• Pediatric and adult forms of both diseases are similar to each other but not identical. |
• Both diseases are chronic without known curative treatment and require ongoing therapy. |
• Both diseases are associated with increased morbidity and mortality. |
• Both diseases have complex and multifactorial effects on the lives of patients and their families requiring a multidimensional assessment of clinical effect of treatments (e.g., pain, health-related QOL, social function, school function, etc.). |
• Prevalence: |
• JIA: 1 per 1,000 individuals (91). |
• Type 1 diabetes: ∼2 per 1,000 aged ≤20 years (4). |
• Relatively rare diseases in which market incentives are unlikely to lead pharmaceutical firms to focus on them. |
• Moderate polygenetic predisposition. |
• Etiologic agent(s) unknown. |
• Preclinical phase can extend over years, is poorly understood, and remains without therapeutic options. |
• In JIA, time is critical in the clinical phase—each month of delay of treatment onset during the first 12 months after the disease onset decreases the ability to reach clinical remission by 1.7 fold (92). Analysis of teplizumab response in recently diagnosed type 1 diabetes showed better preservation of C-peptide if treatment was initiated <6 weeks after the diagnosis (74). |
• Pediatric and adult forms of both diseases are similar to each other but not identical. |
• Both diseases are chronic without known curative treatment and require ongoing therapy. |
• Both diseases are associated with increased morbidity and mortality. |
• Both diseases have complex and multifactorial effects on the lives of patients and their families requiring a multidimensional assessment of clinical effect of treatments (e.g., pain, health-related QOL, social function, school function, etc.). |
• Prevalence: |
• JIA: 1 per 1,000 individuals (91). |
• Type 1 diabetes: ∼2 per 1,000 aged ≤20 years (4). |
• Relatively rare diseases in which market incentives are unlikely to lead pharmaceutical firms to focus on them. |
• Moderate polygenetic predisposition. |
• Etiologic agent(s) unknown. |
• Preclinical phase can extend over years, is poorly understood, and remains without therapeutic options. |
• In JIA, time is critical in the clinical phase—each month of delay of treatment onset during the first 12 months after the disease onset decreases the ability to reach clinical remission by 1.7 fold (92). Analysis of teplizumab response in recently diagnosed type 1 diabetes showed better preservation of C-peptide if treatment was initiated <6 weeks after the diagnosis (74). |