Making Every Patient Count
a Business Imperative
When a rare-disease company built its future around fewer than 5,000 patients — and Andy Jablonski became the face of that mission
Drug companies often say that every patient counts. In the world of orphan diseases, entire business plans are built around that idea.
That is why Andrew E. Jablonski — then a 26-year-old from Lincoln, Nebraska, born missing most of his intestines and living with Short Bowel Syndrome — was a guest of honor at NPS Pharmaceuticals, a small New Jersey company preparing to launch Gattex, its only approved drug.
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Orphan focus
Rare-disease business model
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< 5,000
Patients the drug aimed to reach
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NYT feature
January 2013 coverage
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Mr. Jablonski was central to the company’s effort to connect with the small community of people living with Short Bowel Syndrome — a group small enough that traditional commercial approaches often overlook them, yet large enough that each person’s experience shapes access, education, and trust.
The story captured a turning point: rare-disease drug development was no longer only a scientific challenge. It was also a relationship challenge — with patients, families, and advocates who live the condition every day.
The New York Times · January 31, 2013
“Orphan Drugs for Rare Diseases Gain Popularity with Pharmaceutical Companies” — featuring Andy Jablonski, Short Bowel Syndrome, and the business of rare disease.
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Patient voice in industry Rare-disease development works best when people living with the condition help shape education, access, and community trust. |
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Foundation roots This era helped define SBSF’s ongoing work: patient-led advocacy, education, and connection for the SBS and intestinal failure community. |
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This page preserves a historical media moment from 2013. Product names, company ownership, and availability can change over time. For current treatment decisions, always work with your clinical care team.