Resources

NEWDIGS produces original white papers, research briefs, and free downloadable tools to help stakeholders learn about and implement innovative strategies. We also publish in peer-reviewed journals and industry publications (see below). Additionally, NEWDIGS hosts Paying for Cures, a comprehensive resource for research and strategies to make innovative cell and gene therapies accessible to patients and sustainable systemwide.

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Title Topics Type Date
Implementation Brief: Innovative contracting 101 FoCUS Implementation Briefs August 22, 2024
Implementation Brief: Access considerations for innovative treatments FoCUS Implementation Briefs July 16, 2024
Implementation Brief: Key terms for payment innovation FoCUS Implementation Briefs June 19, 2024
Implementation Brief: Considerations for developers in applying the Multiple Best Price (MBP) rule to improve access to novel therapies FoCUS Implementation Briefs May 21, 2024
BioCentury OpEd: Biomedical Innovation Requires Health System Innovation Articles May 13, 2024
Implementation Brief: Understanding access issues for new therapies FoCUS Implementation Briefs April 25, 2024
Managing the Challenges of Paying for Gene Therapy: Strategies for Market Action and Policy Reform FoCUS Publications, Whitepapers April 23, 2024
Better Outcomes Tracking: A First Step In Improving Access To New Treatments FoCUS Articles, Publications April 8, 2024
Emerging market solutions for financing and reimbursement of gene therapies for sickle cell disease: Why do payment innovation? FoCUS Research Briefs February 28, 2024
Ask PAM: “How many?” FoCUS January 11, 2024
Analyzing 340B and ASP interactions: Do Federal program rules disincentivize the use of VBCs, despite Medicaid Best Price reform? FoCUS Research Briefs October 19, 2023
Are Cell and Gene Therapy programs a better bet? FoCUS Research Briefs October 11, 2023
Extending the vision of adaptive point-of-care platform trials to improve targeted use of drug therapy regimens: An agile approach in the learning healthcare system toolkit Publications August 29, 2023
Innovating methods for planning, producing, and using real-world evidence LEAPS Research Briefs July 20, 2023
Payer and developer perspectives on alternative payment models FoCUS Publications July 14, 2023
A practical approach for defining outcomes and thresholds for predictive healthcare algorithm development using real-world dataOut LEAPS Whitepapers March 28, 2023
Medicaid best price reforms to enable innovative payment models for cell and gene therapies FoCUS Publications December 29, 2022
How can self-insured employers prepare for the portfolio impact of highcost gene therapies coming to market? FoCUS Research Briefs April 28, 2022
Broadly Engaged Team Science Comes to Life in a Design Lab Publications February 27, 2022
Data for modelling US projections of product approvals, patients treated, and product revenues for durable cell and gene therapies FoCUS Publications February 11, 2022
Durable cell and gene therapy potential patient and financial impact: US projections of product approvals, patients treated, and product revenues FoCUS Publications December 24, 2021
Precision Reimbursement for Precision Medicine: Using Real-World Evidence to Evolve From Trial-and-Project to Track-and-Pay to Learn-and-Predict FoCUS Publications October 30, 2021
Precision for Whom? Partnering with patients to ensure that Precision Medicine targets what matters LEAPS Research Briefs September 30, 2021
Tracking the CAR-T Revolution: Analysis of Clinical Trials of CAR-T and TCR-T Therapies for the Treatment of Cancer (1997–2020) FoCUS Publications July 7, 2021
Emerging market solutions for financing and reimbursement of durable cell and gene therapies FoCUS Whitepapers June 16, 2021
Payer perspectives on outcomes tracking for value-based payment arrangements (VBPs) FoCUS Research Briefs March 25, 2021
The resource navigation challenges for patients and caregivers FoCUS Research Briefs January 29, 2021
Warranty Model: A potential precision financing solution for durable cell and gene therapies FoCUS Whitepapers October 30, 2020
Updated projection of US durable cell and gene therapies product-indication approvals based on December 2019 development pipeline FoCUS Research Briefs July 29, 2020
Tracking the CAR-T revolution: Analysis of clinical trials of CAR-T and TCR therapies for the treatment of cancer FoCUS Whitepapers June 25, 2020
Payer Perspectives on Gene Therapy Reimbursement FoCUS Articles April 1, 2020
Downstream Innovation Part II LEAPS Research Briefs February 27, 2020
Impact of FDA Guidelines on Communication between Developers and Payers on Metrics in Performance-Based Agreements FoCUS Research Briefs January 22, 2020
Payer Perspectives on Reimbursement of One-Time High-Cost Durable Therapies FoCUS Articles January 9, 2020
Precision Financing challenges for solid tumor adoptive T-cell therapies FoCUS Research Briefs December 19, 2019
Downstream Innovation Part I LEAPS Research Briefs November 27, 2019
Tools for Implementation of Precision Financing Solutions Within Medicaid Plans FoCUS Whitepapers November 21, 2019
