Value Story for Rare Disease Gene Therapy
Sector
Biotechnology
Company
Mid-Sized Biotech
Therapeutic Area
Rare Disease
Treatment Modality
Gene Therapy
Product Stage
Pre-Launch
Expertise
Scientific Communications
A leading biotech specializing in genetic medicines advanced a gene therapy for Duchenne muscular dystrophy into late-stage development, representing a potential first-in-class treatment in an area of profound unmet medical need. With a complex clinical profile, a high anticipated price point, and intense scrutiny from regulators, payers, and patient advocacy groups, the program faced significant challenges in establishing its value story ahead of launch.
Situation
A leading biotech specializing in genetic medicines advanced a gene therapy for Duchenne muscular dystrophy into late-stage development, representing a potential first-in-class treatment in an area of profound unmet medical need. With a complex clinical profile, a high anticipated price point, and intense scrutiny from regulators, payers, and patient advocacy groups, the program faced significant challenges in establishing its value story ahead of launch.
Situation
Approach
We worked with the team to develop market access and value communications that positioned the therapy credibly with diverse stakeholders. This included translating emerging clinical data into a clear and compelling narrative on functional outcomes, articulating the long-term health economic case for a one-time gene therapy, and creating payer-ready evidence frameworks that could withstand close examination. We also helped anticipate access hurdles by stress-testing scenarios around durability, patient eligibility, and real-world implementation.
We worked with the team to develop market access and value communications that positioned the therapy credibly with diverse stakeholders. This included translating emerging clinical data into a clear and compelling narrative on functional outcomes, articulating the long-term health economic case for a one-time gene therapy, and creating payer-ready evidence frameworks that could withstand close examination. We also helped anticipate access hurdles by stress-testing scenarios around durability, patient eligibility, and real-world implementation.
Approach
Results
This approach enabled early payer dialogues, strengthened alignment through advisory boards, and deepened engagement with patient advocacy groups. By shaping a unified value platform in advance of regulatory milestones, the therapy entered pre-launch with clearer differentiation, stronger stakeholder credibility, and an evidence-based foundation to support timely and sustainable access for patients with Duchenne.
This approach enabled early payer dialogues, strengthened alignment through advisory boards, and deepened engagement with patient advocacy groups. By shaping a unified value platform in advance of regulatory milestones, the therapy entered pre-launch with clearer differentiation, stronger stakeholder credibility, and an evidence-based foundation to support timely and sustainable access for patients with Duchenne.
Situation
A leading biotech specializing in genetic medicines advanced a gene therapy for Duchenne muscular dystrophy into late-stage development, representing a potential first-in-class treatment in an area of profound unmet medical need. With a complex clinical profile, a high anticipated price point, and intense scrutiny from regulators, payers, and patient advocacy groups, the program faced significant challenges in establishing its value story ahead of launch.
Approach
We worked with the team to develop market access and value communications that positioned the therapy credibly with diverse stakeholders. This included translating emerging clinical data into a clear and compelling narrative on functional outcomes, articulating the long-term health economic case for a one-time gene therapy, and creating payer-ready evidence frameworks that could withstand close examination. We also helped anticipate access hurdles by stress-testing scenarios around durability, patient eligibility, and real-world implementation.
Results
This approach enabled early payer dialogues, strengthened alignment through advisory boards, and deepened engagement with patient advocacy groups. By shaping a unified value platform in advance of regulatory milestones, the therapy entered pre-launch with clearer differentiation, stronger stakeholder credibility, and an evidence-based foundation to support timely and sustainable access for patients with Duchenne.
*Impact studies reflect the collective experience of Biography and its partners.
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Biology Century
We help life sciences teams cut through complexity and move from biology to belief—sharper, faster, leaner.
Let's Partner
Generating the
Biology Century
We help life sciences teams cut through complexity and move from biology to belief—sharper, faster, leaner.
Let's Partner
Generating the
Biology Century
We help life sciences teams cut through complexity and move from biology to belief—sharper, faster, leaner.