The Economic and Health Policy Impact of a Uniform National Formulary: Equity and Policy Considerations in the U.S. Using Cystic Fibrosis Treatment with Trikafta as a Cross-Comparator

Abstract

Background: Access to essential medications is critical for an equitable healthcare system, yet the absence of a Uniform National Formulary (UNF) in the U.S. has led to disparities in drug affordability and access. Unlike New Zealand’s Pharmac and Australia’s PBS, which centralize price negotiations and costeffectiveness evaluations, the U.S. relies on a fragmented system controlled by private insurers, PBMs, and Medicare Part D, resulting in inconsistent drug coverage and financial barriers. Using Trikafta (Elexacaftor/Tezacaftor/Ivacaftor) for Cystic Fibrosis (CF) as a case study, we explored how adopting elements of Pharmac and PBS could improve affordability. While the Inflation Reduction Act (IRA) of 2022 introduced Medicare drug price negotiations, Trikafta remains excluded, underscoring the need for policy reform.

Methods: We conducted a comparative policy analysis to assess the economic and health policy implications of a Uniform National Formulary (UNF) in the U.S. It examines New Zealand’s Pharmac and Australia’s PBS as case studies, evaluates the fragmented U.S. formulary system, and compares Cystic Fibrosis (CF) treatment accessibility, focusing on Trikafta pricing and affordability. The analysis assesses cost-sharing structures, formulary decision-making, and regulatory frameworks across the three systems. Additionally, it explores the political and equity considerations of a U.S. national formulary, analyzing policy reports, government data, and peer-reviewed research to provide evidence-based recommendations for improving drug affordability and access.

Results: We examined the economic and policy implications of a Uniform National Formulary (UNF) by comparing Trikafta access for Cystic Fibrosis (CF) patients across New Zealand (Pharmac), Australia (PBS), and the U.S. (Medicare Part D). Pharmac fully subsidizes Trikafta ($0–$5 per prescription), PBS offers fixed co-pays ($31.60, $7.70 concession), while U.S. patients pay $67–$321 under Medicare Part D. Unlike Pharmac and PBS, Medicare Part D does not negotiate drug prices, leaving CF patients with higher costs and limited access. Trikafta is not included in the IRA 2022 drug negotiations, emphasizing the need for policy reform to improve affordability and equity.

Conclusions: Disparities in drug pricing, access, and government involvement across New Zealand, Australia, and the U.S. highlight challenges in implementing a Uniform National Formulary (UNF). Pharmac and PBS negotiate lower drug prices, ensuring affordability, while the U.S. market-driven system leads to higher costs and fragmented access. The Inflation Reduction Act (IRA) of 2022 introduced Medicare drug price negotiations, but Trikafta remains excluded, underscoring the need for reform. Expanding Medicare negotiations, integrating a structured formulary, and reducing access barriers could improve affordability. Lessons from Australia and New Zealand suggest that incremental Medicare reforms could pave the way for a national formulary.