Impact of The Use of Cost-Effectiveness Analysis (CEA) on Patient Access to Medicines: A Comparison of CEA vs. Non-CEA Markets

Approaches to health technology assessment (HTA) vary around the world; some markets apply cost-effectiveness analysis (CEA), including the use of quality-adjusted life-years, as a key determinant of their value assessments, while others prefer to exclude it.  

Aim 

This study explores the impact of the use of CEA on the level and timing of patient access to innovative medicines. 

In addition to this abstract, a presentation on the methodology and key findings can be downloaded from this web page. 

Methods 

Oncology and orphan drugs with approval of the first indication between January 2016 and December 2019, and any subsequent indications to up December 2021, were included in the analysis.  

For these drugs/ indications, relevant regulatory approvals from the US Food and Drug Administration, Australian Therapeutic Goods Administration, and Health Canada were also identified.  

The extent of access restrictions beyond the regulatory label and time to market access decisions were determined and compared across CEA markets (Australia, Canada, England, Scotland, Sweden) and non-CEA markets (France, Germany, Italy, Spain, US). 

The sample included 129 unique drug/indication approval combinations across 70 individual drugs with 861 market access decisions. 

Results 

The time to access decisions was highly variable across markets, with no difference between CEA and non-CEA markets.  

The number and proportion of unrestricted access decisions (i.e., aligned with the regulatory label) was greater in the non-CEA markets (341, 67.9% vs 123, 34.9%) while the number of restricted access decisions was greater in CEA markets (151, 42.9% vs 106, 20.8%).  

A wider range of restriction criteria was applied in CEA markets, and these markets appear to be particularly challenging for drugs intended for orphan conditions and for subsequent indications. 

Figure 1: Types of market access decision by HTA agency 

Conclusions 

Patient access to innovative oncology and rare medicines is more limited in CEA markets than in non-CEA markets. This highlights the importance of ensuring that any approach to value assessment is aligned with societal values and is fit for purpose. 

Download the key findings  

The presentation poster of this study, including the full methodology and key findings, can be downloaded from this page.   

First published 

2022-11, ISPOR Europe 2022 Conference, Vienna, Austria (November 2022)  

Value in Health, Volume 25, Issue 12S (December 2022) 

Authors(s) 

Katsikostas-Michopoulos G¹, Alleman C², Azough A³, Hickson S⁴, Griffin A³, Rollmann D⁴, Mckendrick J^5 
1PRMA Consulting Ltd, Athens, Greece, ²PRMA Consulting Ltd, Rotterdam, Netherlands, ³Johnson & Johnson, High Wycombe, UK, ⁴Janssen Pharmaceuticals, Raritan, NJ, USA, ⁵PRMA Consulting Ltd, Hampshire, HAM, UK