Microcosting itemized list showing the manufacturing costs for gene therapy
| Microcosting list of gene therapy manufacturing costs for 73 patients* . | ||||
|---|---|---|---|---|
| Task . | Total labor cost . | Supplies . | Other expenses . | Total direct cost in US$ . |
| Viral production cell licensing fee | $15 166 | $74 000 | $20 000 | $109 166 |
| Quality assurance master batch production records and associated documentation | $32 921 | $300 | $0 | $33 221 |
| Drug substance cGMP manufacturing Quality control Quality assurance | $394 698 $75 037 $81 371 | $2 297 770 $57 015 $14 000 | $0 $339 500 $0 | $2 692 467 $471 552 $68 977 |
| Drug product | ||||
| cGMP manufacturing | $17 198 | $18 750 | $0 | $95 771 |
| Quality control | $8628 | $7950 | $9500 | $26 078 |
| Quality assurance | $13 395 | $50 | $0 | $13 445 |
| 36-Mo stability study | $37 271 | $37 100 | $21 000 | $95 371 |
| Total direct costs | $675 685 | $2 507 335 | $390 000 | $3 573 019 |
| Microcosting list of gene therapy manufacturing costs for 73 patients* . | ||||
|---|---|---|---|---|
| Task . | Total labor cost . | Supplies . | Other expenses . | Total direct cost in US$ . |
| Viral production cell licensing fee | $15 166 | $74 000 | $20 000 | $109 166 |
| Quality assurance master batch production records and associated documentation | $32 921 | $300 | $0 | $33 221 |
| Drug substance cGMP manufacturing Quality control Quality assurance | $394 698 $75 037 $81 371 | $2 297 770 $57 015 $14 000 | $0 $339 500 $0 | $2 692 467 $471 552 $68 977 |
| Drug product | ||||
| cGMP manufacturing | $17 198 | $18 750 | $0 | $95 771 |
| Quality control | $8628 | $7950 | $9500 | $26 078 |
| Quality assurance | $13 395 | $50 | $0 | $13 445 |
| 36-Mo stability study | $37 271 | $37 100 | $21 000 | $95 371 |
| Total direct costs | $675 685 | $2 507 335 | $390 000 | $3 573 019 |
| Gene therapy manufacturing cost per dose calculations and assumptions . | ||||
|---|---|---|---|---|
| Variable . | Calculations . | Value . | ||
| Batch size (L) | None | 200 | ||
| Yield per liter of bioreactor volume | None | 2.50E+13 | ||
| Total no. of batches | None | 10 | ||
| Total vg titer from total no. of batches | Batch size (L) × yield per liter bioreactor volume × total no. of batches | 5.00E+16 | ||
| vg dose/kg | None | 8.00E+12 | ||
| Patient weight at 18 y per kg | None | 75.6 | ||
| vg per dose based on patient weight | vg dose/kg × patient weight (kg) | 6.05E+14 | ||
| Total no. of patient doses | ([Total no. of vials – vials needed for testing + 10% extra] × total vg/vial)/vg per dose based on patient weight | 73.042 | ||
| Fill volume per vial (mL) | None | 5.3 | ||
| Concentration of drug product (vg/mL) | None | 3.00E+12 | ||
| Total vg/vial | Fill volume per vial (mL) × concentration of drug product (vg/mL) | 1.59E+13 | ||
| Total no. of vials | Total vg from total no. of batches/total vg/vial | 3144.65 | ||
| Vials needed for testing | None | 333 | ||
| Vials needed for testing + 10% extra | None | 366.3 | ||
| Direct cost per dose ($) | Total direct cost/total no. of patient doses | $48 917 | ||
| Cost per dose ($) | Direct cost per dose ($) × 1.58 | $77 289 | ||
| Gene therapy manufacturing cost per dose calculations and assumptions . | ||||
|---|---|---|---|---|
| Variable . | Calculations . | Value . | ||
| Batch size (L) | None | 200 | ||
| Yield per liter of bioreactor volume | None | 2.50E+13 | ||
| Total no. of batches | None | 10 | ||
| Total vg titer from total no. of batches | Batch size (L) × yield per liter bioreactor volume × total no. of batches | 5.00E+16 | ||
| vg dose/kg | None | 8.00E+12 | ||
| Patient weight at 18 y per kg | None | 75.6 | ||
| vg per dose based on patient weight | vg dose/kg × patient weight (kg) | 6.05E+14 | ||
| Total no. of patient doses | ([Total no. of vials – vials needed for testing + 10% extra] × total vg/vial)/vg per dose based on patient weight | 73.042 | ||
| Fill volume per vial (mL) | None | 5.3 | ||
| Concentration of drug product (vg/mL) | None | 3.00E+12 | ||
| Total vg/vial | Fill volume per vial (mL) × concentration of drug product (vg/mL) | 1.59E+13 | ||
| Total no. of vials | Total vg from total no. of batches/total vg/vial | 3144.65 | ||
| Vials needed for testing | None | 333 | ||
| Vials needed for testing + 10% extra | None | 366.3 | ||
| Direct cost per dose ($) | Total direct cost/total no. of patient doses | $48 917 | ||
| Cost per dose ($) | Direct cost per dose ($) × 1.58 | $77 289 | ||
vg, viral genomes.
The number of patients has been rounded down from 73.042. Calculations above show how the number was derived.