OBJECTIVES: Drug prices affect payer spending and patient access, yet price changes are not evaluated at a level that can inform payer or patient decisions. Treatment advances significantly improve the life expectancy of people with CF (PwCF) and many individuals are now living into their 50s and beyond. We examined pricing trends for Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Protein Modulator Therapies and Pancreatic enzymes, in the United States (US), United Kingdom (UK), France, Germany, Spain, and Italy (EU-5).

METHODS: A class-based drug price index was developed for CF treatments. We selected a chained-Laspeyres calculation approach by country and drug type from 2017-2022. We categorized treatments based on mechanism of action, into two distinct classes CFTR Modulators and pancreatic enzymes. The base period for comparison purposes was 2017. Prices and use of selected treatments were extracted from IQVIA MIDAS, the leading data source used to assess market conditions. Quantity was defined as the volume of total prescriptions dispensed each year. Price was defined as the weighted total spending for prescriptions.

RESULTS: All four CFTR modulators and six pancreatic enzyme products were included in the sample. We found year-to-year price fluctuations within each country. In the US, UK, and Spain, prices decreased for CFTR modulators (1%-36%) and increased for pancreatic enzymes (4%-60%). In Italy and France prices increased for CFTR modulators (6%-30%) and decreased for pancreatic enzymes (2%-6%). In Germany, prices increased for both CFTR modulators (35%) and pancreatic enzymes (3%). Limitations of this study include no direct adjustments for manufacturer rebates or product quality, and does not include adjunctive therapies CF.

CONCLUSIONS: Unadjusted drug prices for CF patients have experienced both price increases and decreases from 2017-2022 in the US and EU-5. By disaggregating the market basket into distinct drug classes within a single indication (CF), we can assess market dynamics and competition.