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Research Methods in Neurology
January 12, 2022

Performing Cost-Effectiveness Analyses to Support Policy Making
Key Lessons From the Assessment of Aducanumab

March 1, 2022 issue
98 (9) 360-365

Abstract

The purpose of this article is to describe the process and the methods of cost-effectiveness analysis for clinicians interested in joining or leading aspects of this branch of evidence-based research. Cost-effectiveness is a useful tool for policymakers and is considered a starting point for discussions of fair pricing. Clinicians are important members of teams conducting cost-effectiveness analyses, particularly as it relates to integrating their clinical expertise into the decisions around the design and conduct of the analysis. Their input is essential in assuring that models adequately reflect clinical practice and are informed by expert judgments of how existing data can best be interpreted to build a comprehensive analysis of the clinical and economic outcomes of different treatment options. We illustrate specific contributions that clinicians are well positioned to make in these teams using a recent cost-effectiveness analysis of aducanumab that was conducted to support fair drug pricing. While discussing these contributions, we explain key components of a cost-effectiveness analysis, such as time horizon, health states, and perspective, to support understanding of the methods of cost-effectiveness by the clinical researchers and to promote a common dialogue among these multidisciplinary teams.

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Information & Authors

Information

Published In

Neurology®
Volume 98Number 9March 1, 2022
Pages: 360-365
PubMed: 35022308

Publication History

Received: September 10, 2021
Accepted: December 15, 2021
Published online: January 12, 2022
Published in print: March 1, 2022

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Disclosure

All authors are paid salaries through the Institute for Clinical and Economic Review. The work included within this article was not funded. The authors report no disclosures relevant to this article. Go to Neurology.org/N for full disclosures.

Study Funding

The authors report no targeted funding.

