Adaptive pathway seeks to maximize the positive impact of new drugs on public health by balancing timely access for patients, with the need to provide adequate evolving information on benefits and risks.

Adaptive pathways can be defined as a proactively planned, adaptive approach to bringing drugs to market. This approach enables a medicinal product to be authorised for use in a controlled/limited patient population, during which additional clinical evidence is collected and assessed. The authorisation may then be applied to a larger patient population. The approach allows an expedited access to medicines in unmet medical need areas, while still shielding the more general public from risks associated with the product until further data can be assembled.

The purpose of the Adaptive pathways is to use the regulatory procedures within the current EU legal framework, including scientific advice with Regulators and/or HTA bodies, compassionate use programs, conditional marketing authorisation, marketing authorisation under exceptional circumstances, risk management plans, patient registries, etc.

Adaptive pathways in the EU

Adaptive pathway is based on the three principles defined by EMA:

  1. Iterative development,
  2. Gathering of evidence through real-life data to supplement clinical trial data and
  3. Involvement of patients as well as Health Technology Assessment (HTA) bodies/Payers in the product development design.

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