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Value & Access

Pre-Launch Access Strategy for a Rare Disease Therapy with Surrogate Endpoints

image of pharmacy community engagement activities
Client Profile

Mid-size Biopharmaceutical

Key Stakeholders

Market Access, HEOR, Medical Affairs

Asset

Pre-Launch

Disease Area

Rare Disease

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Client Profile

Mid-size Biopharmaceutical

Key Stakeholders

Market Access, HEOR, Medical Affairs

Asset

Pre-Launch

Disease Area

Rare Disease

Situation

A mid-size biopharmaceutical company was preparing for the pre-launch of a rare disease therapy with limited comparative data.

At the same time, payers were applying increasing scrutiny to surrogate endpoints and questioning whether they reflected meaningful patient benefit.

The client engaged Alkemi to assess payer perceptions, anticipate objections, and define a pre-launch access and evidence strategy to reduce reimbursement risk.

Approach

1. Assessed recent rare disease analogues to understand how payers evaluated surrogate endpoints and value thresholds.

2. Conducted payer and KOL advisory boards to test value messages, assess credibility, and surface anticipated objections.

3. Synthesized findings into clear guidance to strengthen the value narrative and inform pre-launch access planning.

The focus throughout:

Identify access risk early and align evidence strategy to payer expectations.

Deliverables

1
Analogue assessment readout documenting payer perceptions of surrogate endpoints and value thresholds.
2
Advisory board summary capturing payer and KOL feedback on value messages, objections, and evidence gaps.
3
Pre-launch access and evidence strategy outlining near-term mitigation tactics and longer-term evidence priorities.
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Impact

"This was exactly what we needed. You helped us see how payers were interpreting the data and gave us a clear path to address it." - Senior Director, Market Access

Clearer Payer Expectations:

The client gained clarity on evidence thresholds and decision drivers for reimbursement.

Stronger Pre-Launch Narrative:

Value messaging was refined to address payer concerns and reduce access risk.

More Focused Evidence Priorities:

Findings informed both near-term tactics and longer-term evidence generation planning.

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