Papers & Publications

The NEWDIGS Publication Creation & Dissemination Policy can be found here.

Publications

C. Young; C. Quinn; M. Trusheim
Durable cell and gene therapy potential patient and financial impact: US projections of product approvals, patients treated, and product revenues
Drug Discovery Today , 01/2022 , 2022 ,
N.A. Ivica; C.M. Young
Tracking the CAR-T Revolution: Analysis of Clinical Trials of CAR-T and TCR-T Therapies for the Treatment of Cancer (1997–2020)
Healthcare , 08/2021 , 2021 , 10.3390/healthcare9081062
H.G. Eichler; M. Trusheim; B. Schwarser-Daum; K. Larholt; M. Zeitlinger; M. Brunninger; M. Sherman; D. Strutton; G. Hirsch
Precision reimbursement for precision medicine: using real world evidence to evolve from trial-and-project to track-and-pay to lean-and-predict
Clinical Pharmacology & Therapeutics , 10/2021 , 2021 , 10.1002/cpt.2471
K. Nam; K. Larholt; P. Beninger; D. Fritsche; D. Shoda; J. Ferguson; F. Bourgeois; D. Palmer; K. Katz; M. Courtney
Dynamic Dossier in the Cloud: A Sociotechnical Architecture for a Real‑Time and Metrics‑Based Data Tracking System with Gene and Cell Therapies as a Case Study
Therapeutic Innovation & Regulatory Science , 10/2020 , 2020 ,
J.F. Barlow; M.W. Courtney; M. Trusheim
Payer Perspectives on Gene Therapy Reimbursement
Pharmaceutical Executive , 04/2020 , 2020 ,
J.F. Barlow; M.W. Courtney; M. Trusheim
Payer Perspectives on Reimbursement of One-Time High-Cost Durable Therapies
Pharmaceutical Executive , 01/09/2020 , 2020 ,
J.F. Barlow; M. Yang; R. Teagarden
Are Payers Ready, Willing, and Able to Provide Access to New Durable Gene Therapies?
Value in Health , 06/2019 , 2019 , 10.1016/j.jval.2018.12.004
J. Barlow; R. Teagarden; M. Trusheim
Precision Financing of Durable, Potentially Curative Therapies
Pharmaceutical Executive , 01/2019 , 2019 ,
G. Hirsch
Leaping Together Toward Sustainable, Patient-Centered Innovation: The Value of a Multistakeholder Safe Haven for Accelerating System Change
Clinical Pharmacology & Therapeutics , 04/2019 , 2019 , 10.1002/cpt.1237
M.R. Trusheim; W.M. Cassidy; P.B. Bach
Alternative State-Level Financing for Hepatitis C Treatment- The "Netflix Model"
JAMA , 10/29/2018 , 2018 , 10.1001
D. Han; D.S. Mytelka; G.L. Warren; M. Ciarametaro; M. Trusheim
Improving Management of Gene and Cell Therapies: The Orphan Reinsurer and Benefit Manager (ORBM)
PharmaExec,com , 2018 ,
M. Boname; A.Wagner Gee; S. Addie; S.H. Beachy; T. Wizemann; M. Trusheim
Enabling Precision Medicine: The Role of Genetics in Clinical Drug Development: Proceedings of a Workshop
Forum on Drug Discovery, Development, and Translation , 2017 , 10.17226/24829
S. Blackburn
Adaptive Pathways are Creating Win-Win Scenarios for Patients and Pharma
AccessPoint, QuintilesIMS Real-World Insights , 05/2017 , 2017 ,
M.J. Cziraky; K.M. Larholt; M. Pollock
Conducting Pragmatic Trials: Design and Infrastructure Considerations for Successful Implementation of Trials Based on Real-World Evidence
HealthCore , 2017 ,
J.C. Bouvy; P. Jonsson; C. Longson; N. Crabb; S. Garner
Health Technology Assessment in the Context of Adaptive Pathways for Medicines in Europe: Challenges and Opportunities
Clinical Pharmacology & Therapeutics , 2016 , 10.1002/cpt.448
G. Hirsch; M. Trusheim; E. Cobbs; M. Bala; S. Garner; D. Hartman; K. Isaacs; M. Lumpkin; R. Lim; K. Oye; E. Pezalla; P. Saltonstall; H. Selker
Adaptive Biomedical Innovation: Evolving Our Global System to Sustainably and Safely Bring New Medicines to Patients in Need
Clinical Pharmacology & Therapeutics , 2016 , 10.1002/cpt.509
; P.W. Barone; A. Bellisario; C.L. Cooney; P.A. Sharp; A.J. Sinskey; S. Natesan; S.L. Springs
Leveraging Industry-Academia Collaborations in Adaptive Biomedical Innovation
Clinical Pharmacology & Therapeutics , 2016 , 10.1002/cpt.504
H.G. Eichler; B. Bloechl-Daum; P. Bauer; F. Bretz; J. Brown; L.V. Hampson; P. Honig; M. Krams; H. Leufkens; R. Lim; M.M. Lumpkin; M.J. Murphy; F. Pignatti; M. Posch; S. Schneeweiss; M. Trusheim; F. Koenig
“Threshold-crossing”: A Useful Way to Establish the Counterfactual in Clinical Trials?
Clinical Pharmacology & Therapeutics , 2016 , 10.1002/cpt.515
M.R. Trusheim; A.A. Shrier; Z. Antonijevic; R.A. Beckman; R.K. Campbell; C. Chen; K.T. Flaherty; J. Loewy; D. Lacombe; S. Madhavan; H.P. Selker; L.J. Esserman
PIPELINEs: Creating Comparable Clinical Knowledge Efficiently by Linking Trial Platforms