Advancing Healthcare through Innovation and Collaboration LEAPS Whitepapers November 6, 2019
Payer Perspectives on Financing and Reimbursement of One-time High-cost Durable Treatments FoCUS Whitepapers October 11, 2019
Tracking Medicaid Coverage of Durable Cell and Gene Therapies FoCUS Research Briefs September 26, 2019
Transforming Real-World Evidence in Biomedical Innovation: Advancing the Design of Collaborative Disease-Focused Learning Ecosystems LEAPS Research Briefs September 26, 2019
The Role of Stop-Loss Insurance and Reinsurance in Managing Performance-Based Agreements FoCUS Whitepapers September 16, 2019
State Insurance Regulations Regarding Benefit Design (Deductible and Co-Pay Waivers) FoCUS Research Briefs August 23, 2019
Baking the Cake: A decision driven framework for planning fit-forpurpose evidence across stakeholders LEAPS Research Briefs August 22, 2019
Payer Perspectives Survey FoCUS Research Briefs June 26, 2019
Estimating the Clinical Pipeline of Cell and Gene Therapies and Their Potential Economic Impact on the US Healthcare System FoCUS Articles June 1, 2019
Are Payers Ready, Willing, and Able to Provide Access to New Durable Gene Therapies? FoCUS Articles June 1, 2019
Massachusetts Pilot Disease Selection LEAPS Research Briefs May 24, 2019
Pediatric Gene Therapy Launches FoCUS Research Briefs May 24, 2019
Patient & Caregiver Themes Survey FoCUS Research Briefs March 21, 2019
Precision Financing of Durable, Potentially Curative Therapies FoCUS Articles January 27, 2019
Precision Financing Solutions for Durable / Potentially Curative Therapies FoCUS Whitepapers January 24, 2019
Incorporation of Value-Based Payment Agreements into the Calculation of Medicaid Drug Rebates FoCUS Research Briefs January 24, 2019
Clinical trials and investment trends for novel CAR-T and TCR therapies FoCUS Research Briefs December 21, 2018
Model Contracts for Innovative Oncology Therapies FoCUS Research Briefs November 29, 2018
Leaping Together Toward Sustainable, Patient‐Centered Innovation: The Value of a Multistakeholder Safe Haven for Accelerating System Change LEAPS Articles November 2, 2018
Alternative State-Level Financing for Hepatitis C Treatment—The “Netflix Model” FoCUS Articles October 29, 2018
Role of COE networks in curative cellular oncology therapies FoCUS Research Briefs September 21, 2018
Improving Management of Gene and Cell Therapies: The Orphan Reinsurer and Benefit Manager (ORBM) FoCUS Articles September 10, 2018
Improving Management of Gene and Cell Therapies Articles September 10, 2018
Designing financial solutions to ensure affordable access to cures FoCUS Whitepapers August 23, 2018
Stop-Loss Insurance or Reinsurance for Multiyear Contracts FoCUS Research Briefs July 13, 2018
Impact of Patient Mobility on Annuity/Performance-Based Contracting FoCUS Research Briefs June 15, 2018
Orphan Reinsurer Benefit Managers (ORBMs) FoCUS Research Briefs May 22, 2018
Impact of Actuarial Risk on Health Plans FoCUS Research Briefs April 19, 2018
Designing Precision Financing for Cures FoCUS Research Briefs March 15, 2018
The heterogeneity of health care payers suggests that durable therapies need precision financing targeted to each payer segment FoCUS Research Briefs February 16, 2018
Payers Open to Innovative Financing Mechanisms for High Cost Gene Therapies FoCUS Research Briefs December 15, 2017
Adaptive Pathways create win-win scenarios for patients and pharma Adaptive Licensing Articles May 1, 2017
Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop Publications March 8, 2017
Leveraging Industry-Academia Collaborations in Adaptive Biomedical Innovation Articles December 1, 2016
Pharmaceuticals Licensing and Reimbursement in the European Union, United States, and Japan Articles December 1, 2016
A Benefit–Risk Analysis Approach to Capture Regulatory Decision-Making: Non-Small Cell Lung Cancer Articles December 1, 2016
Health Technology Assessment in the Context of Adaptive Pathways for Medicines in Europe: Challenges and Opportunities Adaptive Licensing Articles December 1, 2016
“Threshold‐crossing”: A Useful Way to Establish the Counterfactual in Clinical Trials? Articles September 21, 2016
PIPELINEs: Creating Comparable Clinical Knowledge Efficiently by Linking Trial Platforms Articles September 19, 2016
Adaptive Biomedical Innovation: Evolving Our Global System to Sustainably and Safely Bring New Medicines to Patients in Need Articles September 14, 2016
Medicines Adaptive Pathways to Patients (MAPPs): A Story of International Collaboration Leading to Implementation Adaptive Licensing Articles December 10, 2015
The clinical benefits, ethics, and economics of stratified medicine and companion diagnostics Articles December 1, 2015
The next frontier: Fostering innovation by improving health data access and utilization Articles November 1, 2015
From adaptive licensing to adaptive pathways: Delivering a flexible life-span approach to bring new drugs to patients Adaptive Licensing Articles March 1, 2015
The Janus initiative: A multi-stakeholder process and tool set for facilitating and quantifying Adaptive Licensing discussions Adaptive Licensing Publications December 1, 2014
