The aim of the Agence de l'innovation en santé is to facilitate the development and availability of healthcare innovations that benefit patients, professionals or the healthcare system. It acts along the entire innovation value chain, from research to the actual delivery of innovations. For its part, the F-CRIN National Infrastructure, an organization providing expertise in clinical research, has a priority focus on long-term clinical trials in its "2024-2028" roadmap. Together, AIS and F-CRIN have decided to carry out a study to assess the performance, value and safety of methods other than RCT (Randomised Clinical Trials), which remains the gold standard in clinical research, based on practical applications, and to determine the terms and conditions for their use in setting up clinical trials.
Presentation of the mission
The AIS collaboration with the F-CRIN Infrastructure
The establishment of the Health Innovation Agency (AIS) is one of the measures of the Health Innovation 2030 plan, the health component of France 2030. One of its missions is to help accelerate the effective availability of innovations by proposing simplifications to existing processes, including the clinical evaluation phase.
In order to facilitate market access and boost France's attractiveness for clinical research, the AIS wanted to develop the use of new clinical research methodologies, a potential guarantee of acceleration in obtaining clinical trial results with the same level of safety. Several actions have been put in place as part of the France 2030 plan, including funding for data warehouses and cohorts.
To continue this work, the AIS, in association with the F-CRIN clinical research infrastructure, has set up a think tank to steer a focus group on these new clinical research methodologies. The aim of this expert group is to promote the dissemination and reinforce the acceptability of new methodological approaches using real-life modelling and data. The first results of this work are expected in late 2023-early 2024.
New methodological approaches to conducting clinical trials
Three categories of innovative methodological approaches have been identified that the collective will focus on:
- Optimization of current clinical trial design (combined trials; platform trials)
- Integration of external information (Bayesian trials ; digital health data/EHR)
- Benefit-risk extrapolation (via external control groups such as registers or cohorts ; synthetic arm/"in silico" type mechanistic models)
Scope of work
The aim of this group is to propose new clinical research modalities to be mobilized in cases where the traditional scheme of clinical trials, which take place in successive stages, following a very rigid evaluation process is too complex to implement. The aim will therefore be to propose complementary or alternative methods to address the specific limitations raised by the randomized controlled trial: patient volume not always compatible, implementation of the control arm.
The objective of the expert collective brought together by the Agence de l'innovation en santé (AIS) and F-CRIN is to go beyond previous work and make a significant contribution to the effective recognition of these approaches, notably through the monitoring of case studies and the evaluation of their impact in terms of performance and value. The aim is to specify the conditions and terms of use of these alternative approaches, including for which pathologies they are suitable and at what stage of clinical development they can be employed.
The aim is not to replace the traditional model, but to demonstrate the added value of these new approaches at key moments in the clinical evaluation of healthcare products. With this in mind, the Innovation Santé 2030 program, aimed at stimulating healthcare innovation in France, offers an incentive framework for the adoption of these new approaches.
Expected deliverables
- Current 2023: Review of current situation, hearings; Analysis of case studies and "success stories", i.e. clinical trials conducted using methodologies other than RCT and which have been taken into account for the registration and reimbursement of a healthcare product
- End of 2023: Formulation of recommendations for use (use cases) and definition of acceptability criteria
- 2024/2025: Follow-up of pilot cases selected on the basis of a call for expressions of interest, with a view to demonstration
A process of reflection conducted in conjunction with the regulatory authorities
Concerned not to duplicate reflections already conducted or underway, contacts have been made with the ANSM and HAS to inform them of the launch of the work and to define with them the areas where the use of new methodological approaches would be most useful.
Composition of the expert group and setting up of working groups
A group of 31 experts
A wide-ranging collective of experts, including methodologists, members of health agencies, professionals from healthcare establishments and public research organizations, professionals representing biotechs, medtechs and digital companies in healthcare and artificial intelligence, are joining forces to move on to demonstrating the performance and value of new methodological approaches and defining the framework and terms of their use.
Three sub-groups have been set up
- Group 1: "Accelerate development and enrich the level of evidence in the initial phase of evaluation for marketing and reimbursement"
- Group 2: "Measuring performance under early and long-term conditions of use"
- Group 3: "Data management: level of structuring, quality and validity of data"
.