Authors

Affiliations & Disclosures

Jonathan D. Campbell, PhD
From the Institute for Clinical and Economic Review, Boston, MA.
Disclosure
Scientific Advisory Boards:
1.
NONE
Gifts:
1.
NONE
Funding for Travel or Speaker Honoraria:
1.
NONE
Editorial Boards:
1.
NONE
Patents:
1.
NONE
Publishing Royalties:
1.
NONE
Employment, Commercial Entity:
1.
NONE
Consultancies:
1.
(1) Merck and Company, (2) Ipsen Pharmaceuticals, (3) Institute for Clinical and Economic Review (prior to employment).
Speakers' Bureaus:
1.
NONE
Other Activities:
1.
NONE
Clinical Procedures or Imaging Studies:
1.
NONE
Research Support, Commercial Entities:
1.
ICER received financial support for non-assessment activities from the following entities: (1) Aetna, (2) America's Health Insurance Plans, (3) Anthem, (4) AbbVie, (5) Alnylam, (6) AstraZeneca, (7) Biogen, (8) Blue Shield of CA, (9) Cambia Health Services, (10) CVS, (11) Editas, (12) Express Scripts, (13) Genentech/Roche, (14) GlaxoSmithKline, (15) Harvard Pilgrim, (16) Health Care Service Corporation, (17) Health Partners, (18) Johnson & Johnson (Janssen), (19) Kaiser Permanente, (20) LEO Pharma, (21) Mallinckrodt, (22) Merck, (23) Novartis, (24) National Pharmaceutical Council, (25) Premera, (26) Prime Therapeutics, (27) Regeneron, (28) Sanofi, (29) Spark Therapeutics, (30) United Healthcare, (31) HealthFirst, (32) Prizer, (33) Boehringer-Ingelheim, (34) uniQure, (35) Evolve Pharmacy Solutions, (36) Humana, (37) Sun Life.
Research Support, Government Entities:
1.
NONE
Research Support, Academic Entities:
1.
NONE
Research Support, Foundations and Societies:
1.
ICER received grants from: (1) Arnold Ventures, (2) Kaiser Foundation Health Plan Inc., (3) The Patrick and Catherine Weldon Donaghue Medical Research Foundation. Dr. Campbell received grant funding from PhRMA Foundation while on faculty at University of Colorado, prior to joining ICER.
Stock/stock Options/board of Directors Compensation:
1.
NONE
License Fee Payments, Technology or Inventions:
1.
NONE
Royalty Payments, Technology or Inventions:
1.
NONE
Stock/stock Options, Research Sponsor:
1.
NONE
Stock/stock Options, Medical Equipment & Materials:
1.
NONE
Legal Proceedings:
1.
NONE
Melanie D. Whittington, PhD
From the Institute for Clinical and Economic Review, Boston, MA.
Disclosure
Scientific Advisory Boards:
1.
NONE
Gifts:
1.
NONE
Funding for Travel or Speaker Honoraria:
1.
NONE
Editorial Boards:
1.
NONE
Patents:
1.
NONE
Publishing Royalties:
1.
NONE
Employment, Commercial Entity:
1.
NONE
Consultancies:
1.
(1) Merck, (2) Ipsen Biophamaceuticals, (3) GalbraithWight All three consultancies were prior to my current employment at the Institute for Clinical and Economic Review.
Speakers' Bureaus:
1.
NONE
Other Activities:
1.
NONE
Clinical Procedures or Imaging Studies:
1.
NONE
Research Support, Commercial Entities:
1.
NONE
Research Support, Government Entities:
1.
(1) Veteran's Health Administration Center of Innovation for Veteran-Centered and Value-Driven Care, PI, 2017-2019
Research Support, Academic Entities:
1.
(1) University of Colorado Anschutz Medical Campus (2) University of Kansas Medical Center
Research Support, Foundations and Societies:
1.
(1) PhRMA Foundation, (2) Arnold Ventures, (3) The Patrick and Catherine Weldon Donaghue Medical Research Foundation, (4) Kaiser Foundation Health Plan Inc.
Stock/stock Options/board of Directors Compensation:
1.
NONE
License Fee Payments, Technology or Inventions:
1.
NONE
Royalty Payments, Technology or Inventions:
1.
NONE
Stock/stock Options, Research Sponsor:
1.
NONE
Stock/stock Options, Medical Equipment & Materials:
1.
NONE
Legal Proceedings:
1.
NONE
Steven D. Pearson, MD, MSc
From the Institute for Clinical and Economic Review, Boston, MA.
Disclosure
Scientific Advisory Boards:
1.
NONE
Gifts:
1.
NONE
Funding for Travel or Speaker Honoraria:
1.
NONE
Editorial Boards:
1.
NONE
Patents:
1.
NONE
Publishing Royalties:
1.
NONE
Employment, Commercial Entity:
1.
NONE
Consultancies:
1.
NONE
Speakers' Bureaus:
1.
NONE
Other Activities:
1.
NONE
Clinical Procedures or Imaging Studies:
1.
NONE
Research Support, Commercial Entities:
1.
NONE
Research Support, Government Entities:
1.
Commonwealth of Massachusetts, HPC-2020-001-PharmPricing, 2020-2021 US Department of Veterans Affairs, 36C24E20C0014, 2020-2021
Research Support, Academic Entities:
1.
NONE
Research Support, Foundations and Societies:
1.
California Health Care Foundation Kaiser Foundation Health Plan Inc. Laura and John Arnold Foundation Patrick and Catherine Weldon Donaghue Medical Research Foundation
Stock/stock Options/board of Directors Compensation:
1.
NONE
License Fee Payments, Technology or Inventions:
1.
NONE
Royalty Payments, Technology or Inventions:
1.
NONE
Stock/stock Options, Research Sponsor:
1.
NONE
Stock/stock Options, Medical Equipment & Materials:
1.
NONE
Legal Proceedings:
1.
NONE

Notes

Correspondence Dr. Campbell [email protected]
Go to Neurology.org/N for full disclosures. Funding information and disclosures deemed relevant by the authors, if any, are provided at the end of the article.

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  2. New International Pharmaco-Economic Collaboration on Alzheimer's Disease (IPECAD) Open-Source Model Framework for the Health Technology Assessment of Early Alzheimer’s Disease Treatment: Development and Use Cases, Value in Health, (2024).https://doi.org/10.1016/j.jval.2024.07.009
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