Strategic launch in a rare hematologic disease

The challenge

A biopharmaceutical company preparing to launch a novel treatment in a rare hematologic disease faced the challenge of entering a market with an entrenched incumbent rooted in industry-standard efficacy measures. Despite the therapy demonstrating superior efficacy while maintaining safety standards, challenges remained around the route of administration, dosing, and treatment frequency.

The client sought support establishing strategic direction pre-launch and effectively communicating the therapy’s value to physicians and other priority stakeholders. The organization lacked clarity on how to achieve its aspiration of becoming the new first-line standard of care at launch.

The Acumetis approach

Acumetis partnered with the client to support strategic direction pre-launch through an integrated Medical Affairs approach focused on scientific positioning, stakeholder engagement, and launch readiness.

Scientific narrative and positioning strategy

Our team conducted a situation analysis to align the client’s offering with market needs and establish positioning based on the therapy’s clinical value versus the standard of care. Acumetis then developed a strategic scientific platform and differentiated narrative designed to support the client’s updated launch goals and desired positioning within the rare hematologic disease landscape.

Cross-functional launch alignment

Acumetis helped reset launch goals and facilitated collaboration across Medical Affairs, Commercial, and Market Access stakeholders to develop a cross-functional strategy for cohesive communication, congress activities, and internal training. This integrated approach helped establish consistency across launch messaging and stakeholder engagement strategies.

Rare disease stakeholder insights

To help refine the therapy’s positioning and communication strategy, Acumetis engaged with key opinion leaders (KOLs) to gather insights on treatment dynamics, unmet needs, and stakeholder perspectives within the rare disease landscape. These insights directly informed the scientific narrative and broader launch activities.

Launch readiness support

The team also supported broader launch readiness efforts by helping organize strategic planning activities, refine engagement approaches, and develop materials designed to support internal alignment and external scientific communication.

The outcome

The engagement helped position the client’s therapy against the standard of care within the evolving rare hematologic disease landscape and supported a more coordinated and strategically aligned launch approach. By connecting scientific evidence, stakeholder insight, and cross-functional strategy, the client strengthened its ability to communicate the therapy’s clinical value and support launch execution within a highly specialized market.

Why it worked

• Aligned the client’s offering with key stakeholder insights to strengthen communication strategy and support targeted data generation
• Reframed expectations around efficacy measures to more effectively position the therapy’s clinical data narrative
• Initiated publications focused on outcome measures to reinforce the therapy’s clinical story and novel mechanism of action
• Clarified the therapy’s value for uncontrolled patients on standard of care with no alternative treatment options while shaping positioning considerations for first-line use

Acumetis medical affairs team

Acumetis Medical Affairs partners with biopharma teams to know the science and own the narrative, turning scientific strategy, evidence generation, and medical communications into credible stories that shape decisions and build confidence. Our teams anticipate evidence needs early and work across functions to ensure clinical endpoints, evidence plans, and communications are rigorous, relevant, and aligned with regulatory expectations.

To learn how Acumetis can support your medical affairs strategy and planning, contact us to start the conversation.