OBJECTIVES:

Rare diseases can have a catastrophic impact on patients’ lives. The European Medical Agency (EMA) has developed new Health Technology (HTA) regulations and has recently launched a Joint Clinical Assessment (JCA) for medicines (including orphan drugs [ODs]), which is now in the pilot phase. The JCA process aims to ensure a uniform clinical assessment at European (EU) level.

This review explores how the new JCA route has been implemented for ODs following publication of the new EMA regulations (December 2021).

METHODS:

A literature review was conducted using Embase® (2017–2022) using “rare diseases”, “orphan drugs” and “JCA” or “HTA” (and related synonyms) specific terms. Abstracts and full texts were screened against pre-specified eligibility criteria including articles related to the assessment of ODs.

RESULTS:

The search identified five relevant publications. Of these, only one described the landscape for approval of ODs via the new JCA route, three described joint assessment via other joint HTA initiatives such as EUnetHTA, FINOSE, BeNeLuxA, the Valletta Declaration and FAAP, whilst the remaining one investigated a multitude of European programmes for ODs’ assessment. Whilst the three publications on other JCA routes indicated the clear need to align processes to ensure consistent evaluations and approvals within the EU, the article on the new JCA route called for enhanced alignment of regulatory and HTA evidence requirements among others. One additional publication highlighted that only one advanced therapy medicinal product (ATMP) had been approved via the new JCA route, elivaldogene autotemcel for the treatment of cerebral adrenoleukodystryophy.

CONCLUSIONS:

This review identified that no novel ODs were assessed via the new JCA EMA route for rare diseases in the pilot phase. The new EMA JCA route could provide a more homogenous clinical assessment across EU countries that could enable faster pricing and reimbursement negotiations specific to rare diseases.