As part of the CRA Life Sciences series on leveraging RADAR, our orphan drug data repository, we analyzed the relationship between annual list prices achieved at launch and comparator arm types used in pivotal trials in France. The analysis covers 69 rare disease products assessed by the Transparency Commission between 2013 and 2024.
Key findings:
– Products with a natural history (NH) study used as the comparator arm achieved, on average, over twice the price granted for other products. However, this insight is based on a small sample size – only Strensiq, Kanuma, and Brineura – launched in France with NH comparative data. All three of those products were developed as first-to-market treatments for ultra-rare diseases – in these circumstances, the absence of placebo control arm may be justified.
– Approximately 40% of orphan drugs with a negotiated price in France launched with placebo-controlled data, another 40% with comparative data vs. SoC, and ~15% with single arm trial data. One notable observation is that the median price for each of those three categories is relatively similar.
– While payers generally prefer direct SoC comparisons, these results suggest payers do recognize the difficulty of providing such data in rare diseases, given that many orphan drugs are first-in-class products, and may be willing to recognize the value of novel treatments addressing high unmet needs.
Key takeaways:
– Orphan drugs with NH comparative arms, although being limited in number, have achieved relatively high prices – likely driven by the clinical benefits demonstrated and recognized by payers, as well as by the high unmet needs addressed, rather than by the comparator arm type itself.
– The choice of comparator arm among placebo, single arm, or SoC , appears to have limited impact on negotiated price at launch, suggesting that payers do recognize the difficulty of providing comparative data vs. SoC in rare diseases and may be willing to recognize the value of novel treatments that address high unmet medical needs.
