Uncertainty in assessing value of oncology treatments.

C. Daniel Mullins, Russ Montgomery, Sean Tunis

Research output: Contribution to journalArticle

Abstract

Patients, clinicians, payers, and policymakers face an environment of significant evidentiary uncertainty as they attempt to achieve maximum value, or the greatest level of benefit possible at a given level of cost in their respective health care decisions. This is particularly true in the area of oncology, for which published evidence from clinical trials is often incongruent with real-world patient care, and a substantial portion of clinical use is for off-label indications that have not been systematically evaluated. It is this uncertainty in the knowledge of the clinical harms and benefits associated with oncology treatments that prevents postregulatory decision makers from making accurate assessments of the value of these treatments. Because of the incentives inherent in the clinical research enterprise, randomized control trials (RCTs) are designed for the specific purpose of regulatory approval and maximizing market penetration. The pursuit of these goals results in RCT study designs that achieve maximal internal validity at the expense of generalizability to diverse real-world patient populations that may have significant comorbidities and other clinically mitigating factors. As such, systematic reviews for the purposes of coverage and treatment decisions often find relevant and high-quality evidence to be limited or nonexistent. For a number of reasons, including frequent off-label use of medications and the expedited approval process for cancer drugs by the U.S. Food and Drug Administration, this situation is exacerbated in the area of oncology. This paper investigates the convergence of incentives and circumstances that lead to widespread uncertainty in oncology and proposes new paradigms for clinical research, including pragmatic clinical trials, methodological guidance, and coverage with evidence development. Each of these initiatives would support the design of clinical research that is more informative for postregulatory decision makers, and would therefore reduce uncertainty and provide greater confidence in conclusions about the value of these treatments.

Original languageEnglish (US)
Pages (from-to)58-64
Number of pages7
JournalOncologist
Volume15 Suppl 1
DOIs
StatePublished - 2010
Externally publishedYes

Fingerprint

Uncertainty
Off-Label Use
Motivation
Pragmatic Clinical Trials
Drug Approval
United States Food and Drug Administration
Therapeutics
Research
Comorbidity
Decision Making
Patient Care
Research Design
Clinical Trials
Delivery of Health Care
Costs and Cost Analysis
Population
Neoplasms

ASJC Scopus subject areas

  • Medicine(all)

Cite this

Uncertainty in assessing value of oncology treatments. / Mullins, C. Daniel; Montgomery, Russ; Tunis, Sean.

In: Oncologist, Vol. 15 Suppl 1, 2010, p. 58-64.

Research output: Contribution to journalArticle

Mullins, CD, Montgomery, R & Tunis, S 2010, 'Uncertainty in assessing value of oncology treatments.', Oncologist, vol. 15 Suppl 1, pp. 58-64. https://doi.org/10.1634/theoncologist.2010-S1-58
Mullins, C. Daniel ; Montgomery, Russ ; Tunis, Sean. / Uncertainty in assessing value of oncology treatments. In: Oncologist. 2010 ; Vol. 15 Suppl 1. pp. 58-64.
@article{4fbf3db0ebd042fa9aebd5f6e9fa50bd,
title = "Uncertainty in assessing value of oncology treatments.",
abstract = "Patients, clinicians, payers, and policymakers face an environment of significant evidentiary uncertainty as they attempt to achieve maximum value, or the greatest level of benefit possible at a given level of cost in their respective health care decisions. This is particularly true in the area of oncology, for which published evidence from clinical trials is often incongruent with real-world patient care, and a substantial portion of clinical use is for off-label indications that have not been systematically evaluated. It is this uncertainty in the knowledge of the clinical harms and benefits associated with oncology treatments that prevents postregulatory decision makers from making accurate assessments of the value of these treatments. Because of the incentives inherent in the clinical research enterprise, randomized control trials (RCTs) are designed for the specific purpose of regulatory approval and maximizing market penetration. The pursuit of these goals results in RCT study designs that achieve maximal internal validity at the expense of generalizability to diverse real-world patient populations that may have significant comorbidities and other clinically mitigating factors. As such, systematic reviews for the purposes of coverage and treatment decisions often find relevant and high-quality evidence to be limited or nonexistent. For a number of reasons, including frequent off-label use of medications and the expedited approval process for cancer drugs by the U.S. Food and Drug Administration, this situation is exacerbated in the area of oncology. This paper investigates the convergence of incentives and circumstances that lead to widespread uncertainty in oncology and proposes new paradigms for clinical research, including pragmatic clinical trials, methodological guidance, and coverage with evidence development. Each of these initiatives would support the design of clinical research that is more informative for postregulatory decision makers, and would therefore reduce uncertainty and provide greater confidence in conclusions about the value of these treatments.",
author = "Mullins, {C. Daniel} and Russ Montgomery and Sean Tunis",
year = "2010",
doi = "10.1634/theoncologist.2010-S1-58",
language = "English (US)",
volume = "15 Suppl 1",
pages = "58--64",
journal = "Oncologist",
issn = "1083-7159",
publisher = "AlphaMed Press",

