricing, ancillary costs, stop-loss limitations, and employer response options.

Plan sponsorBriefingCost trends
Open resource

Truveris

Gene Therapy: Key Payer Considerations

Checklist-style article on benefit assessment, contract terms, prior authorization, monitoring, and affordability considerations.

ChecklistPayerAffordability
Open resource

Avalere Health

Specialty Pharmacies Adapt to Support Cell and Gene Therapies

Useful overview of the expanded specialty pharmacy role in benefits investigation, logistics, tracking, and financial support.

Specialty pharmacyOperationsPatient journey
Open resource

Mercer

Employers’ Top Four Questions About Cell and Gene Therapies

Helpful reference for large employer concerns around financial predictability, lasers, reinsurance, and future market expansion.

MercerLarge employersStop-loss
Open resource

National Alliance of Healthcare Purchaser Coalitions

Employer Playbook on Biosimilars

Not CGT-specific, but valuable for broader specialty pharmacy governance, formulary strategy, site-of-care management, and employer questioning of PBM practices.

PlaybookSpecialty pharmacyFormulary
Open resource

Global Genes

Financial Literacy and Navigation in Rare Disease

Useful lens on hidden financial burden, coverage denials, navigation support, and questions employers may want vendors and centers to answer.

Rare diseaseFinancial navigationMember support
Open resource

Section

Access, Utilization and Provider Networks and Resources

Resources that support provider selection, treatment center quality, patient navigation, specialty pharmacy operations, genetic counseling access, and longitudinal outcomes tracking.

FACT

FACT Accredited Institutions Directory

Searchable directory of FACT-accredited institutions for cellular therapy programs.

DirectoryAccreditationCenters of excellence
Open resource

FACT

Foundation for the Accreditation of Cellular Therapy

FACT overview and standards hub for cellular therapy quality and accreditation.

AccreditationQuality standardsProvider evaluation
Open resource

NMDP

NMDP BioTherapies

Overview of sourcing, donor matching, logistics, outcomes data, and support infrastructure for next-generation cell therapies.

NMDPAllogeneic sourcingInfrastructure
Open resource

NMDP

NMDP Advanced Cell Therapy Lab Solutions

Resource on cryopreservation, processing, dose customization, and advanced product handling support.

Lab supportProcessingLogistics
Open resource

CIBMTR

CIBMTR Collaborations

Overview of major CIBMTR initiatives, outcomes databases, trials collaboration, and sickle cell disease efforts.

Outcomes dataResearchRegistry
Open resource

CIBMTR

CMS Innovation Center CGT Access Model for Sickle Cell Disease

Detailed resource on CIBMTR’s role in collecting clinical and patient-reported outcomes for the CMS model.

CIBMTRPatient-reported outcomesSickle cell
Open resource

CIBMTR Patient Resource

CGT Access Model for Sickle Cell Disease

Patient-facing summary of CIBMTR data collection and quality-of-life tracking under the CGT Access Model.

Patient resourcePROsSickle cell
Open resource

NMDP Patient Support Center

BMT Support & Resources

Patient and caregiver support center with guidance, resources, and practical assistance during treatment journeys.

Patient supportCaregiversNavigation
Open resource

NMDP

Patient and Caregiver Emotional Support (PACES)

Free emotional support resources that can inform employer thinking around whole-person support during complex therapy episodes.

Behavioral supportCaregiversWhole-person care
Open resource

NSGC

About Genetic Counselors

Explains the role of genetic counselors and why they matter before and after testing and treatment decisions.

Genetic counselingTelehealthPre-treatment support
Open resource

AABB

AABB Toolkit to Support Cell and Gene Therapy Pharmacy Accreditation

Helpful resource for understanding how pharmacy programs are building accredited handling and distribution capabilities for CGT products.

AABBPharmacyAccreditation
Open resource

Orsini

Cell & Gene Therapy Center of Excellence

Specialty pharmacy example showing supported therapies, enrollment pathways, and site-of-care coordination structures.

Specialty pharmacyCenter of excellenceCare coordination
Open resource

Section

Future Trends and Employer Readiness

Forward-looking market intelligence, regulatory developments, pipeline trackers, and employer planning resources to help benefits leaders anticipate the next wave of CGT activity.

ASGCT

Quarterly Industry Landscape Report

Central landing page for ASGCT quarterly landscape reports tracking approvals, pipeline shifts, and capital activity.

PipelineApprovalsQuarterly updates
Open resource

ASGCT

Gene, Cell, & RNA Therapy Landscape Report Q4 2025

Detailed year-end snapshot of approvals, pipeline composition, financing activity, and notable disease targets.

Q4 2025PipelineMarket snapshot
Open resource

ASGCT

Gene, Cell, & RNA Therapy Landscape Report Q3 2025

Quarterly update highlighting clinical trial activity, diversification into non-oncology indications, and current approvals.

Q3 2025Clinical trialsTrend report
Open resource

ASGCT

Gene, Cell, & RNA Therapy Landscape Report Q2 2025

Useful reference for pipeline growth and therapeutic area shifts during 2025.

Q2 2025Trend reportPipeline
Open resource

ARM

Alliance for Regenerative Medicine

Sector-wide organization offering policy, market, and commercialization updates across regenerative medicine.

Sector overviewPolicyCommercial trends
Open resource

Alliance for Regenerative Medicine

ARM Resources

Direct access to sector snapshots and topical resources including late-2025 updates.

Sector snapshotResourcesMarket updates
Open resource

FDA

FDA Increases Flexibility on Requirements for Cell and Gene Therapies to Advance Innovation

Press release outlining FDA’s more flexible approach to CMC requirements for CGT development.

FDACMCRegulatory trend
Open resource

FDA

Flexible Requirements for Cell and Gene Therapies to Advance Innovation

Operational FDA detail on investigational and commercial specification flexibility for CGT products.

FDAManufacturingRegulatory detail
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FDA

Regenerative Medicine Advanced Therapy Designation

Key FDA designation page that helps employers and benefit advisors understand accelerated development pathways.

RMATFDAExpedited pathways
Open resource

FDA

Innovative Designs for Clinical Trials of Cellular and Gene Therapy Products in Small Populations

Guidance document reflecting how rare-disease CGT evidence generation may continue to evolve.

Clinical trialsGuidanceRare disease
Open resource

Mercer

Employers’ Top Four Questions About Cell and Gene Therapies

Useful readiness piece for employer planning around market growth, coverage expectations, and evolving plan strategy.

Employer readinessForecastMarket outlook
Open resource

Suggested uses for MBGH members

This hub can support employer education, vendor due diligence, internal committee briefings, and specialty pharmacy strategy discussions. It also works well as a starting point for webinar follow-up, committee packets, or member self-service exploration of high-cost therapy trends.

Best for committee review

  • Benefits and pharmacy committees
  • Finance and risk management stakeholders
  • Clinical and population health leaders
  • Consultants supporting plan strategy and vendor evaluation

Maintenance tips

  • Refresh FDA and ASGCT market trackers quarterly
  • Revisit CMS and AMCP policy pages as models evolve
  • Update stop-loss and carve-out references during renewal season
  • Add MBGH-specific guidance, checklists, or webinar recordings over time

Prepared for the Midwest Business Group on Health Specialty Pharmacy Toolkit. Last updated March 31, 2026. External resources remain the property of their respective organizations.