OBJECTIVES: Close alignment of evidentiary requirements and wider use of available data between HTA agencies and regulatory authorities could facilitate timely market access by optimising trial design and reducing the number of studies undertaken. This is particularly important in rare conditions with high unmet need, like haematological malignancies (HM), where patient sample sizes are challenging to achieve along with the emergence of adapted regulatory approval pathways which appraise less comprehensive and mature evidence. Aim: Ascertain and analyse the alignment of the market access clinical evidence base assessed by regulators and HTA agencies for HM technologies

METHODS: Clinical evidence data sources (clinical trials, observational studies, national statistics, registry data) were extracted from EMA’s European public assessment reports (EPAR) and the 9 HTA agencies assessment reports for 11 HM products (n=60).

RESULTS: Aggregated HTA and regulator clinical evidence showed overlap/commonality at 58% with variance by HTA agency (mean range 28-67%) and product (mean range 25-92%). Individual assessments, 1/4 reached 100% congruence and 5% were 100% divergent. Primary EPAR efficacy study and pivotal trial for HTA effectiveness assessment were congruent in 92% of cases. Real world evidence was used in 15% (11/71) of reports with alignment in 2 cases. None overlapping evidence instances and use of primary outcomes were explored. Designated orphan products showed no difference in alignment of the evidence base compared with non-orphan products. Products with accelerated access conditional EMA approval had high rates of alignment, lower numbers of additional studies, earlier phase trials but a higher rate of negative/restrict decisions.

CONCLUSIONS: Results indicate a closer evidence alignment than may be expected of the primary evidence source. This suggest caution with regard to narrowing the evidence gap as seen in expediated assessments if fewer additional evidence sources and earlier phase studies are used to meet HTA requirements leading to negative decisions.