TBD: Impact of expediting regulatory measures on market access decisions and availability of medicines in Belgium
KU Leuven Belgium
This study aims to i) assess the impact of regulatory measures on the rate of and time to availability of centrally authorised medicines in Belgium and ii) evaluate the impact of regulatory measures on national market access procedures and decisions.
Included medicines were granted a European centralised marketing authorization between 2015 and 2020, excluding generic products/biosimilars. The correlation between regulatory measures and i) the time to and rate of availability in Belgium and ii) market access decisions were statistically tested.
A total of 324 medicines were included of which the time to and rate of availability were calculated and the impact of regulatory measures assessed. Included regulatory measures were installed at the European level and relied on the unmet medical need concept as eligibility criterium. Analysis resulted in 79 (24.4%) medicines with orphan designation, 11 (3.4%) included in the PRIME scheme, 25 (7.7%) accelerated approved, 22 (6.8%) conditionally authorized, and 11 (3.4%) authorized under exceptional circumstance, with some medicines making use of several of these regulatory measures. Availability was defined as medicines that are nationally authorised and available for patients, despite reimbursement. The availability rate was 49.1%. of which 98.9% was reimbursed available and 11.1% were only limited available, meaning that those medicines were not reimbursed but available via a national early access scheme (24.2%) or at the patients’ personal expense (75.8%). Survival analysis was performed with a survival time defined as the interval between marketing authorization application and availability and predictors the identified regulatory measures. No significant results were obtained except for medicines included in the PRIME scheme (p=.003), where medicines included in the PRIME scheme were found to be more likely to obtain availability in the identified time period. Fisher’s Exact test showed a significant association between (i) the inclusion in a managed entry agreement at the national level and accelerated approval (p<.001), (ii) the inclusion in a medical need program and orphan designation (p=.035), and (iii) the inclusion in the PRIME scheme (p=.044), and accelerated approval (p=.010) of the medicine at central regulatory level.
Results showed that most European regulatory measures to expedite authorisation for medicines addressing high unmet medical needs are not associated with higher availability rates nor shorter times to availability in Belgium. Only medicines that were included in the EMA PRIME scheme for priority medicines were found more likely to get early availability at the national level. A possible explanation for the limited impact of regulatory measures on the rate of and time to availability of medicines is the diverging understanding of the unmet medical need concept between the EMA and the national payers/regulators, which is reflected in the limited association between the set of medicines that was applied a regulatory measure and those applied a market access measure. Inclusion in expediting regulatory pathways often implicates a higher acceptance of uncertainties which can be justified considering the high need for those medicines. However, if the national payer/regulator does not agree upon the relative pressing nature of the respective need, the remaining uncertainties can be unacceptable impacting pricing and reimbursement decisions. Better alignment between the EMA and the national HTA body/payer on eligibility criteria for medicines to be included in expediting pathways and the underlying required evidence to support regulatory and HTA/payer decision-making could ensure that medicines for which there is an important unmet medical need reach the patients earlier, not only in theory but also in practice.