Clinical Pharmacology & Therapeutics , 2016 , 10.1002/cpt.514
D. Schulthess; L.G. Baird; M. Trusheim; T.F. Unger; M. Lumpkin; A. Hoos; S. Garner; P. Gavin; M. Goldman; N. Seigneuret; M. Chlebus; K. Van Baelen; R. Bergstrom; G. Hirsch
Medicines Adaptive Pathways to Patients (MAPPs): A Story of International Collaboration Leading to Implementation
Therapeutic Innovation & Regulatory Science , 2016 , 10.1177/2168479015618697
K.A. Oye; H.G. Eichler; A. Hoos; Y. Mori; T.M. Mullin; M. Pearson
Pharmaceuticals Licensing and Reimbursement in the European Union, United States, and Japan
Clinical Pharmacology & Therapeutics , 2016 , 10.1002/cpt.505
G.K. Raju; K. Gurumurthi; R. Domike; D. Kazandjian; G. Blumenthal; R. Pazdur; J. Woodcock
A Benefit–Risk Analysis Approach to Capture Regulatory Decision-Making: Non-Small Cell Lung Cancer
Clinical Pharmacology & Therapeutics , 2016 , 10.1002/cpt.501
H.G. Eichler; L.G. Baird; R. Barker; B. Bloechl-Daum; F. Børlum-Kristensen; J. Brown; R. Chua; S. Del Signore; U. Dugan; J. Ferguson; S. Garner; W. Goettsch; J. Haigh; P. Honig; A. Hoos; P. Huckle; T. Kondo; Y. Le Cam; H. Leufkens; R. Lim; C. Longson; M. Lumpkin; J. Maraganore; B. O'Rourke; K. Oye; E. Pezalla; F. Pignatti; J. Raine; G. Rasi; T. Salmonson; D. Samaha; S. Schneeweiss; P.D. Siviero; M. Skinner; ; T. Tominaga; M.R. Trusheim; S. Tunis; T.F. Unger; S. Vamvakas; G. Hirsch
From adaptive licensing to adaptive pathways: Delivering a flexible life-span approach to bring new drugs to patients
Clinical Pharmacology & Therapeutics , 2015 , 10.1002/cpt.59
K.A. Oye; G. Jain; M. Amador; R. Arnaout; J.S. Brown; W. Crown; J. Ferguson; E. Pezalla; J.A. Rassen; H.P. Selker; M. Trusheim; G. Hirsch
The next frontier: Fostering innovation by improving health data access and utilization
Clinical Pharmacology & Therapeutics , 2015 , 10.1002/cpt.191
M.R. Trusheim; E.R. Berndt
The clinical benefits, ethics, and economics of stratified medicine and companion diagnostics
Drug Discovery Today , 2015 , https://doi.org/10.1016/j.drudis.2015.10.017
M.R. Trusheim; L.G. Baird; S. Garner; R. Lim; N. Patel; G. Hirsch
The Janus initiative: A multi-stakeholder process and tool set for facilitating and quantifying Adaptive Licensing discussions
Health Policy and Technology , 2014 , https://doi.org/10.1016/j.hlpt.2014.10.004
J.R. Beshansky; P.R. Sheehan; K.J. Klima; N. Hadar; E.M. Vickery; H.P. Selker
A community consultation survey to evaluate support for and success of the IMMEDIATE trial
Clinical Trials , 2014 , 10.1177/1740774514526476
L.G. Baird; R. Banken; H.G. Eichler; F.B. Kristensen; D.K. Lee; J.C.W. Lim; R. Lim; C. Longson; E. Pezalla; T. Salmonson; D. Samaha; S. Tunis; J. Woodcock; G. Hirsch
Accelerated Access to Innovative Medicines for Patients in Need
Clinical Pharmacology & Therapeutics , 2014 , 10.1038/clpt.2014.145
R. Teagarden; T.F. Unger; G. Hirsch
Access and availability of orphan drugs in the United States: advances or cruel hoaxes?
Expert Opinion on Orphan Drugs , 2014 , 10.1517/21678707.2014.947265
L. Baird; G. Hirsch
Adaptive licensing: creating a safe haven for discussions
Scrip Regulatory Affairs , 20 August , 2013 ,
K. Oye; L.G. Baird; A. Chia; S. Hocking; P.B. Hutt; D. Lee; L. Norwalk; V. Salvatore
Legal Foundations of Adaptive Licensing
Clinical Pharmacology & Therapeutics , 2013 , 10.1038/clpt.2013.95
M. Papadaki; G. Hirsch
Curing Consortium Fatigue
Science Translational Medicine , 2013 , 10.1126/scitranslmed.3006903
L.G. Baird; M.R. Trusheim; H.G. Eichler; E.R. Berndt; G. Hirsch
Comparison of Stakeholder Metrics for Traditional and Adaptive Development and Licensing Approaches to Drug Development
Therapeutic Innovation & Regulatory Science , 2013 , 10.1177/2168479013487355
H.G. Eichler; K. Oye; L.G. Baird; E. Abadie; J. Brown; C.L. Drum; J. Ferguson; S. Garner; P. Honig; M. Hukkelhoven; J.C.W. Lim; R. Lim; M.M. Lumpkin; G. Neil; B. O'Rourke; E. Pezalla; D. Shoda; V. Seyfert-Margolis; E.V. Sigal; J. Sobotka; D. Tan; T.F. Unger; G. Hirsch
Adaptive licensing: taking the next step in the evolution of drug approval.
Clin Pharmacol Ther , 2012 Mar , 2012 , 10.1038/clpt.2011.345
J. Orloff; F. Douglas; J. Pinheiro; S. Levinson; M. Branson; P. Chaturvedi; E. Ette; P. Gallo; G. Hirsch; C. Mehta; N. Patel; S. Sabir; S. Springs; D. Stanski; M.R. Evers; E. Fleming; N. Singh; T. Tramontin; H. Golub
The future of drug development: advancing clinical trial design
Nat Rev Drug Discov , 2009/12//print , 2009 ,

