German watchdog criticises EMA adaptive pathways approval

Germany’s health cost effectiveness watchdog has raised  serious concerns about the European Medicine Agency’s plans to accelerate approval for promising new drugs, the so-called Adaptive Pathways initiative.

Earlier this month the EMA published a report on a pilot project of the adaptive pathways, a provisional approval procedure which allows faster access to potentially groundbreaking drugs in areas of high unmet medical need.

The concept is that drugs can be rapidly licensed in a small patient population first, with further studies conducted subsequently to determine whether they are safe and effective in broader populations.

But Germany’s Institute for Quality and Efficiency in Health Care (IQWiG) says the report has confirmed its fears about the project – namely that there is no clear plan for how post-marketing real world data can be used to provide concrete evidence of clinical and cost effectiveness.

In a commentary released this week it states: “Evidently neither industry nor EMA has a concept as to how real world data can be used after drug approval to allow drawing reliable conclusions on benefit and harm.”

The Adaptive Pathways plan.is part of a wider move among regulators to increasingly move towards a more conditional approval process – recognising that patients need new treatments faster, and that full data is in any case rarely likely to be available at launch.

In particular, the EMA’s project follows in the footsteps of the FDA’s Breakthrough Therapy Designation, which has had a major impact on the approval rates in priority areas over the last 2-3 years.

However IQWiG underlines that the EMA’s own report on its pilot project has found shortcomings in pharma plans for real world data collection, a vital component of its scheme.

The Germany agency points out that the report found that “The majority of the plans were vague in terms of the purpose of collection of real world data to supplement RCTs, and on the practical elements for implementation there was insufficient detail in the submitted proposals to explore the refinement of the safety profile, and even less about to what extent efficacy could be confirmed or augmented in the post-authorisation phase.”

The report concluded: “a critical discussion on the quality, potential for bias, and reliability of the data acquired in the post authorisation setting, and their suitability for regulatory and HTA purpose, was lacking.”

IQWiG says the EMA also lacks a plan to address these issues, and therefore concludes it is “high time to pause for a moment and rethink the whole concept” rather than the EMA’s current plan of moving to expand the programme.

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