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Updated: Jan 14, 2026
07:04
Forskolin-induced Swelling in Intestinal Organoids: An In Vitro Assay for Assessing Drug Response in Cystic Fibrosis Patients
Published on: February 11, 2017
20.0K
Research and development spending versus revenues after approval for CFTR modulators
Jonathan Guo1, Grace Hennessy1, Benedict Young1
1School of Public Health, Faculty of Medicine, Imperial College London, United Kingdom.
Summary
High prices of Cystic Fibrosis Transmembrane conductance Regulator (CFTR) modulators hinder access, particularly in low-income countries. Despite substantial R&D costs, revenues quickly recouped these expenses, raising concerns about global health equity.
Area of Science:
- Pharmaceutical economics
- Drug pricing analysis
- Health equity research
Background:
- High costs of Cystic Fibrosis Transmembrane conductance Regulator (CFTR) modulators create access barriers for patients, especially in lower-income nations.
- Research and Development (R&D) expenses are frequently identified as a primary driver of drug pricing.
- The unique circumstances surrounding CFTR modulators allow for a direct comparison between pre-approval R&D investments and post-approval financial returns.
Purpose of the Study:
- To analyze the relationship between R&D expenditure and revenue for Cystic Fibrosis Transmembrane conductance Regulator (CFTR) modulators.
- To evaluate the financial sustainability and accessibility of CFTR modulator therapies.
- To inform future strategies for drug development and pricing in rare diseases.
Main Methods:
- Extracted R&D expenditure and revenue data for CFTR modulators from 2001 to 2024 using company SEC filings.
- Adjusted all financial values to 2024 inflation levels.
- Incorporated the cost of capital using a 10.5% discount rate, with sensitivity analyses at 7% and 14%.
Main Results:
- Total R&D spending before elexacaftor/tezacaftor/ivacaftor marketing approval reached $18.5 billion, with an estimated cost of capital of $24.8 billion.
- Cumulative revenues from 2012-2024 amounted to $63.8 billion, exceeding R&D expenditures by Q2 2020.
- Elexacaftor/tezacaftor/ivacaftor generates over $10.2 billion annually, positioning it among the highest-earning pharmaceutical products.
Conclusions:
- CFTR modulator R&D costs were recovered early in their commercial lifecycle due to high revenues.
- Current pricing strategies for these drugs jeopardize global health equity for individuals with CF and strain healthcare system finances.
- Urgent development of sustainable models is needed to balance equitable access with financial incentives for rare disease therapies, including emerging genetic treatments.
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