OBJECTIVES:

From 2025, select pharmaceuticals will be mandated to undergo a joint clinical assessment (JCA) at the European level. In preparation, EUnetHTA21 (2021-2023) is developing methodological guidelines for JCAs. This research evaluates indirect treatment comparison (ITC) acceptance in JCAs and assesses how the corresponding ITCs faired during national assessments.

METHODS:

Publicly available information on EUnetHTA JCAs from JA3 (2016-2021) for individual drugs were identified alongside the associated appraisals by national HTAs from Germany (G-BA), France (HAS), Sweden (TLV).

RESULTS:

Twenty JCAs were identified, of which 16 achieved marketing authorisation (MA). 10/16 JCAs contained an ITC. The ITC methodology varied between submissions: 4/10 Bucher; 1/10 Matching Adjusted Indirect Comparison (MAIC); 5/10 Bayesian, and 1/10 propensity score weighting (PSW). Out of the ten JCA submissions containing an ITC, only two ITCs were considered appropriate for use, using Bucher and Bayesian methodology.

Of the ten products with a JCA which contained an ITC: eight were submitted to the GBA, with only 1/8 containing an ITC (unknown method); which was not accepted. Eight were submitted to HAS with 3/8 containing an ITC (MAIC, Bayesian, PSW), one achieved ASMR≤3; however the ITC (PSW) was not accepted. Six were submitted to the TLV with 2/6 containing an ITC (MAIC) of which one ITC was accepted. Critiques of ITCs across both JCA and national assessments were generally driven by the heterogeneity of the included studies.

CONCLUSIONS:

Despite the majority of JCA submissions containing ITCs, acceptance of the methodology during evaluation was low. Furthermore, in the corresponding national submissions, ITCs were often excluded, and acceptance was also unlikely. With the new HTA regulation, there is an opportunity to increase uptake of ITCs at European and national level; however there is a risk that without strong direction from the EC, the use of ITCs may delay access to new pharmaceuticals in Europe.