Workshop G: Danielle Rollmann, Cell and Gene Therapy from Laboratory to Market
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A Rare International Dialogue (Sunday, May 12, 2019) Theme One: Diagnosis and Beyond Cell and Gene Therapy from Laboratory to Market Workshop G-Danielle Rollmann, Next Wave Consulting
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Workshop G: Danielle Rollmann, Cell and Gene Therapy from Laboratory to Market
- 1. Global Access Discussions RDI/CORD Meeting Workshop G: Cell and Gene Therapy from Laboratory to Market May 12, 2019 Danielle Rollmann, Next Wave Consulting, LLC
- 2. Exciting time – lots of promise – but access has not always been easy Source: Alliancefor RegenerativeMedicines,Meeting on the Mediterranean2019, Day 2: WelcomeRemarks April 24, 2019, https://alliancerm.org/wp-content/uploads/2019/04/Med-Day-2-Welcome-Remarks-FINAL.pdf,accessedMay 8, 2019 © Next Wave Consulting,LLC 2019
- 3. Access Challenges Financing Affecting • Coverage • HTA • Valuation • Reimbursement Uncertainty of effect • Initial treatment success/effectiveness • Duration of effect 1 Y1 Y2 Y3 Y4 Y5 Transformative Therapies Benefit Cost Y1 Y2 Y3 Y4 Y5 Traditional Payment Timing • Misalignment of cost and benefits • Patient mobility 2 Actuarial Risk (risk of having a patient) • Eligible population size • Payer beneficiary mix • Challenges to predict uptake • Payer size • Existing risk management approaches 3 © Next Wave Consulting,LLC 2019
- 4. Innovative financing approaches may help address these challenges Performance Guarantee Milestone-based Contract Payment Timing Payment over Time Actuarial Uncertainty Reinsurance/Risk Pooling Uncertainty of effect Coverage with evidence development © Next Wave Consulting,LLC 2019
- 5. Performance Guarantee • Concept • To address clinical uncertainty • Refund or discount if target performance not achieved • Questions • Ability to align on relevant outcomes measures and terms for particular treatment • Definition of covered population • Outcome measure and threshold • Basis for refund (by patient or population) • Contract length • Variety of “what if” scenarios • Tracking patients over time • Administrative effort to establish and track • Coordination of care • Patient mobility Contract Treat X Assess Refund © Next Wave Consulting,LLC 2019
- 6. Milestone-based contract • Questions • Ability to align on relevant milestone, outcomes measures and terms for particular treatment • Fit with local pricing regulations • Fit with stop-loss or reinsurance • Concept • Refunds and/or payments driven by pre-specified events Contract Treat X Assess Manufacture Assess Duration of effect Initial payment Initial treatment effect Note: Consortiasuch as MIT NEWDIGS FoCUS have been advancinga variety of alternativepaymentmodel concepts. Milestone-basedcontractis a term FoCUS has used, thoughthe descriptionhere includesmore variations.© Next Wave Consulting,LLC 2019
- 7. Payment over Time • Concept • Spread initial cost over multiple years • Can also be linked to outcomes (performance guarantee) • To better align costs with expected benefits, spread costs for smaller payers, to help address a bolus of prevalent patients Y1 Y2 Y3 Y4 Y5 Transformative Therapies Benefit Cost Y1 Y2 Y3 Y4 Y5 • Questions • Ability to sign multi-year contracts • Accounting cost and revenue recognition rules may necessitate third party financial solutions • Fit with local pricing regulations • Fit with stop-loss or reinsurance • Patient mobility© Next Wave Consulting,LLC 2019
- 8. Reinsurance/Risk pooling • Concept: • Manage actuarial risk • Reinsurance / stop-loss insurance protects payers against unexpected, high cost claims • Specific stop-loss: protection against a high claim for any one individual • Aggregate stop-loss: cap on total amount of claims in a period of time • Risk pooling • The larger the risk pool the more predictable and stable claims and premiums • Considerations • Payers and self-insured employers will want to consider whether they have appropriate approaches and coverage • Over time, predictable claims would typically be carved out of these types of solutions, otherwise premiums will rise • New reinsurance solutions may be developed if the economics support it © Next Wave Consulting,LLC 2019
- 9. Access challenges Operational Gene Therapy Payers Providers Patients Care- givers Develop- ers Govern- ments • Education/expectations • Eligibility • COEs • Cross-border access and reimbursement • Out of pocket costs/Copay • Monitoring over time • Appropriate Reimbursement • Billing codes • Buy and bill • Shifts in site of care • COEs • Follow-on monitoring • Coordination of care © Next Wave Consulting,LLC 2019
- 10. Therapy Diversity Unique Therapy Patient Population Clinical benefit Alternatives Adminis- tration Duration of Effect Treatment Location Performance Guarantee Milestone-based Contract Payment Timing Payment over Time Actuarial Uncertainty Reinsurance/Risk Pooling Uncertainty of effect Coverage with evidence development Gene Therapy Payers Providers Patients Care- givers Develop- ers Govern- ments One size does not fit all… but there are common approaches © Next Wave Consulting,LLC 2019
- 11. Conclusions • Gene and cell therapies offer great promise, but have also a history of market access challenges for a variety of reasons • We need to be as innovative in our access approaches as these treatments are in their science • Need to start having the dialogue at a country level, working together to find solutions • Start early, as it will take time © Next Wave Consulting,LLC 2019