Rare & Orphan Disease Programmes
Akrivia enables access to rich, curated data in underserved populations – crucial for precision medicine in rare CNS and neurodegenerative disorders.
Teams developing orphan CNS therapies and neurodegenerative assets
- Advanced patient detection (via NLP & EHR)
- Disease understanding and natural history
- Treatment pathway mapping
- Trial design and feasibility
- Accelerating development and regulatory acceptance
- Market access and value demonstration
- Natural history studies (complement phase 1 & 2)
Key Capabilities:
Rare Disease Patient Identification
- Leverage NLP and longitudinal EHR records to:
- detect undiagnosed or misdiagnosed cases
Treatment Pathway Mapping
- Build evidence:
- on current treatment paradigms and
- patient journeys to inform trial design and access strategies
RWE Generation for Regulatory and Market Access Support
- Retrospective and prospective RWE studies:
- to support new drug applications (NDAs)
- supplemental indication
- or label expansions.
- Generation of evidence on treatment patterns, unmet needs, and patient outcomes in real-world settings.
- Retrospective and prospective RWE studies:
- Support for post-marketing commitments (e.g., Phase IV observational studies) using real-world data.
- Regulatory Submission Support
- HTA and Payer Engagement (require evidence beyond RCTs to assess cost-effectiveness and real-world value)
- Generation of comparative effectiveness evidence vs. standard of care
- Technology Assessment (HTA) agencies require evidence beyond RCTs to assess cost-effectiveness and real-world value
- Budget impact modelling and resource utilization data using real-world cohorts
- Development of value dossiers and payer-friendly evidence packages tailored to local health systems (e.g., NICE, SMC)
- Market Access Acceleration (demonstrating real-world value and safety to facilitate faster reimbursement and adoption)
- Early insights into target populations, disease burden and care pathways to inform pricing and access strategy.
- Evidence on time to treatment, treatment switching, and adherence to support formulary inclusion.
- Localized RWE to support regional access in decentralized markets
- Label Expansion and Lifecycle Management (identify opportunities for extending product lifeycle and broadening therapeutic use)
- Identification of off-label use patterns and new indications through advanced analytics.
- Longitudinal tracking of real-world outcomes to support real-time value demonstration.
- Evidence to support label expansions and supplemental submissions.