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Paying for Cures Toolkit

Our free interactive resource helps you explore challenges and plan solutions to financing new cell and gene therapies.
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Quinn, C., Ciarametaro, M., Sils, B., Phares, S., & Trusheim, M. R. (2023). Value-based performance arrangements for chronic conditions: an economic simulation of Medicaid Drug Rebate Program reforms. Expert Review of Pharmacoeconomics & Outcomes Research, 0(0), 1–12.
Schaumberg, D., Larholt, K., Apgar, E., Pashos, C. L., & Hirsch, G. (2023). Examining Endpoint Concordance in Clinical Trials and Real-World Clinical Practice to Advance Real-World Evidence Utilization. Therapeutic Innovation & Regulatory Science.
Quinn, C., Ciarametaro, M., Sils, B., Phares, S., & Trusheim, M. (2023). Medicaid best price reforms to enable innovative payment models for cell and gene therapies. Expert Review of Pharmacoeconomics & Outcomes Research, 23(2), 191–203.
Eichler, H., Trusheim, M., Schwarzer‐Daum, B., Larholt, K., Zeitlinger, M., Brunninger, M., Sherman, M., Strutton, D., & Hirsch, G. (2022). Precision Reimbursement for Precision Medicine: Using Real‐World Evidence to Evolve From Trial‐and‐Project to Track‐and‐Pay to Learn‐and‐Predict. Clinical Pharmacology & Therapeutics, 111(1), 52–62.
Payer Perspectives on Reimbursement of One-Time High-Cost Durable Therapies | Pharmaceutical Executive. (n.d.). Retrieved February 12, 2020, from
Eichler, H.-G., Baird, L., Barker, R., Bloechl-Daum, B., Børlum-Kristensen, F., Brown, J., Chua, R., Del Signore, S., Dugan, U., Ferguson, J., Garner, S., Goettsch, W., Haigh, J., Honig, P., Hoos, A., Huckle, P., Kondo, T., Le Cam, Y., Leufkens, H., … Hirsch, G. (2015). From adaptive licensing to adaptive pathways: Delivering a flexible life-span approach to bring new drugs to patients. Clinical Pharmacology & Therapeutics, 97(3), 234–246.
Resnick, J. (n.d.). AccessPoint is published twice yearly by QuintilesIMS Real-World Insights. 60.
Hirsch, G. (2019). Leaping Together Toward Sustainable, Patient‐Centered Innovation: The Value of a Multistakeholder Safe Haven for Accelerating System Change. Clinical Pharmacology & Therapeutics, 105(4), 798–801.
Barlow, J. F., Yang, M., & Teagarden, J. R. (2019). Are Payers Ready, Willing, and Able to Provide Access to New Durable Gene Therapies? Value in Health, 22(6), 642–647.
Trusheim, J. B. R. T. M. (n.d.). Precision Financing of Durable, Potentially Curative Therapies. Retrieved May 24, 2019, from
How to Design and Implement Pragmatic Trials for Success: Generate Relevant and High-Level Real-World Evidence. (n.d.). HealthCore. Retrieved November 14, 2018, from
Eichler, H.-G., Oye, K., Baird, L. G., Abadie, E., Brown, J., Drum, C. L., Ferguson, J., Garner, S., Honig, P., Hukkelhoven, M., Lim, J. C. W., Lim, R., Lumpkin, M. M., Neil, G., O’Rourke, B., Pezalla, E., Shoda, D., Seyfert-Margolis, V., Sigal, E. V., … Hirsch, G. (2012). Adaptive licensing: taking the next step in the evolution of drug approval. Clinical Pharmacology and Therapeutics, 91(3), 426–437.
Orloff, J., Douglas, F., Pinheiro, J., Levinson, S., Branson, M., Chaturvedi, P., Ette, E., Gallo, P., Hirsch, G., Mehta, C., Patel, N., Sabir, S., Springs, S., Stanski, D., Evers, M. R., Fleming, E., Singh, N., Tramontin, T., & Golub, H. (2009). The future of drug development: advancing clinical trial design. Nature Reviews Drug Discovery, 8(12), 949–957.
Baird, L. G., Trusheim, M. R., Eichler, H.-G., Berndt, E. R., & Hirsch, G. (2013). Comparison of Stakeholder Metrics for Traditional and Adaptive Development and Licensing Approaches to Drug Development. Therapeutic Innovation & Regulatory Science, 47(4), 474–483.
Papadaki, M., & Hirsch, G. (2013). Curing Consortium Fatigue. Science Translational Medicine, 5(200), 200fs35-200fs35.
Oye, K., Baird, L. G., Chia, A., Hocking, S., Hutt, P. B., Lee, D., Norwalk, L., & Salvatore, V. (2013). Legal Foundations of Adaptive Licensing. Clinical Pharmacology & Therapeutics, 94(3), 309–311.
