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PRO Strategy and Communication Framework to Support Regulatory and Investor Confidence

image of pharmacy community engagement activities
Client Profile

Mid-size Biopharmaceutical

Key Stakeholders

Health Outcomes, Regulatory Affairs, Investor Relations

Asset

Phase III

Disease Area

Oncology

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Client Profile

Mid-size Biopharmaceutical

Key Stakeholders

Health Outcomes, Regulatory Affairs, Investor Relations

Asset

Phase III

Disease Area

Oncology

Situation

A mid-size biopharmaceutical company was 18 months from launching a therapy for a rare oncologic disease with no prior approved treatments.

As Phase III progressed, the team needed clarity on how to select, position, and communicate patient-reported outcomes (PROs) to support regulatory interactions and external communications.

The client engaged Alkemi to develop an evidence-based PRO strategy and translate complex trial outcomes into a compelling narrative for regulators and investors.

Approach

1. Conducted a rapid review of validated PRO instruments, prior use cases, and regulatory precedent.

2. Assessed analogue therapies to inform endpoint selection and positioning strategy.

3. Developed structured guidance and documentation to support DCOA review and regulatory submissions.

4. Synthesized patient, provider, and outcomes data into a burden and value framework.

5. Created investor-ready communication materials translating PRO outcomes into clear, meaningful impact.

The focus throughout:

Elevate patient-reported outcomes into a strategic asset.

Deliverables

1
Rapid PRO evidence and use-case review.
2
Disease burden and value framework integrating clinical and PRO outcomes.
3
Publications development plan to elevate physician- and patient-reported outcomes.
4
Investor-ready PRO communication materials for press, congress, and milestone announcements.
5
6

Impact

“Alkemi helped us translate complex PRO data into a clear, compelling narrative that resonated with both regulators and investors.” - Senior Director, Regulatory Affairs

Regulatory Alignment:

Internal teams aligned on PRO instrument selection and positioning strategy for clinical development.

Stronger External Narrative:

Clear articulation of disease burden and meaningful patient impact strengthened investor communications.

Elevated Patient Voice:

PRO data were positioned as a core component of the therapy’s value story.

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