New HTA Evidence Guidelines In EU Could Spell Trouble For Advanced Therapy Companies

New guidelines that cast doubt over the use of single-arm and non-randomized studies are “discouraging” for rare disease patients and the advanced therapy sector, says ARM, the Alliance for Regenerative Medicines.

Missed Target
New evidence guidelines could see HTA Regulation Miss its target of reducing duplication of work • Source: Shutterstock

New guidelines on direct and indirect comparisons for generating evidence for forthcoming EU-level joint clinical assessments (JCAs) will likely jeopardize national level implementation of the EU Health Technology Assessment (HTA) Regulation and put at risk swifter access to medicines, ARM, the Alliance for Regenerative Medicines, has warned.

The alliance said that together with what it considers to be a flawed draft Implementing Act, the guidelines mean the “JCA

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