Orphan Drugs: New thinking on RWE generation and value
From clinical trial planning and better education of HCPs to meeting the demands of diverse stakeholder groups, real-world evidence (RWE) is increasingly playing a pivotal role in the successful development and launch of an orphan drug. But how can pharma generate persuasive RWE that supports their proposition and meets stakeholders needs? Why are patient-reported outcomes growing in importance? How can AI deliver better insights from wearable technology? In this report experts in the field examine the RWE challenges and identify practical solutions.
Companies
Novartis, Lilly, Pfizer, Merck, BioMarin Pharmaceutical, Bluebird Bio, Insilico Medicine, Terumo Blood and Cell Technologies, PTC Therapeutics, Centogene, Celgene, Vertex
- Subject synopsis
- Research methodology and objectives
- Methodology
- Key research objectives
- Key insights summary
- Issues and insights
- Orphan drugs: definitions and market size
- Issue summary
- Questions
- Key insights
- Supporting quotes
- Intelligence exhibits
- The importance of a real-world evidence strategy
- Issue summary
- Questions
- Key insights
- Supporting quotes
- Factors to consider when generating RWE for orphan drugs
- Issue summary
- Questions
- Key insights
- Supporting quotes
- Intelligence exhibits
- RWE limitations and challenges
- Issue summary
- Questions
- Key insights
- Supporting quotes
- Implications of RWE in drug development and commercialisation
- Issue summary
- Questions
- Key insights
- Supporting quotes
- Intelligence exhibits
- Partnering with patient advocacy groups
- Issue summary
- Questions
- Key insights
- Supporting quotes
- Intelligence exhibit
- Opportunities for AI and machine learning
- Issue summary
- Questions
- Key insights
- Supporting quotes
- Outlook for RWE in orphan drugs
- Issue summary
- Questions
- Key insights
- Supporting quotes
- Intelligence exhibit
- Further Reading
- Abbreviations
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