Can the Development of Orphan Drugs Include Wider Patient Engagement? A Citizens' Jury to Explore a Promissory Notion
Notice bibliographique
Résumé
BACKGROUND: Drug development practices for rare diseases promote a promissory notion that the drug pipeline will succeed (with the next new drug/drug trial) and an imaginary of widening patient engagement (often desired but yet to be enacted). Contemporary industry practices of patient engagement are shaped by this promissory, and engagement often includes limited patient perspectives, typically relying on patients from established Patient Organisations or identified by Contract Research Organisations. OBJECTIVE: We sought to explore whether more deliberative methods might enable wider, more diverse patient engagement for orphan drug trials. DESIGN: Citizen's Jury co-designed with patient advisors. SETTING AND PARTICIPANTS: Results of an earlier ethnography of a biotech company's patient engagement practices, along with findings from clinical trials for patients with idiopathic pulmonary fibrosis, clinical practice and patient testimony, were presented to the Citizens' Jury made up of patients and carers. RESULTS: Jurors discussed and offered a 'verdict' on trial materials and processes that would optimise the engagement of more diverse patients for drug trials for rare diseases, suggesting that more could be done at an organisational level to ensure that potential trial participants were able to 'engage'. They asserted that the industry could do more to understand the unmet needs and wishes of a wider group of patients and should seek input from more diverse groups. DISCUSSION: The Citizens' Jury called for practices to enable wider engagement-for both drug trials and drug trial design-and more transparency about the risks associated with engagement, for individual patients and currently marginalised groups. CONCLUSIONS: Current drug development practices reify expert patient perspectives and often ignore the views of the wider group of patients who may participate in trials and/or use new medicines developed. More deliberative methods of engagement have the potential to democratise drug development and ensure that new medicines and trials meet the needs of a broader patient demographic. PATIENT OR PUBLIC CONTRIBUTION: A patient advisory group (PAG) comprising six people with IPF gave input into all aspects of the research design and conduct, including co-design of the Citizens' Jury. Two patients from international patient organisations served as a steering group (SG). Members of both groups provided their interpretations of the study findings and gave insight into their experiences in clinical design and participation.
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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 | 0,001 | 0,000 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,000 | 0,000 |
| Bibliométrie | 0,000 | 0,001 |
| Études des sciences et des technologies | 0,003 | 0,000 |
| Communication savante | 0,000 | 0,000 |
| Science ouverte | 0,000 | 0,000 |
| Intégrité de la recherche | 0,000 | 0,000 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,000 | 0,000 |
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; un appel candidat d’une seule tête enseignante, pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».