Current prices versus minimum costs of production for CFTR modulators

Area of Science:

• Pharmaceutical Science
• Health Economics
• Global Health Equity

Background:

• Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) modulators offer significant clinical benefits for cystic fibrosis (CF) patients.
• High drug prices limit access to these therapies, particularly in low-income countries.
• Limited data exists on the global accessibility of these life-changing CFTR modulator treatments.

Purpose of the Study:

• To estimate the minimum production costs of CFTR modulators.
• To assess the feasibility of expanding global access to CFTR modulator therapies.
• To compare estimated production costs with current market prices.

Main Methods:

• Utilized a validated algorithm to estimate minimum production costs per kilogram of active pharmaceutical ingredient.
• Identified cost-limiting key starting materials from published chemical synthesis routes.
• Compared estimated production costs with global import/export data and published list prices.

Main Results:

• Estimated annual production cost for elexacaftor/tezacaftor/ivacaftor is $5,676, over 90% less than the US list price.
• The total annual cost for all eligible CF patients globally at estimated production cost is $489 million, versus $31.2 billion at US list price.
• Synthetic pathways for elexacaftor/tezacaftor/ivacaftor are relatively straightforward, suggesting feasibility for generic production.

Conclusions:

• Generic production of elexacaftor/tezacaftor/ivacaftor is feasible at a fraction of current list prices.
• Existing pricing models create significant barriers to accessing optimal CF treatment, worsening health inequalities.
• Strategies like originator-issued voluntary licenses are needed to improve worldwide availability of this triple combination therapy.