Highlights
- •CFTR modulators are the best treatment for CF but are priced over $250,000 per year
- •Costs of production were estimated using an established and previously validated model
- •Costs of production for CFTR modulators could be 90% lower than current prices
- •The high prices of CFTR modulators perpetuate international disparities in CF care
- •Generic production of CFTR modulators is already feasible and could form a solution
Abstract
Background
Methods
Results
Conclusions
Keywords
1. Introduction
- Guo J
- Garratt A
- Hill A.
FAQs — CF Buyers Club n.d. https://www.cfbuyersclub.org/faqs (accessed April 30, 2021).
- da Silva Filho LVRF
- Zampoli M
- Cohen-Cymberknoh M
- Kabra SK.
2. Methods
2.1 Minimum costs of production
瀚闻资讯 n.d. http://www.sinoimex.com/ (accessed October 1, 2021).
PharmaCompass – Grow Your Pharma Business Digitally n.d. https://www.pharmacompass.com/ (accessed March 12, 2022).
- Fortunak J
- Ngozwana S
- Gebre-Mariam T
- Ellison T
- Watts P
- Emeje M
- et al.
2.2 Sensitivity Analysis
2.3 Current list prices
3. Results
3.1 Minimum costs of production
Export Data and Price of ivacaftor | Zauba n.d. https://www.zauba.com/export-ivacaftor-hs-code.html (accessed November 20, 2021).
List of Drug Master Files (DMFs) | FDA n.d. https://www.fda.gov/drugs/drug-master-files-dmfs/list-drug-master-files-dmfs (accessed November 20, 2021).
3.1.1 Lumacaftor/Ivacaftor

3.1.2 Tezacaftor/Ivacaftor
3.1.3 Elexacaftor/Tezacaftor/Ivacaftor
3.2 Current list prices for CFTR modulators
Drug, Duration and Dose | Highest List Price (per annum) | Lowest List Price (per annum) | Estimated Production Cost (per annum) |
---|---|---|---|
PO lumacaftor/ivacaftor 400/250mg PO BD | $284,800 US (Commercial) | $79,900 Argentina | $9,700 |
$15,000 UK CF Buyers Club | |||
PO tezacaftor/ivacaftor 100/150mg OD + ivacaftor 150mg OD | $305,000 US (Commercial) | $94,300 Argentina | $3,900 |
$15,000 UK CF Buyers Club | |||
PO elexacaftor/tezacaftor/ivacaftor 200/100/150mg OD + ivacaftor 150mg OD | $325,300 US (Commercial) | $102,300 Argentina | $5,700 |

4. Discussion
FAQs — CF Buyers Club n.d. https://www.cfbuyersclub.org/faqs (accessed April 30, 2021).
A generic drug from Argentina offers cystic fibrosis families hope | New Scientist n.d. https://www.newscientist.com/article/2205916-a-generic-drug-from-argentina-offers-cystic-fibrosis-families-hope/ (accessed November 27, 2021).
- Morgan SG
- Vogler S
- Wagner AK.
- Barber MJ
- Gotham D
- Khwairakpam G
- Hill A.
Vertex Reports First-Quarter 2021 Financial Results 2021. https://investors.vrtx.com/node/28361/pdf (accessed May 2, 2021).
- Guo J
- Garratt A
- Hill A.
- Veit G
- Roldan A
- Hancock MA
- da Fonte DF
- Xu H
- Hussein M
- et al.
Shelton B, Margolis P. The Association of Socioeconomic Status with Outcomes in Cystic Fibrosis Patients in the United States PEDSnet and PCORnet View project Collaborative Chronic Care Network View project n.d. doi:10.1164/ajrccm.163.6.9912100.
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- Zampoli Marco
- et al.
5. Conclusion
Funding
Data sharing statement
CRediT authorship contribution statement
Declaration of Competing Interest
Acknowledgements
Appendix. Supplementary materials
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