A systematic review of real-world evidence (RWE) supportive of new drug and biologic license application approvals in rare diseases.
Shailja VaghelaKaniz Afroz TanniGeetanjoli BanerjeeVanja SikiricaPublished in: Orphanet journal of rare diseases (2024)
This systematic review highlights potential benefits of appropriately conducted RWE studies in RD, which can strengthen the clinical evidence for efficacy comparison and contextualization to support product approval efforts, particularly when a large magnitude of effect is observed for the new intervention. Nonetheless, quality and completeness of RWD and its comparability with trial data remain areas of concern that can serve as valuable learnings for advancing future science and regulatory approvals.
Keyphrases
- systematic review
- randomized controlled trial
- rheumatoid arthritis
- meta analyses
- quality improvement
- study protocol
- public health
- clinical trial
- electronic health record
- transcription factor
- emergency department
- big data
- phase ii
- climate change
- human health
- case control
- deep learning
- machine learning
- drug induced
- clinical evaluation