July 4, 2023
The Alliance for Regenerative Medicine (ARM) says 90% of advanced therapies on the market would have been rejected under proposed EU clinical assessment methods.
The industry raised the concerns about the European Union (EU) joint clinical assessment (JCA) regulation in June, based on an assessment of 18 ATMPs that currently hold EU marketing authorization, including several cell and gene therapies (CGTs).
According to the ARM the methodologies proposed by the EUnetHTA-21 consortium for consideration by the HTA Coordination Group would have rejected 16 of those ATMPs because long-term durability could not be proved at launch, they were not studied in randomized control trials (RCTs), or both.
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ARM CEO Tim Hunt called on the HTA to take a more flexible approach, explaining “there is a clear risk that rare disease patients in the EU […] will not benefit from the next generation of transformative cell and gene therapies under the JCA methodologies proposed by the EUnetHTA-21 consortium.
“We urge the HTA Coordination Group to develop a modernized framework for the JCA rather than defaulting to conservative approaches that were built for yesterday’s pharmaceuticals.”
The EUnetHTA-21 proposal requiring evidence from RCTs for approval – as they are for traditional small molecule drugs – is a major issue according to the ARM.
“Methods developed for traditional pharmaceuticals are not suited for the arrival of ATMPs, which can deliver a profound, durable, and potentially curative impact in a single dose. Single-arm trials are often medically, scientifically, and ethically necessary in the case of ATMPs.”
In addition, the industry group wrote that “patient populations in rare diseases are small, the diseases targeted advance rapidly and leave small windows for treatment, and there are often no treatment alternatives.”
The ARM also argued that it is not feasible to use the length of trials to determine the durability of ATMPs’ treatment effect on the basis that, for ATMPs, clinical trials would need to last for decades and perhaps lifetimes.
Instead the industry group suggested there are other ways to address evaluators’ concerns about the durability and efficacy in the absence of RCTs, citing comparative assessment as an example.
“Comparing single-arm results to a synthetic comparator from a robust disease registry can provide evidence of added benefit, while gathering real-world evidence (RWE) post-approval can provide evidence of durability not available at the time of approval.”
The regulation is due to enter into application in 2025.
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