Advancing Transthyretin Amyloidosis Drug Development in an Evolving Treatment Landscape: Amyloidosis Forum Meeting Proceedings.
Mathew S MaurerPrem SomanAdrian F HernandezPablo García-PavíaJames E SignorovitchLee-Jen WeiMazen HannaFrederick L RubergMichelle M KittlesonDhruv S KaziSharmila DorbalaKristen HsuIsabelle LousadaRosalyn O AdigunPreston DunnmonJeffrey W KellyJulian David Gillmorenull nullPublished in: Advances in therapy (2024)
Given rapid evolution of natural history in ATTR amyloidosis, the utility of historical control data is limited. Leveraging contemporary real-world data is essential for clinical trial design. Evidence generation from clinical trials should address clinically relevant questions. Key factors in successful trial design must be informed by up-to-date data on natural history, prognostic factors, clinically meaningful thresholds, and sharing available clinical trial data. The Amyloidosis Forum includes the community of patients with ATTR amyloidosis, the physicians who treat them, and the sponsors and regulators who collectively stand ready to support further studies in order to develop novel effective therapies.
Keyphrases
- clinical trial
- electronic health record
- phase ii
- prognostic factors
- multiple myeloma
- big data
- phase iii
- study protocol
- open label
- primary care
- healthcare
- double blind
- mental health
- randomized controlled trial
- data analysis
- transcription factor
- machine learning
- social media
- single cell
- quantum dots
- smoking cessation
- case control