OBJECTIVES:

To systematically evaluate all PRIME (PRIority MEdicines) designations to date and how these have translated into reimbursement and patient access.

INTRODUCTION:

In March 2016, the European Medicines Agency (EMA) launched the PRIME (PRIority MEdicines) scheme. PRIME enhances early dialogue and regulatory support for putative medicinal products that address severe unmet treatment needs. PRIME also enables EMA accelerated assessment (which reduces the assessment timeframe from 210 days to 150 days).

METHODS:

The EMA website was screened and medicines with PRIME designation identified (as of 06-MAR-2019). For any with EC marketing authorisation (MA), the corresponding HTA assessments by NICE, SMC, G-BA and HAS were screened and their outcomes extracted.

RESULTS:

Since 2016, fifty therapies have received PRIME designations (2016: 15; 2017: 19; 2018: 14; 2019: 2). The most common therapy indications were in oncology (26%) and haematology (18%). 40% were submitted by SMEs. 20% no longer have PRIME designation (3 discontinued development, 5 submitted for EC MA, and 2 received EC MA: the CAR-T cell therapies: tisagenlecleucel [in ALL] and axicabtagene ciloleucel). All HTA bodies in scope had appraised both, 6/8 with positive outcomes (though 4/6 conditionally): NICE recommended both (for inclusion in the CDF), the SMC accepted tisagenlecleucel but not axicabtagene ciloleucel, G-BA reported that both offered a non-quantifiable additional benefit, and HAS deemed these offered an ASMR IV and III, respectively. The median delay from MA to positive HTA assessment was 5.2 months.

CONCLUSIONS:

Many therapies have received PRIME designation, but to date more have had their development discontinued than have received European MA. Nevertheless, the two EMA-approved therapies with PRIME designations have received broad reimbursement across several major markets, but these recommendations are frequently conditional and add additional delays to patient access. Better integration with payers may be necessary to ensure that accelerated regulatory approval programs translate into patent access.