Trusheim, M. R., Baird, L. G., Garner, S., Lim, R., Patel, N., & Hirsch, G. (2014). The Janus initiative: A multi-stakeholder process and tool set for facilitating and quantifying Adaptive Licensing discussions. Health Policy and Technology, 3(4), 241–247.
Teagarden, J. R., Unger, T. F., & Hirsch, G. (2014). Access and availability of orphan drugs in the United States: advances or cruel hoaxes? Expert Opinion on Orphan Drugs, 2(11), 1147–1150.
Baird, L. G., Banken, R., Eichler, H.-G., Kristensen, F. B., Lee, D. K., Lim, J. C. W., Lim, R., Longson, C., Pezalla, E., Salmonson, T., Samaha, D., Tunis, S., Woodcock, J., & Hirsch, G. (2014). Accelerated Access to Innovative Medicines for Patients in Need. Clinical Pharmacology & Therapeutics, 96(5), 559–571.
Beshansky, J. R., Sheehan, P. R., Klima, K. J., Hadar, N., Vickery, E. M., & Selker, H. P. (2014). A community consultation survey to evaluate support for and success of the IMMEDIATE trial. Clinical Trials, 11(2), 178–186.
Trusheim, M. R., & Berndt, E. R. (2015). The clinical benefits, ethics, and economics of stratified medicine and companion diagnostics. Drug Discovery Today, 20(12), 1439–1450.
Oye, K. A., Jain, G., Amador, M., Arnaout, R., Brown, J. S., Crown, W., Ferguson, J., Pezalla, E., Rassen, J. A., Selker, H. P., Trusheim, M., & Hirsch, G. (2015). The next frontier: Fostering innovation by improving health data access and utilization. Clinical Pharmacology & Therapeutics, 98(5), 514–521.
Bouvy, J. C., Jonsson, P., Longson, C., Crabb, N., & Garner, S. (2016). Health Technology Assessment in the Context of Adaptive Pathways for Medicines in Europe: Challenges and Opportunities. Clinical Pharmacology & Therapeutics, 100(6), 594–597.
Raju, G. K., Gurumurthi, K., Domike, R., Kazandjian, D., Blumenthal, G., Pazdur, R., & Woodcock, J. (2016). A Benefit–Risk Analysis Approach to Capture Regulatory Decision-Making: Non-Small Cell Lung Cancer. Clinical Pharmacology & Therapeutics, 100(6), 672–684.
Oye, K. A., Eichler, H. G., Hoos, A., Mori, Y., Mullin, T. M., & Pearson, M. (2016). Pharmaceuticals Licensing and Reimbursement in the European Union, United States, and Japan. Clinical Pharmacology & Therapeutics, 100(6), 626–632.
Medicines Adaptive Pathways to Patients (MAPPs): A Story of International Collaboration Leading to Implementation - Duane Schulthess, Lynn G. Baird, Mark Trusheim, Thomas F. Unger, Murray Lumpkin, Anton Hoos, Sarah Garner, Pamela Gavin, Michel Goldman, Nathalie Seigneuret, Magda Chlebus, Karin Van Baelen, Richard Bergstrom, Gigi Hirsch, 2016. (n.d.). Retrieved November 14, 2018, from
Trusheim, M., Shrier, A., Antonijevic, Z., Beckman, R., Campbell, R., Chen, C., Flaherty, K., Loewy, J., Lacombe, D., Madhavan, S., Selker, H., & Esserman, L. (2016). PIPELINEs: Creating Comparable Clinical Knowledge Efficiently by Linking Trial Platforms. Clinical Pharmacology & Therapeutics, 100(6), 713–729.
“Threshold‐crossing”: A Useful Way to Establish the Counterfactual in Clinical Trials? - Eichler - 2016 - Clinical Pharmacology & Therapeutics - Wiley Online Library. (n.d.). Retrieved November 14, 2018, from
Stewart, S. R., Barone, P. W., Bellisario, A., Cooney, C. L., Sharp, P. A., Sinskey, A. J., Natesan, S., & Springs, S. L. (2016). Leveraging Industry-Academia Collaborations in Adaptive Biomedical Innovation. Clinical Pharmacology & Therapeutics, 100(6), 647–653.
Leveraging Industry‐Academia Collaborations in Adaptive Biomedical Innovation - Stewart - 2016 - Clinical Pharmacology & Therapeutics - Wiley Online Library. (n.d.). Retrieved November 14, 2018, from
Adaptive Biomedical Innovation: Evolving Our Global System to Sustainably and Safely Bring New Medicines to Patients in Need - Hirsch - 2016 - Clinical Pharmacology & Therapeutics - Wiley Online Library. (n.d.). Retrieved November 14, 2018, from
Read “Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop” at (n.d.).
Ciarametaro, M. T. D. S. M. G. L. W. D. H. M. (n.d.). Improving Management of Gene and Cell Therapies. Retrieved November 13, 2018, from
Trusheim, M. R., Cassidy, W. M., & Bach, P. B. (2018). Alternative State-Level Financing for Hepatitis C Treatment—The “Netflix Model.” JAMA.

