A Comprehensive Algorithm for Approval of Health Technologies With, Without, or Only in Research: The Key Principles for Informing Coverage Decisions

TY - JOUR

T1 - A Comprehensive Algorithm for Approval of Health Technologies With, Without, or Only in Research

T2 - The Key Principles for Informing Coverage Decisions

AU - Claxton, Karl

AU - Palmer, Stephen

AU - Longworth, Louise

AU - Bojke, Laura

AU - Griffin, Susan

AU - Soares, Marta

AU - Spackman, Eldon

AU - Rothery, Claire

N1 - © 2016, International Society for Pharmacoeconomics and Outcomes Research (ISPOR). Published by Elsevier Inc.This is an author-produced version of the published paper. Uploaded in accordance with the publisher’s self-archiving policy. Further copying may not be permitted; contact the publisher for details.

PY - 2016/9/1

Y1 - 2016/9/1

N2 - BACKGROUND: The value of evidence about the performance of a technology and the value of access to a technology are central to policy decisions regarding coverage with, without, or only in research and managed entry (or risk-sharing) agreements.OBJECTIVES: We aim to outline the key principles of what assessments are needed to inform "only in research" (OIR) or "approval with research" (AWR) recommendations, in addition to approval or rejection.METHODS: We developed a comprehensive algorithm to inform the sequence of assessments and judgments that lead to different types of guidance: OIR, AWR, Approve, or Reject. This algorithm identifies the order in which assessments might be made, how similar guidance might be arrived at through different combinations of considerations, and when guidance might change.RESULTS: The key principles are whether the technology is expected to be cost-effective; whether the technology has significant irrecoverable costs; whether additional research is needed; whether research is possible with approval and whether there are opportunity costs that once committed by approval cannot be recovered; and whether there are effective price reductions. Determining expected cost-effectiveness is only a first step. In addition to AWR for technologies expected to be cost-effective and OIR for those not expected to be cost-effective, there are other important circumstances when OIR should be considered.CONCLUSIONS: These principles demonstrate that cost-effectiveness is a necessary but not sufficient condition for approval. Even when research is possible with approval, OIR may be appropriate when a technology is expected to be cost-effective due to significant irrecoverable costs.

AB - BACKGROUND: The value of evidence about the performance of a technology and the value of access to a technology are central to policy decisions regarding coverage with, without, or only in research and managed entry (or risk-sharing) agreements.OBJECTIVES: We aim to outline the key principles of what assessments are needed to inform "only in research" (OIR) or "approval with research" (AWR) recommendations, in addition to approval or rejection.METHODS: We developed a comprehensive algorithm to inform the sequence of assessments and judgments that lead to different types of guidance: OIR, AWR, Approve, or Reject. This algorithm identifies the order in which assessments might be made, how similar guidance might be arrived at through different combinations of considerations, and when guidance might change.RESULTS: The key principles are whether the technology is expected to be cost-effective; whether the technology has significant irrecoverable costs; whether additional research is needed; whether research is possible with approval and whether there are opportunity costs that once committed by approval cannot be recovered; and whether there are effective price reductions. Determining expected cost-effectiveness is only a first step. In addition to AWR for technologies expected to be cost-effective and OIR for those not expected to be cost-effective, there are other important circumstances when OIR should be considered.CONCLUSIONS: These principles demonstrate that cost-effectiveness is a necessary but not sufficient condition for approval. Even when research is possible with approval, OIR may be appropriate when a technology is expected to be cost-effective due to significant irrecoverable costs.

KW - cost-effectiveness

KW - coverage with evidence development

KW - health technology assessment

KW - only in research

UR - http://www.scopus.com/inward/record.url?scp=84966648431&partnerID=8YFLogxK

U2 - 10.1016/j.jval.2016.03.2003

DO - 10.1016/j.jval.2016.03.2003

M3 - Article

C2 - 27712718

SN - 1098-3015

VL - 19

SP - 885

EP - 891

JO - Value in Health

JF - Value in Health

IS - 6

ER -