Designing an Early Access Pathway for a CNS Therapy Using Real-World and Claims Insights

Background:

A mid-stage biopharma company was advancing a novel CNS therapy into late Phase II, with early clinical signals suggesting meaningful benefit for patients with progressive neurological decline. While the science was promising, leadership faced a familiar CNS challenge: patients needed treatment sooner than regulatory and reimbursement timelines would allow.

Slow disease progression, subjective clinical endpoints, and long follow-up periods meant approval and payer decisions were still years away. Meanwhile, clinicians were encountering patients with limited alternatives, highlighting a growing access gap well before launch.

The Challenge:

The company needed an early access strategy that balanced urgency with responsibility. Key challenges included:

• Defining which real-world patients should qualify beyond the trial population
• Limited visibility into current treatment pathways and unmet needs
• Payer and regulator concerns around uncontrolled access and future reimbursement risk
• CNS-specific complexity, where short-term outcomes are difficult to measure

Objective:

The client partnered with Thelansis to design an asset-specific early access pathway that would:

• Enable responsible pre-approval patient access
• Use real-world and claims data to ground decisions in evidence
• Align early access design with future payer and HTA expectations
• Support regulatory, ethics committee, and clinician discussions with defensible insights

Our Approach:

1. Mapping the Real-World Patient Landscape

Using longitudinal claims and real-world datasets, we reconstructed how patients were actually diagnosed, treated, and progressed outside clinical trials. This allowed us to:

• Identify patient sub-segments most likely to deteriorate before approval
• Quantify delays between diagnosis, treatment initiation, and disease progression
• Highlight points where patients routinely fell outside standard care pathways

This step reframed early access from a “compassionate option” into a data-defined intervention for a specific unmet need.

2. Quantifying Unmet Need and Treatment Gaps

We translated real-world patterns into clear metrics that resonated with decision-makers:

• Size of the population with no viable treatment alternatives
• Duration of untreated or sub-optimally treated disease
• Geographic and system-level variation in access

These insights formed the backbone of the early access rationale, helping move discussions beyond anecdote toward evidence.

3. Designing an Evidence-Backed Early Access Model

Based on market dynamics and regulatory norms, we evaluated multiple pathways, including:

• Named-patient programs
• Managed access agreements
• Compassionate use frameworks

Each option was stress-tested against payer expectations, future pricing considerations, and operational feasibility. The result was a targeted early access design, not a one-size-fits-all program.

4. Supporting Stakeholder Dialogue

We translated complex data into clear, credible narratives tailored for different audiences:

• Regulators and ethics committees: patient safety, eligibility logic, and governance
• Payers: population size control, evidence generation, and downstream value
• Clinicians: clarity on who qualifies and why

Importantly, early access was positioned as a bridge to launch, not a substitute for reimbursement.

Outcome:

The client emerged with a clearly defined early access pathway that:

• Enabled pre-approval access for precisely defined patient segments
• Provided regulators and ethics committees with a credible, evidence-based rationale
• Preserved long-term pricing and reimbursement integrity
• Supported patients who could not afford to wait for full approval

In CNS, waiting for perfect data often leaves patients behind. This case demonstrates how real-world and claims insights can transform early access from a reactive measure into a strategic, patient-centric pathway.

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