Alarm bell rung over EU’s unified HTA plan

The EU’s joint clinical assessment (JCA) initiative has been set up to accelerate patient access to new therapies, but could end up restricting the use of new technologies like cell and gene therapies, according to patient advocacy groups.

The Alliance for Regenerative Medicine (ARM), EveryLife Foundation for Rare Diseases, and Rare Diseases International claim that almost 90% of the advanced therapy medicinal products (ATMPs) already authorised in the EU would have been rejected under the JCA methodology.

The JCA was introduced to provide a centralised health technology assessment (HTA) process to aid reimbursement deliberations in EU member states where pricing and reimbursement infrastructure is lacking.

The idea is that it covers four key principles of an HTA – identifying a health problem and current therapies, and assessing the technical characteristics of a new treatment, as well as its safety and efficacy. That can be used as a starting point for national authorities so they don’t have to duplicate those elements.

That’s a worthy objective, but according to ARM, the baseline methodologies proposed do not recognise the unique characteristics of ATMPs. If applied to current therapies, they would have rejected all seven gene therapies currently approved in the EU for rare genetic diseases.

Overall, out of 18 approved ATMPs, 16 would not have made the cut because they would be unable to show long-term durability at launch, were not studied in randomised clinical trials, or both, according to ARM.

As they generally target rare diseases, ATMPs are often tested in single-arm studies as patient populations are small, the diseases advance rapidly and have short windows of opportunity for treatment.

It is also often not feasible to use trials to determine the durability of a treatment effect, as they would have to “last for decades and perhaps lifetimes,” according to a position paper from the advocacy groups. A better alternative would be the use of patient registries to gather real-world evidence on durability, it suggests.

The JCA will be administered by the HTA Coordination Group, using methodologies proposed by the European Network for Health Technology Assessment (EUnetHTA-21) consortium, which includes representatives of around a dozen HTA agencies from EU member states.

The implementation schedule is that oncology medicines and ATMPs will be assessed starting in 2025, followed by orphan medicines in 2028, and all new medicines and some high-risk medical devices in 2030.

“There is a clear risk that rare disease patients in the EU – many of whom face death or serious disability – will not benefit from the next generation of transformative cell and gene therapies under the JCA methodologies proposed by the EUnetHTA-21 consortium,” said ARM chief executive, Tim Hunt.

“We urge the HTA Coordination Group to develop a modernised framework for the JCA, rather than defaulting to conservative approaches that were built for yesterday’s pharmaceuticals.”

ARM is hosting a panel discussion at the Karolinska Institute in Sweden next week to discuss the impact of the JCA on the competitiveness of Europe’s ATMP sector.