OBJECTIVES

Innovative drugs for rare diseases are increasingly expensive. Previous publications assess annual cost and group together gene/acute-therapies and chronic treatments, which may not provide a complete picture. This study investigates costs of gene/acute therapies versus chronic treatments for rare diseases, on an annual and lifetime basis to provide an accurate comparison.

METHODS

A literature review of PubMed and grey databases including terms: high cost, most expensive treatment, therapy, annual, lifetime was conducted to identify the most expensive drugs in the US on an annual and lifetime cost basis. 25 products were identified, and drug costs calculated using dosing, USD WAC prices, age of disease onset and life-expectancy to arrive at the top 20 most costly treatments from an annual and lifetime perspective.

RESULTS

On an annual basis, the top 3 most expensive drugs in the US are Zolgensma, Luxterna and Ravicti. However, considering the lifetime costs of these treatments, places them 14^th, 17^th and 5^th respectively. In addition to Zolgensma and Luxterna, 4 further gene/acute therapies (Folotyn, Chenodal, Kymriah and Yescarta) all feature in the top 20 on an annual basis but fall at the bottom of the list based on lifetime cost.

Furthermore, within the product basket, the top 3 most expensive drugs on a lifetime basis are Cinryze, Juxtapid and Takhzyro which place only 9^th, 8^th and 10^th respectively from an annual treatment perspective.

The average first year costs of $0.8M for the 6 gene/acute treatments is much higher versus $0.5M for the 14 chronic treatments. However, the most expensive lifetime cost for a chronic treatment exceeds $18.0M with an average of $9.3M compared to a lifetime average of only $0.9M for gene/acute treatments.

CONCLUSIONS

Consideration of the lifetime costs and overall budget impact of gene/acute therapies rather than focusing on annual costs will help manufacturers address potential payer affordability concerns.