IPRF Reflection Paper on Biodistribution
Notice bibliographique
Résumé
In a session held on May 15, 2015 during the 18th Annual American Society of Gene and Cell Therapy (ASGCT) Meeting, members of the International Pharmaceutical Regulators Forum Gene Therapy Working Group (IPRF GTWG) presented the current expectations of various international regulatory authorities for preclinical-nonclinical biodistribution (BD) studies for gene therapy products (excluding genetically modified cells) (http://www.iprp.global/working-group/gene-therapy). This session was a significant accomplishment because it allowed regulators from across the globe to have an open dialog on an issue facing stakeholders in this complex and fast-moving field. A summary of these presentations and the panel discussion was subsequently published as a meeting report.1Huang Y. Havert M. Gavin D. Serabian M. Ong L.L. McIntyre M.C. Ferry N. Kume A. Petrin D. Fu Y.-H. et al.Biodistribution studies: understanding international expectations.Mol. Ther. Methods Clin. Dev. 2016; 3: 16022Abstract Full Text Full Text PDF Scopus (4) Google Scholar This meeting report noted that regulators had similar perspectives worldwide on the definition of BD and the importance of collecting BD data. The regulators recognized that this agreement was a critical first step, but more work was needed to achieve a consensus position on the regulatory approach to BD assessment. A key effort for the IPRF GTWG since the 2015 ASGCT meeting has been continued dialog to develop consensus on regulatory expectations for the design and timing of BD studies and scientific considerations related to BD data analysis. To this end, a draft consensus position document was developed through subsequent IPRF GTWG meetings and finalized in a face-to-face meeting in May 2017. This document was then distributed to all members of the IPRF GTWG for additional comment and approval before acceptance by the IPRF Management Committee. We are now pleased to report that, in July 2018, the document was posted on the IPRF website as a Reflection Paper (http://development.iprp.backend.dev6.penceo.com/sites/default/files/2018-09/IPRP_GTWG_ReflectionPaper_BD_Final_2018_0713.pdf). Completion of the Reflection Paper is an important bookend to the 2015 ASGCT meeting. It represents a key milestone on a topic that was originally initiated to expedite the development of promising gene therapy products. Although this Reflection Paper is not a substitute for formal guidance from any regulatory authority, the scientific considerations outlined in this paper can be a starting point for discussion and will therefore likely have an impact on product development programs in this area. The BD Reflection Paper is also the first official document produced by the IPRF GTWG and is noteworthy as the first international document with regulatory consensus on a gene therapy topic since 2009 (http://www.ich.org/products/consideration-documents.html). As the interest in gene therapy products to treat a myriad of serious medical conditions continues to grow, the IPRF is poised to contribute a key role in this effort. (IPRF changed names in January 2018 and is now referred to as The International Pharmaceutical Regulators Programme [IPRP].)
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
Comment cette classification a été obtenuedéplier
Prédiction distillée sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Apprise à partir de 10 348 étiquettes directes de Codex et de 10 348 étiquettes directes de Gemma. Le mode candidate est l'union des têtes enseignantes seuillées; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont ni des étiquettes humaines ni des étiquettes directes de modèles de pointe.
Scores Codex et Gemma par catégorie
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,005 | 0,001 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,001 | 0,000 |
| Bibliométrie | 0,000 | 0,000 |
| Études des sciences et des technologies | 0,000 | 0,001 |
| Communication savante | 0,000 | 0,000 |
| Science ouverte | 0,000 | 0,000 |
| Intégrité de la recherche | 0,001 | 0,001 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,002 | 0,001 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; les deux têtes enseignantes s’accordent sur ce qui est montré ici.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».