An open science pathway for drug marketing authorization—Registered drug approval

Naudet, Florian; Siebert, Maximilian; Boussageon, Rémy; Cristea, Ioana A.; Turner, Erick H.

doi:10.1371/journal.pmed.1003726

Cited by 14 publications

(15 citation statements)

References 9 publications

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“… 61 Data limitations at the time of regulatory approval may complicate estimation of cost-effectiveness for reimbursement decisions, 62 which may lead to potential waste of scarce public insurance resources. 63 , 64 Because regulatory approval standards represent not only the current thinking of regulators, but also guide the vision of what a “good drug” should be, 65 our findings highlight the need for more attention to the clinical meaningfulness—in terms of type and magnitude of benefit 66 —of newly authorized and investigational cancer therapies in China. 67 , 68 …”

Section: Discussionmentioning

confidence: 99%

Overall Survival Benefits of Cancer Drugs Approved in China From 2005 to 2020

et al. 2022

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Key Points Question What is the overall survival benefit of cancer drugs approved in China between 2005 and 2020? Findings In this mixed-methods study comprising a systematic review and cross-sectional analysis of 78 cancer drugs for 141 indications, 68 new cancer drug indications approved by Chinese authorities between 2005 and 2020 had documented evidence of overall survival benefit, and 34 did not prolong life. Meaning Given regulatory reforms in recent years, these findings highlight the need to routinely monitor the clinical benefits of new cancer therapies in China.

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Section: Discussionmentioning

confidence: 99%

Overall Survival Benefits of Cancer Drugs Approved in China From 2005 to 2020

et al. 2022

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“…In addition, HAS calls for greater transparency in the whole process of generating evidence through initiatives such as registered report publications, data and clinical study reports sharing. 20 Finally, HAS will remain attentive to emerging methods to reduce evidence gaps and accelerate clinical developments. In particular, the increasing use of artificial intelligence, in silico trials and disease modelling, might become a complementary path to optimising clinical development, as illustrated by CRESIM in helping to choose the best design in rare diseases.…”

Section: Box 1 Methodological Points Of Attention Has Should Consider...mentioning

confidence: 99%

Rapid access to innovative medicinal products while ensuring relevant health technology assessment. Position of the French National Authority for Health

Vanier

Fernandez

Kelley

et al. 2023

BMJ EBM

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“…Success stories like the Good Pharma Score Card show that data-sharing rates rise when sponsors are made aware of its inaccessibility [54]. We have recently proposed the concept of registered drug approvals, an open science pathway for drug marketing authorisation which could incentivise data-sharing, among other open sciences practices [55].…”

Section: Perspectivesmentioning

confidence: 99%

Data-sharing and re-analysis for main studies assessed by the European Medicines Agency—a cross-sectional study on European Public Assessment Reports

Siebert

Gaba

Renault

et al. 2022

BMC Med

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Background Transparency and reproducibility are expected to be normative practices in clinical trials used for decision-making on marketing authorisations for new medicines. This registered report introduces a cross-sectional study aiming to assess inferential reproducibility for main trials assessed by the European Medicines Agency. Methods Two researchers independently identified all studies on new medicines, biosimilars and orphan medicines given approval by the European Commission between January 2017 and December 2019, categorised as ‘main studies’ in the European Public Assessment Reports (EPARs). Sixty-two of these studies were randomly sampled. One researcher retrieved the individual patient data (IPD) for these studies and prepared a dossier for each study, containing the IPD, the protocol and information on the conduct of the study. A second researcher who had no access to study reports used the dossier to run an independent re-analysis of each trial. All results of these re-analyses were reported in terms of each study’s conclusions, p-values, effect sizes and changes from the initial protocol. A team of two researchers not involved in the re-analysis compared results of the re-analyses with published results of the trial. Results Two hundred ninety-two main studies in 173 EPARs were identified. Among the 62 studies randomly sampled, we received IPD for 10 trials. The median number of days between data request and data receipt was 253 [interquartile range 182–469]. For these ten trials, we identified 23 distinct primary outcomes for which the conclusions were reproduced in all re-analyses. Therefore, 10/62 trials (16% [95% confidence interval 8% to 28%]) were reproduced, as the 52 studies without available data were considered non-reproducible. There was no change from the original study protocol regarding the primary outcome in any of these ten studies. Spin was observed in the report of one study. Conclusions Despite their results supporting decisions that affect millions of people’s health across the European Union, most main studies used in EPARs lack transparency and their results are not reproducible for external researchers. Re-analyses of the few trials with available data showed very good inferential reproducibility. Trial registration https://osf.io/mcw3t/

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An open science pathway for drug marketing authorization—Registered drug approval

Abstract: Florian Naudet and co-authors propose a pathway involving registered criteria for evaluation and approval of new drugs.

Cited by 14 publications

References 9 publications

Overall Survival Benefits of Cancer Drugs Approved in China From 2005 to 2020

Overall Survival Benefits of Cancer Drugs Approved in China From 2005 to 2020

Rapid access to innovative medicinal products while ensuring relevant health technology assessment. Position of the French National Authority for Health

Data-sharing and re-analysis for main studies assessed by the European Medicines Agency—a cross-sectional study on European Public Assessment Reports

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