As CRA Life Sciences continues the series on leveraging RADAR, CRA’s orphan drug data repository, they focus on PMA trends from reimbursement (CT outcome) to price publication.
Eleven products are excluded from the graph due to their time to price publication exceeding 800 days, and four post-ATU phase products were also omitted from the analysis. Generally, price negotiation durations are consistent across ASMR outcomes, except for Orphacol, the sole product achieving ASMR 1. Many ASMR 1, 2, and 3 products had early access through the ATU (now AAP/AAC) system, which ensures patient access during negotiations and reduces urgency, explaining why ASMR 2s have similar negotiation times to ASMR 3s or 4s. ASMR 5s, however, have the shortest time to price publication, experiencing roughly 50% less delay due to clearer CEPS price discount expectations, though this sometimes leads to MNFs withdrawing from reimbursement (n=3). Mixed ASMR prices take the longest to negotiate, likely due to their complexity. The Accord Cadre (agreement between CEPS payers and industry on regulation of price negotiations (link)) showed no significant impact on negotiation times, with similar publication times observed before and after 2020.
Key takeaways:
- 5 products are not represented on the graph as time to CT outcome was over 800 days. Whilst Ravicti, Zalmoxis and Jorveza were under the 3-year timeframe, Scenesse and Coagadex took over 5 years to obtain reimbursement
- Only 1 product, Orphacol, has achieved an ASMR 1 since 2013, and none have been granted since 2014. ASMR1 for Orphacol was granted within 160 days of EMA approval, at first submission
- 4 products received ASMR 2, namely Strensiq, Luxturna, Givlaari and Kaftrio. All except Strensiq had secured early access through the ATU prior to receiving reimbursement
- 32 products overall received an ASMR 3 including 5 as part of a mixed ASMR, an average of 3 per year. Amongst these, 9 received reimbursement restrictions (mixed SMR), including 2 with mixed ASMRs