}

TY - JOUR

T1 - Uncertainty in assessing value of oncology treatments.

AU - Mullins, C. Daniel

AU - Montgomery, Russ

AU - Tunis, Sean

PY - 2010

Y1 - 2010

N2 - Patients, clinicians, payers, and policymakers face an environment of significant evidentiary uncertainty as they attempt to achieve maximum value, or the greatest level of benefit possible at a given level of cost in their respective health care decisions. This is particularly true in the area of oncology, for which published evidence from clinical trials is often incongruent with real-world patient care, and a substantial portion of clinical use is for off-label indications that have not been systematically evaluated. It is this uncertainty in the knowledge of the clinical harms and benefits associated with oncology treatments that prevents postregulatory decision makers from making accurate assessments of the value of these treatments. Because of the incentives inherent in the clinical research enterprise, randomized control trials (RCTs) are designed for the specific purpose of regulatory approval and maximizing market penetration. The pursuit of these goals results in RCT study designs that achieve maximal internal validity at the expense of generalizability to diverse real-world patient populations that may have significant comorbidities and other clinically mitigating factors. As such, systematic reviews for the purposes of coverage and treatment decisions often find relevant and high-quality evidence to be limited or nonexistent. For a number of reasons, including frequent off-label use of medications and the expedited approval process for cancer drugs by the U.S. Food and Drug Administration, this situation is exacerbated in the area of oncology. This paper investigates the convergence of incentives and circumstances that lead to widespread uncertainty in oncology and proposes new paradigms for clinical research, including pragmatic clinical trials, methodological guidance, and coverage with evidence development. Each of these initiatives would support the design of clinical research that is more informative for postregulatory decision makers, and would therefore reduce uncertainty and provide greater confidence in conclusions about the value of these treatments.

AB - Patients, clinicians, payers, and policymakers face an environment of significant evidentiary uncertainty as they attempt to achieve maximum value, or the greatest level of benefit possible at a given level of cost in their respective health care decisions. This is particularly true in the area of oncology, for which published evidence from clinical trials is often incongruent with real-world patient care, and a substantial portion of clinical use is for off-label indications that have not been systematically evaluated. It is this uncertainty in the knowledge of the clinical harms and benefits associated with oncology treatments that prevents postregulatory decision makers from making accurate assessments of the value of these treatments. Because of the incentives inherent in the clinical research enterprise, randomized control trials (RCTs) are designed for the specific purpose of regulatory approval and maximizing market penetration. The pursuit of these goals results in RCT study designs that achieve maximal internal validity at the expense of generalizability to diverse real-world patient populations that may have significant comorbidities and other clinically mitigating factors. As such, systematic reviews for the purposes of coverage and treatment decisions often find relevant and high-quality evidence to be limited or nonexistent. For a number of reasons, including frequent off-label use of medications and the expedited approval process for cancer drugs by the U.S. Food and Drug Administration, this situation is exacerbated in the area of oncology. This paper investigates the convergence of incentives and circumstances that lead to widespread uncertainty in oncology and proposes new paradigms for clinical research, including pragmatic clinical trials, methodological guidance, and coverage with evidence development. Each of these initiatives would support the design of clinical research that is more informative for postregulatory decision makers, and would therefore reduce uncertainty and provide greater confidence in conclusions about the value of these treatments.

UR - http://www.scopus.com/inward/record.url?scp=77954651388&partnerID=8YFLogxK

UR - http://www.scopus.com/inward/citedby.url?scp=77954651388&partnerID=8YFLogxK

U2 - 10.1634/theoncologist.2010-S1-58

DO - 10.1634/theoncologist.2010-S1-58

M3 - Article

C2 - 20237219

AN - SCOPUS:77954651388

VL - 15 Suppl 1

SP - 58

EP - 64

JO - Oncologist

JF - Oncologist

SN - 1083-7159

ER -