Research Briefs & White Papers

FoCUS Research Brief 2022F204v055 Self-Insured Employers
LEAPS Research Brief 2021L309-v054 Patient Engagement
FoCUS White Paper: Emerging market solutions for financing and reimbursement of durable cell and gene therapies
FoCUS Research Brief 2021F203v053 Outcomes Tracking
FoCUS Research Brief 2021F201-v052 Resource Navigation
FoCUS White Paper: Warranty Model
FoCUS Research Brief 2020F207-v051 Pipeline Analysis
FoCUS White Paper: Tracking the CAR-T revolution
LEAPS Research Brief 2020L302v46 Downstream Innovation Part II
FoCUS Research Brief 2020F201-v050 Federal Communication Guidelines
FoCUS Research Brief 2019F212v048 Financing Challenges for Solid-Tumor T-Cell Therapies
LEAPS Research Brief 2019L311v045 Downstream Innovation Part I
FoCUS White Paper: Milestone-Based Contracts with Medicaid Plans
FoCUS White Paper: Payer Perspectives on Financing and Reimbursement of One-time High-cost Durable Treatments
FoCUS Research Brief 2019F209v043 SYLK Medicaid Review
LEAPS Research Brief 2019L309v042 Evidence Generation Platforms
FoCUS White Paper: The Role of Stop-Loss Insurance and Reinsurance in Managing Performance-Based Agreements
FoCUS Research Brief 2019F208v040 Expedited Insurance
LEAPS Research Brief 2019L308v041 Baking the Cake
FoCUS Research Brief 2019F206v039 Payer Perspectives Survey
LEAPS Research Brief 2019L305v010 MA Pilot Disease Selection
FoCUS Research Brief 2019F205v038 Pediatric Gene Therapy Launches
FoCUS Research Brief 2019F203-v037-Patient Caregiver Themes
FoCUS Research Brief 2019F201v032 Medicaid Best Price, January 2019
FoCUS White Paper: Precision Financing Solutions for Durable / Potentially Curative Therapies
FoCUS Research Brief 2018F212v030 CAR-Ts, December 2018
FoCUS Research Brief 2018F211v028 Model Contracts, November 2018
FoCUS Research Brief 2018F209-v026-COEs, September 2018
FoCUS White Paper: Designing financial solutions to ensure affordable access to cures
FoCUS Research Brief 2018F207-v024-Reinsurance, July 2018
FoCUS Research Brief 2018F206v022-Patient Mobility, June 2018
FoCUS Research Brief 2018F205v021-Orphan Reinsurer Benefit Manager, May 2018
FoCUS Research Brief 2018F204v018-Actuarial Risk, April 2018
FoCUS Research Brief 2018F203v015-Framework for Precision Financing, March 2018
FoCUS Research Brief 2018F202v014-Payer Segmentation, February 2018
FoCUS Research Brief 2017F212v012-Payer Survey, December 2017
Drug Safety Futures 2020
Catalyzing the Transformation of Healthcare Innovation