In:
PLOS ONE, Public Library of Science (PLoS), Vol. 17, No. 6 ( 2022-6-24), p. e0265712-
Abstract:
The FDA’s Accelerated Approval program (AA) is a regulatory program to expedite availability of products to treat serious or life-threatening illnesses that lack effective treatment alternatives. Ideally, all of the many stakeholders such as patients, physicians, regulators, and health technology assessment [HTA] agencies that are affected by AA should benefit from it. In practice, however, there is intense debate over whether evidence supporting AA is sufficient to meet the needs of the stakeholders who collectively bring an approved product into routine clinical care. As AAs have become more common, it becomes essential to be able to determine their impact objectively and reproducibly in a way that provides for consistent evaluation of therapeutic decision alternatives. We describe the basic features of an approach for evaluating AA impact that accommodates stakeholder-specific views about potential benefits, risks, and costs. The approach is based on a formal decision-analytic framework combining predictive distributions for therapeutic outcomes (efficacy and safety) based on statistical models that incorporate findings from AA trials with stakeholder assessments of various actions that might be taken. The framework described here provides a starting point for communicating the value of a treatment granted AA in the context of what is important to various stakeholders.
Type of Medium:
Online Resource
ISSN:
1932-6203
DOI:
10.1371/journal.pone.0265712
DOI:
10.1371/journal.pone.0265712.g001
DOI:
10.1371/journal.pone.0265712.g002
DOI:
10.1371/journal.pone.0265712.g003
DOI:
10.1371/journal.pone.0265712.t001
DOI:
10.1371/journal.pone.0265712.t002
DOI:
10.1371/journal.pone.0265712.t003
DOI:
10.1371/journal.pone.0265712.t004
DOI:
10.1371/journal.pone.0265712.t005
DOI:
10.1371/journal.pone.0265712.t006
DOI:
10.1371/journal.pone.0265712.t007
DOI:
10.1371/journal.pone.0265712.s001
DOI:
10.1371/journal.pone.0265712.s002
DOI:
10.1371/journal.pone.0265712.s003
Language:
English
Publisher:
Public Library of Science (PLoS)
Publication Date:
2022
detail.hit.zdb_id:
2267670-3
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