Abstract
Florian Naudet and co-authors propose a pathway involving registered criteria for evaluation and approval of new drugs.
Highlights
Systematic investigations demonstrate that approvals based on weak and limited evidence are the rule rather than the exception [4,5], there are notable instances where approval was based on strong evidence, such as the recent case of Coronavirus Disease 2019 (CAOUVI:DP-l1e9a)sveancoctienthesa.tCAOs aVrIeDsuÀ lt,1m9hoarsebdereungdsewfinitehdalisttCleo,riof naanvyi,raudsdDeidsebaesnee2f0it1a9rinetbhreosuengthetntcoeSystematic the market in a process increasingly reliant on disputable evidence [6] and divorced from public interest.AU : Anabbreviationlisthasbeencompiledforthoseusedthroughoutthetext:Pleaseverifythatallen
We propose to adapt the concept of “registered reports” to the process of regulatory drug approval and marketing authorization
The required number of direct pivotal trials as well as study designs will be set a priori for the research program, including large simple trials and nonambiguous criteria for success, e.g., 2 positive confirmatory studies with low risk of bias and a prospective meta-analysis. These criteria will define the prespecified analyses and criteria for statistical significance, and the precise criteria for clinical relevance, i.e., a minimal clinically important difference defined on clinically relevant outcomes or net benefit
Summary
It has been recommended for publication in PLOS Medicine. In Europe, companies can, at their discretion, obtain prior scientific advice from the EMA This consultative process between sponsor and regulator is not fit for purpose, as there is, in practice, no clear a priori consensus on the exact criteria that will be applied to adjudicate success.
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