The observed discrepancy between effects of a health intervention in routine clinical practice as compared with the effects demonstrated in randomised controlled clinical trials.
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:
- Iterative development,
- Gathering of evidence through real-life data to supplement clinical trial data and
- Involvement of patients as well as Health Technology Assessment (HTA) bodies/Payers in the product development design.
Latest Glossary Definitions
Documentation submitted to facilitate an Agency meeting, for example to request scientific advice in relation to a proposed drug development pathway. It should contain company questions, justifications or company positions, and appropriate summary information.
The effect that something is likely to have on the treatment of a particular group of people, or on the results of treating that group.
In relation to Policy 70, CCI shall mean any information contained in the clinical reports submitted to EMA by the applicant/MAH which is not in the public domain or publicly available and where disclosure may undermine the legitimate economic interest of the applicant/MAH.
Required under the new MEDDEV 2.7.1 Rev. 3 guidelines. Document containing information and results generated in conducting an evaluation of the clinical safety and performance of a medical device.
A CE Technical File is a comprehensive collection of documents providing medical device composition, specifications, manufacturing process, rationale for qualification and classification, non-clinical testing results and clinical evaluations, risk analysis and instructions for use/Labeling.
A systematic evaluation of the evidence on the outcomes of different drugs or other options for treating, preventing, or diagnosing a medical condition.
The conduct and/or synthesis of research comparing different benefits and harms of alternative interventions and strategies to prevent, diagnose, treat, and monitor health conditions in routine clinical practice (i.e. the real-world setting).
Core value dossiers are used in the frame of reimbursement negotiations and to support HTA submissions, as well as national and local payers.
A core reimbursement dossier is submitted during the late stage of development of a product, in order to be considered for reimbursement.