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HEOR

Early Value and Evidence Roadmap for a Rare Disease Therapy

image of pharmacy community engagement activities
Client Profile

Small R&D focused Biotech

Key Stakeholders

R&D, Clinical Development, Commercial Lead, HEOR

Asset

Novel Phase IIb Therapy

Disease Area

Rare Disease

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Client Profile

Small R&D focused Biotech

Key Stakeholders

R&D, Clinical Development, Commercial Lead, HEOR

Asset

Novel Phase IIb Therapy

Disease Area

Rare Disease

Situation

The client was advancing a novel therapy for a rare disease and needed to define value early, before pivotal decisions were locked in.

With limited data, multiple stakeholders, and a compressed timeline, teams needed clarity on which outcomes would matter most to future payers, clinicians, and regulators.

The client engaged Alkemi to rapidly align on an early value story and define an evidence strategy that could support development decisions and future access.

Approach

1. Conducted a rapid review of available clinical data to identify emerging value themes and evidence gaps.

2. Led cross-functional working sessions with Clinical, R&D, HEOR, and Commercial leaders to align on priority value messages and decision needs.

3. Assessed potential clinical and RWE endpoints against future access and evaluation requirements.

4. Developed a clear, actionable evidence roadmap outlining recommended studies, sequencing, and rationale.

The focus throughout:

Create early clarity to guide development and reduce downstream access risk.

Deliverables

1
Preliminary value story grounded in available clinical data and future access considerations.
2
Endpoint recommendations to support clinical development and evidence generation planning.
3
Evidence gap assessment identifying priority value messages and data needs.
4
Comprehensive evidence roadmap guiding planning across Clinical, HEOR, and Commercial teams.
5
6

Impact

"Alkemi helped us clarify exactly what evidence we needed and gave us a roadmap we could implement immediately." - VP of Medical Affairs

Stronger Early Value Alignment:

Teams aligned early on how value should be articulated and supported as the program advanced.

Clear Development Direction:

The roadmap provided clarity on which endpoints and studies would best support future access and reimbursement.

Reduced Downstream Risk:

Early alignment helped the client make informed development decisions and prepare for future market access expectations.

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