Situation:
A young biotechnology company had developed a promising gene therapy for a rare, debilitating genetic disease. However, they lacked the experience and resources to navigate the complex US market for rare diseases.
Challenges:
Limited understanding of the specific patient population and disease burden.
Difficulty identifying relevant patient advocacy groups and key opinion leaders (KOLs) with expertise in the rare disease area.
Unclear understanding of existing treatment landscape and potential competitive threats from other gene therapies or investigational drugs.
Impact:
Conducted a comprehensive market research analysis, including patient surveys, healthcare provider interviews, and a review of scientific literature.
Identified key patient advocacy groups and established relationships to gain insights into patient needs and priorities.
Developed a targeted KOL identification strategy using disease-specific databases and conferences, connecting the company with relevant experts for early feedback on the gene therapy's potential impact.
Supported the development of a compelling medical value proposition focusing on the potential for improved quality of life and reduced disease burden for patients with this rare condition.
Results:
In-depth market research provided valuable insights and targeted KOL engagement fostered understanding of patient needs. This led to the development of a compelling value proposition for the novel gene therapy.
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