A Cost-Benefit Analysis of Using Evidence of Effectiveness in Terms of Progression Free Survival in Making Reimbursement Decisions on New Cancer Therapies

Stevens Warren (), Tomas Philipson, Wu Yanyu, Chen Connie and Darius Lakdawalla
Additional contact information
Stevens Warren: Precision Health Economics, 11100 Santa Monica Blvd Suite 500, Los Angeles, CA 90025, USA
Wu Yanyu: Precision Health Economics, 800 Boylston Street, 16th Fl, Boston, MA 02199, USA
Chen Connie: Pfizer, 235 East 42nd Street, New York, NY 10017, USA

Forum for Health Economics & Policy, 2014, vol. 17, issue 1, 21-52

Abstract: Payers increasingly require evidence of a statistically significant difference in overall survival (OS) for reimbursement of new cancer therapies. At the same time, it becomes increasingly costly to design clinical trials that measure OS endpoints instead of progression-free survival (PFS) endpoints. While PFS is often an imperfect proxy for OS effects, it is also faster and cheaper to measure accurately. This study develops a general cost-benefit framework that quantifies the competing trade-offs of the use of PFS versus that of OS in oncology reimbursement. We then apply this general framework to the illustrative case of metastatic renal cell carcinoma (mRCC). In the particular case of mRCC, the framework demonstrates that the net benefit to society from basing reimbursement decisions on PFS endpoints could be between $271 and $1271 million in the United States, or between €171 and €1128 million in Europe. In longevity terms, waiting for OS data in this case would result in a net loss of 3549–14,557 life-years among US patients, or 6785–27,993 life-years for European patients. While more stringent standards for medical evidence improve accuracy, they also impose countervailing costs on patients in terms of foregone health gains. These costs must be weighed against the benefits of greater accuracy. The magnitudes of the costs and benefits may vary across tumor types and need to be quantified systematically.

Keywords: cancer; cost-benefit; evidence; reimbursement; surrogate endpoints (search for similar items in EconPapers)
Date: 2014
References: View references in EconPapers View complete reference list from CitEc
Citations:

Downloads: (external link)
https://doi.org/10.1515/fhep-2013-0025 (text/html)
For access to full text, subscription to the journal or payment for the individual article is required.

Related works:
This item may be available elsewhere in EconPapers: Search for items with the same title.

Export reference: BibTeX RIS (EndNote, ProCite, RefMan) HTML/Text

Persistent link: https://EconPapers.repec.org/RePEc:bpj:fhecpo:v:17:y:2014:i:1:p:32:n:5

Ordering information: This journal article can be ordered from
https://www.degruyter.com/journal/key/fhep/html

DOI: 10.1515/fhep-2013-0025

Access Statistics for this article

Forum for Health Economics & Policy is currently edited by Dana Goldman

More articles in Forum for Health Economics & Policy from De Gruyter
Bibliographic data for series maintained by Peter Golla ().