Key Takeaways
- Prevalence describes potential disease burden—not the commercially addressable market.
- Diagnosis, referral, eligibility, access and treatment initiation each reduce the opportunity.
- Concentrated expertise and referral leakage often matter more than raw patient density.
- Treating prevalence as the market can inflate forecasts, organization size and board expectations.
Rare disease market assessments frequently begin with prevalence.
They should not end there.
A population estimate can describe how many people may live with a condition. It does not describe how many patients can realistically receive therapy. For commercial strategy, that distinction is fundamental.
Leadership should separate total prevalence, diagnosed prevalence, clinically eligible patients, addressable patients, accessible patients and treated patients. Each transition can materially reduce the opportunity—and each requires different commercial decisions.
Executive opinion paper
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About Joseph McCoy
Joseph McCoy is Founder and Principal Consultant of PharmaKonsult and an executive commercial strategy advisor with more than three decades of pharmaceutical and biotechnology commercial leadership experience across neuroscience, rare disease, endocrinology, institutional healthcare and specialty pharmaceuticals.
