White Paper
Defining the Disease
The most successful rare disease launches aren't won with brand campaigns — they're won by teaching physicians what the disease actually is before ever mentioning the drug. Drawing on 45 years in biopharma, this white paper lays out a four-pillar framework for science-first commercial strategy and traces it through three landmark rare disease launches: Spinraza/Zolgensma for SMA, Cerezyme for Gaucher disease, and Vyndaqel/Onpattro for hereditary ATTR amyloidosis.
What’s Inside
- The four pillars of disease-defining strategy: biological narrative, patient phenotype, therapeutic rationale, and community building
- Three landmark case studies — SMA, Gaucher disease, and hereditary ATTR amyloidosis — showing the framework in action
- A phased implementation roadmap from pre-approval disease characterization through post-launch infrastructure
- Why disease-first strategy outperforms brand-first on defensibility, payer credibility, and physician trust
