💥PICO-Exercise for Accelerated Assessment!
 

📅Etranacogene dezaparvovec (Hemgenix) was approved for the treatment of Haemophilia B in adult patients without a history of Factor IX inhibitors. Hemgenix started the MAA process on March 24th, 2022, with an accelerated assessment, even though it reverted back to a standard procedure later in the process. Marketing Authorization was issued on February 20th, 2023.
 

In addition, the final label differed from the one which had been originally submitted by the applicant (https://www.ema.europa.eu/en/documents/assessment-report/hemgenix-epar-public-assessment-report_en.pdf).

 

PICO exercise for Hemgenix resulted in 7️⃣ PICOs:

•Of these, 5️⃣ were PICOs for the whole population, which differed in terms of the Comparator and the list of Outcomes.

•Another 2️⃣ PICOs were defined for subpopulations, differing by treatment strategy (prophylactic vs event driven, both stating the same Comparator).

•4️⃣out of the 5️⃣PICOs for the full population required different subgroup analyses by disease severity, anti-AAV5 antibodies, and annualised bleeding rate.

 

❗️For all PICOs, the intervention was defined as “Etranacogene dezaparvovec according to SmPC”.
 

Hemgenix has been assessed for this indication by G-BA on Oct 19th, 2023, under the orphan drug privilege. The G-BA did not define a Comparator in this case. However, Eftrenonacog alfa (Alprolix) has been assessed on Feb 1st , 2024, outside the orphan drug privilege. The Comparator was defined as “recombinant or human plasma-derived coagulation factor IX preparations”. Note that the PICO exercise for Hemgenix does not result in this PICO.

 

❓️It is not clear how this scope of assessment could have been used for Germany outside of the orphan drug privilege.

 

💭Considering the importance of PICOs for EU HTA, we see a lot to learn from the PICO Exercises. Follow our thoughts in this new series Reflections On Second Wave Of PICO Exercises to Get Ready For EU HTA.

 

📖 More on how to prepare for EU HTA in our book: EU HTA 101 (updated in October 2024) https://lnkd.in/eAsxt7NZ