This group of experts, united to demonstrate the performance and value of new methodological approaches, is subdivided into three subgroups, 1. to "Accelerate development and enrich the level of evidence in the initial phase for marketing and reimbursement", 2. to "Measure performance under conditions of use, both early and over the long term", 3. to "Manage data: level of structuring, quality and validity of data".
Accelerate development and enrich the level of evidence in the initial evaluation phase for marketing and reimbursement
Coordinated by Sarah Zohar (INRIA), Raphaël Porcher (AP-HP)
This group is reflecting on the conditions of acceptability of methodologies other than the "gold standard" RCT (randomised control trial) with a view to market authorisation and access to reimbursement n focusing on three use cases:
- Demonstrating organizational impact, particularly for remote monitoring devices
- Digital therapy (TDX)
- Rare disease or condition with small numbers
Feedback has been sought from the HAS to guide the working group's thinking and work and the choice of use cases on:
- The areas, cases, where the use of new methodologies is most useful to evaluate (in order to clear up any areas of vagueness, lack of demonstration of interest)
- Cases (trials) of which you may be aware, using new methodologies in the drug, DM or digital field, either having obtained registration or, on the contrary, not having obtained it, specifying the reasons.
For aspects concerning digital DM (remote monitoring, TDX), the group held hearings with institutions and project leaders. These highlighted the most pressing issues to be addressed in the course of this work, and the initial levers to be mobilized. These hearings will provide input for the specifications of the cases to be proposed and monitored.
The same working methodology will be applied to rare or slowly evolving diseases. Work will focus primarily on the challenges of in silico arms (patients or virtual cohorts). Initial hearings will be aimed at methodology providers and project leaders who have made use of these methodologies.
The main deliverable
This will be a recommendation for project managers, for each scenario, on the design to be used, the data to be mobilized, the operational implementation of the study.
For the regulator, the group will propose the keys to evaluating the methodologies retained and advised in the recommendations, training for evaluators as well as the modalities for setting up a monitoring and advisory system for authorities.
Measure performance under early and long-term use conditions
Coordinated by Fabrice André (IGR) and Emmanuel Pham (Sté Novadiscovery)
The aim of this sub-group will be to establish the innovative methodologies to be mobilized to be able to evaluate the efficiency of a healthcare product over time, throughout its life cycle and use. This will enable the regulator to be equipped with evaluation tools for the implementation of contracts and performance-based management of innovation. To this will also be added the evaluation of prevention, in a second phase.
Thus, this group will work on measuring the variability of a response to a treatment, attempting to go as far as identifying patients who would not respond to a treatment.
To our knowledge, there is little work on this measurement of product efficiency under conditions of use by mobilizing new clinical research methodologies.
This group decided to take 3 different situations as a starting point and see what methodologies could be mobilized to address them:
- The health product with very high efficacy but for which the data collected are incomplete and patchy
- The health product has demonstrated "state-of-the-art" efficacy, but the benefit remains modest
- The health product is highly effective and needs to be evaluated in a short cycle, which leads to therapeutic de-escalation
The main deliverable
This will be a recommendation for project sponsors, for each scenario, on the design to be employed, the data to be mobilized, the operational implementation.
For the regulator, the group will propose changes to the regulatory framework and requests to be made to industrialists and academic sponsors (type of data/analysis to be generated, timeframes for evaluation...).
This group will also provide the keys to assessing the methodologies adopted and recommended in the recommendations, training for assessors, and ways of setting up a monitoring and advisory system for the authorities.
Data management: level of structuring, quality and validity of data (how to ensure that data is of high quality and can be put to best use)
Coordinated by Mathieu Robain (Unicancer) and Stéphanie Kervestin (ARIIS).
This group is transverse and collaborates with Axis 1 and Axis 2.
In the first instance, recommendations will focus on data quality and governance, as these two factors have an impact on the sustainability of cohorts and databases.
This group will propose recommendations on the role of the patient in managing these data (information, consent?) or recommendations on making these data available (fast-track for data access?). As such, this group will focus on :
- The tools and technologies to be mobilized
- The data collection methodology and quality level
- Data use
- Regulatory aspects (patient information, CNIL...)
- Interoperability and sharing
In a second phase, we could also explore the experience of traditional cohorts used for clinical research, and propose recommendations on structuring "cohorts of the future", generated using data collected by digital medical devices (connected bracelet, diabetes self-test, remote monitoring tool).