Accelerated Access to Innovative Medicines for Patients in Need Articles November 1, 2014
Access and availability of orphan drugs in the United States: advances or cruel hoaxes? Articles November 1, 2014
A community consultation survey to evaluate support for and success of the IMMEDIATE trial Articles April 1, 2014
Legal Foundations of Adaptive Licensing Adaptive Licensing Publications September 1, 2013
Curing Consortium Fatigue Publications August 28, 2013
Comparison of Stakeholder Metrics for Traditional and Adaptive Development and Licensing Approaches to Drug Development Adaptive Licensing Publications July 1, 2013
Adaptive licensing: taking the next step in the evolution of drug approval Adaptive Licensing Publications March 24, 2012
Catalyzing the Transformation of Healthcare Innovation Whitepapers January 25, 2010
The future of drug development: advancing clinical trial design Publications December 1, 2009
Drug Safety Futures 2020 Whitepapers May 25, 2009

Publications

Van Overbeeke, E., Michelsen, S., Toumi, M., Stevens, H., Trusheim, M., Huys, I., & Simoens, S. (2021). Market access of gene therapies across Europe, USA, and Canada: challenges, trends, and solutions. Drug Discovery Today, 26(2), 399–415. https://doi.org/10.1016/j.drudis.2020.11.024
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. https://doi.org/10.1080/14737167.2023.2193331
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. https://doi.org/10.1007/s43441-022-00492-z
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. https://doi.org/10.1080/14737167.2023.2159813
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. https://doi.org/10.1002/cpt.2471
Payer Perspectives on Reimbursement of One-Time High-Cost Durable Therapies | Pharmaceutical Executive. (n.d.). Retrieved February 12, 2020, from http://www.pharmexec.com/payer-perspectives-reimbursement-one-time-high-cost-durable-therapies
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. https://doi.org/10.1002/cpt.59
Resnick, J. (n.d.). AccessPoint is published twice yearly by QuintilesIMS Real-World Insights. 60. https://www.iqvia.com/-/media/quintilesims/pdfs/accesspoint/quintilesims-rwi-accesspoint-may2017.pdf
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. https://doi.org/10.1002/cpt.1237
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. https://doi.org/10.1016/j.jval.2018.12.004
Trusheim, J. B. R. T. M. (n.d.). Precision Financing of Durable, Potentially Curative Therapies. Retrieved May 24, 2019, from http://www.pharmexec.com/precision-financing-durable-potentially-curative-therapies
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 https://www.healthcore.com/pct/
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. https://doi.org/10.1038/clpt.2011.345
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. https://doi.org/10.1038/nrd3025
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. https://doi.org/10.1177/2168479013487355
Papadaki, M., & Hirsch, G. (2013). Curing Consortium Fatigue. Science Translational Medicine, 5(200), 200fs35-200fs35. https://doi.org/10.1126/scitranslmed.3006903
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. https://doi.org/10.1038/clpt.2013.95
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. https://doi.org/10.1016/j.hlpt.2014.10.004
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. https://doi.org/10.1517/21678707.2014.947265
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. https://doi.org/10.1038/clpt.2014.145
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. https://doi.org/10.1177/1740774514526476
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. https://doi.org/10.1016/j.drudis.2015.10.017
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. https://doi.org/10.1002/cpt.191
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. https://doi.org/10.1002/cpt.448
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. https://doi.org/10.1002/cpt.501
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. https://doi.org/10.1002/cpt.505
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 https://journals.sagepub.com/doi/10.1177/2168479015618697
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. https://doi.org/10.1002/cpt.514
“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 https://ascpt.onlinelibrary.wiley.com/doi/full/10.1002/cpt.515
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. https://doi.org/10.1002/cpt.504
Leveraging Industry‐Academia Collaborations in Adaptive Biomedical Innovation - Stewart - 2016 - Clinical Pharmacology & Therapeutics - Wiley Online Library. (n.d.). Retrieved November 14, 2018, from https://ascpt.onlinelibrary.wiley.com/doi/abs/10.1002/cpt.504
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 https://ascpt.onlinelibrary.wiley.com/doi/full/10.1002/cpt.509
Read “Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop” at NAP.edu. (n.d.). https://doi.org/10.17226/24829
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 http://www.pharmexec.com/improving-management-gene-and-cell-therapies
Trusheim, M. R., Cassidy, W. M., & Bach, P. B. (2018). Alternative State-Level Financing for Hepatitis C Treatment—The “Netflix Model.” JAMA. https://doi.org/10.1001/jama.2018.15782