XTalks Webinar: Why early evidence and real-world data are a strategic asset in today’s biopharma landscape
Real-world evidence (RWE) has moved from a supporting role to a central position in biopharma launch and access planning. Once viewed as a supplementary asset, it is now a fundamental component of how organisations navigate approval, pricing and long-term commercial viability. This shift was articulated clearly in the recent xTalks session with Andrew Cummins, Vice President Business Development at Sciensus, and Noolie Gregory, Head of Evidence Generation at Sciensus, who provided candid insight into how the industry is adapting.
The message was consistent throughout the discussion: Early evidence is no longer an optional exercise. It is a strategic requirement.
Why RWE matters most where evidence is scarce
Real world evidence (RWE) has broad value, but its importance is most pronounced in rare diseases, oncology and specialised conditions where clinical trials are small, highly controlled or limited to specific regions. Andrew highlighted that some therapies enter market access discussions with only a handful of patients in their pivotal trial. In these situations, regulators and payers face significant uncertainty.
Early RWE helps reduce this uncertainty by answering the practical questions that trials cannot fully address:
- How do patients respond outside controlled environments
- What is the lived experience of dosing or titration adjustments
- What are the early signs of adherence challenges
- How do comorbidities influence outcomes
This insight provides better pricing confidence and strengthens the credibility of reimbursement submissions. In rare disease, where population size is small and every data point counts, early RWE can become the difference between conditional access and routine commissioning.
Real world evidence relevance varies strongly by country
United Kingdom (UK)
The UK is progressing toward a more formal RWE framework with a new regulatory model expected in 2026. The Medicines and Healthcare products Regulatory Agency (MHRA) has an active data strategy and a scientific dialogue programme focused on methodology and data quality. RWE is seen as meaningful when provenance and accuracy are clear.
Germany
Germany places strong emphasis on post-launch RWE, particularly for benefit assessments. Local RWE can influence negotiations and subsequent price adjustments with the AMNOG (Arzneimittelmarkt-Neuordnungsgesetz / Pharmaceuticals Market Reorganisation Act).
France
France prioritises clinical trial results but relies on RWE to support follow up assessments of therapeutic benefit. Real-world data often strengthens arguments for broader access or sustained reimbursement.
Italy
Italy’s managed entry agreements create a formal route for RWE. Evidence collected through these schemes helps determine continued funding, making early data generation especially valuable.
Start early and define the purpose
Choose methods based on feasibility, not aspiration
Focus on endpoints that matter to payers and prescribers
Three examples that show RWE in practice
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CAR-T therapies in the UK Cancer Drugs Fund
Patients accessed CAR-T therapy through conditional approval while additional RWE was gathered through the systemic anti-cancer therapy dataset. The outcomes matched clinical trial findings and demonstrated effectiveness across a broader population. This helped the therapy shift from conditional access to routine commissioning faster than expected.
Learning: Structured RWE can accelerate permanent reimbursement and reduce payer uncertainty.
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Rare paediatric therapy supported by community-based nursing
In a rare paediatric indication, Sciensus ran an EAP which transitioned into commercial distribution. The same nurse teams who supported administration now collect the mandated post-authorisation data. They also helped schools understand the condition and treatment.
Learning: Integrated service models can reduce burden on sites, improve adherence and create high-quality RWE.
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Early access vs non-interventional study evidence mapping
One client sought to measure some endpoints matched to their clinical trial including biomarkers and quality of life. The team mapped each metric to what could reasonably be collected in an EAP versus a structured non-interventional study. The result showed that some endpoints were feasible in EAPs while others required dedicated study infrastructure.
Learning: Evidence ambitions must be matched to realistic operational pathways. Extensive endpoints cannot be captured through early access alone.
RWE as a source of sustainable competitive advantage
What the discussion throughout the webinar made clear is that the companies gaining the strongest foothold in launch and reimbursement are those that integrate evidence planning with their commercial strategy. These companies recognise that approval alone is no longer enough. Regulators want assurance. Payers want proof. Clinicians and patients want to understand how a treatment behaves in real-world settings, not just in a clinical trial.
Early evidence offers that assurance. It helps shape pricing discussions. It informs local HTA narratives. It supports uptake and strengthens long-term value. Above all it ensures that patients receive effective therapies without delay.
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