OBJECTIVES: Medicinal products are assessed by the French HTA body (TC) to obtain their reimbursement. The clinical trials supporting MA application of targeted therapies are often non-comparative trials with a limited number of patients. These levels of evidence diverge from the methodological standards required by the TC doctrine. This research aims at identifying challenges of market access for targeted therapies in solid oncology, at assessing the management of uncertain data by the TC.

METHODS: All TC opinions of targeted therapies in solid oncology from 2018 to February 2024 were extracted. Therapies that submitted phases I, I/II or II clinical trials and for which the TC assessment considered that there was no or low added value (ASMR IV, V or SMR insufficient) were selected for a further analysis of transcripts of TC debates and levels of prices.

RESULTS: Among the 97 indications of targeted therapies extracted, 29 had submitted early phases clinical trials and demonstrated no progress or low added value. 15/29 had an insufficient SMR which is 16 times higher than the proportion of all indications assessed by the TC in 2022, whereas 12/29 had an ASMR V which is twice as large as the proportion of indications assessed in 2022. The median time for the TC evaluation was 156 days, 49 days longer than the median time of standard indications in 2022. The mean time for the prices negotiation was 505 days, 287 days longer than the mean time for all indications evaluated in 2022.

CONCLUSIONS: Early phase trials can lead to deteriorated assessment, lack of reimbursement or a complex negotiation of the prices of targeted therapies. Promoting access to these therapies is necessary to offer treatment alternatives to patients.