CRA Life Sciences continues its series on leveraging RADAR, its orphan drug data repository, with a focus on PMA trends between ASMR outcomes and average annual treatment costs. The analysis shows a clear correlation between the ASMR rating and the level of price achieved. Notably, ASMR1 and ASMR2 products tend to secure higher negotiated prices, though it’s important to note that these categories represent only a small number of products (one and three, respectively).
The average annual treatment costs for ASMR1 to ASMR4 are high, at €285,000, €200,000, and €100,000, respectively. These figures are influenced by a few outliers with high prices in smaller indications. For instance, ASMR3 drugs like Vimizim (€1,128,400 for Mucopolysaccharidosis type IVA), Bilvay (€651,569 for PFIC), Brineura (€525,815 for CLN2), and Blincyto (€48,669 for 2L pediatric Ph-ALL) contribute to these costs. Similarly, ASMR4 includes drugs like Procysbi (€405,880 for nephropathic cystinosis), Waylivra (€409,500 for hyperlipoproteinemia), and Xospata (€328,083 for 3L AML).
The average annual treatment costs decrease by approximately 50% between ASMR1-2, ASMR3, ASMR4, and ASMR5. Mixed ASMR products show a wide range in treatment costs, from €460,000 for Kanuma (ASMR3,5) to €11,000 for Adempas (ASMR4,5). This range reflects more complex negotiations, as demonstrated by the longer negotiation durations (refer to a previous infographic for details).
Finally, no definitive conclusions can be drawn regarding the impact of the Accord Cadre—an agreement between CEPS payers and industry on regulating price negotiations (link)—on the relationship between price and ASMR outcomes, largely due to the small number of products approved since its implementation.
Key takeaways:
- There is a correlation between negotiated list price and ASMR rating
- Average annual treatment costs for ASMR1-2 reach close to €300,000
- Average annual treatment costs for ASMR3s are 50% lower at €200,000
- Average annual treatments costs for ASMR4s drop another 50% to €100,000
- ASMR5s average annual treatment costs are the lowest, averaging just about €50,000
- Average annual treatment costs of mixed ASMRs vary highly between €11,000 and €460,000
