Table 1.
Sampled rare diseases and medicines
| Rare disease | Prevalence | Therapeutic regimen | Health expenditure per year (1,000 yuan) | Health expenditures as a share of per capita GDP |
|---|---|---|---|---|
| Duchenne Muscular Dystrophy (DMD) | 5/100000 | Methylprednisolone Tablet, 0.6 mg/Kg/day | 1.20 | 0–1 |
| Alport Syndrome (AS) | 2/100000 | ACEI combined with ARB | 7.50 | 0–1 |
| Tuberous Sclerosis Complex (TSC) | 8.8/100000 | Sirolimus Tablet, 1 mg bid | 40 | 0–1 |
| Amyotrophic Lateral Sclerosis (ALS) | 5/100000 | Riluzole Tablet, 50 mg bid | 55 | 1–3 |
| Idiopathic Pulmonary Fibrosis (IPF) | 16.7/100000 | Pirfenidone Tablet, 200–400 mg tid | 86 | 1–3 |
| Pulmonary Arterial Hypertension (PAH) | 5/100000 | Bosentan Tablet, 125 mg bid | 240 | >3 |
| Gaucher Disease (GD) | 1/100000 | Imiglucerase for Injection, 60 U/kg, fortnightly | 2000 | >3 |