Research briefs


How can self-insured employers prepare for the portfolio impact of highcost gene therapies coming to market?

April 28, 2022

Payer perspectives on outcomes tracking for value-based payment arrangements (VBPs)

March 25, 2021

The resource navigation challenges for patients and caregivers

January 29, 2021

Updated projection of US durable cell and gene therapies product-indication approvals based on December 2019 development pipeline

July 29, 2020

Impact of FDA Guidelines on Communication between Developers and Payers on Metrics in Performance-Based Agreements

January 22, 2020

Precision Financing challenges for solid tumor adoptive T-cell therapies

December 19, 2019

Tracking Medicaid Coverage of Durable Cell and Gene Therapies

September 26, 2019

State Insurance Regulations Regarding Benefit Design (Deductible and Co-Pay Waivers)

August 23, 2019

Payer Perspectives Survey

June 26, 2019

Pediatric Gene Therapy Launches

May 24, 2019

Patient & Caregiver Themes Survey

March 21, 2019

Incorporation of Value-Based Payment Agreements into the Calculation of Medicaid Drug Rebates

January 24, 2019

Clinical trials and investment trends for novel CAR-T and TCR therapies

December 21, 2018

Model Contracts for Innovative Oncology Therapies

November 29, 2018

Role of COE networks in curative cellular oncology therapies

September 21, 2018

Stop-Loss Insurance or Reinsurance for Multiyear Contracts

July 13, 2018

Impact of Patient Mobility on Annuity/Performance-Based Contracting

June 15, 2018

Orphan Reinsurer Benefit Managers (ORBMs)

May 22, 2018

Impact of Actuarial Risk on Health Plans

April 19, 2018

Designing Precision Financing for Cures

March 15, 2018