Platform Trials

A step closer to adaptive platform trials for four diseases with high unmet needs

By Sarai Rodríguez Navarro and Joan Genescà (VHIR)

EU-PEARL (European Union-Patient-cEntric clinicAl tRial pLatforms), an IMI (Innovative Medicines Initiative)-founded project,  was conceived as a means to change the paradigm of clinical trials in Europe, by promoting more adaptive, efficient and patient-centered clinical trials, thus contributing to accelerate drug development and the access to new and more effective treatments, while addressing unmet health needs.

Four disease areas of high unmet clinical need with different design challenges were chosen as a model that would then serve for other disease areas: 1) major depressive disorder (MDD) as an example of a mental health condition which needs a better phenotypic characterization to optimize therapeutic targeting, 2) tuberculosis (TB) as a highly prevalent infectious disease entailing an enormous economical and medical burden and that is markedly concentrated in low-resources countries, 3) non-alcoholic steatohepatitis (NASH) as a largely prevalent disease, rapidly increasing in parallel with the global epidemics of obesity and diabetes mellitus, and 4) neurofibromatosis (NF) as an example of a rare disease with limited patient population.

Four disease areas of high unmet clinical need with different design challenges were chosen as a model that would then serve for other disease areas: major depressive disorder (MDD); tuberculosis (TB), non-alcoholic steatohepatitis (NASH), neurofibromatosis (NF).

In these two and a half years, the project has been developing a generic framework and additional tools and guidance materials to conduct collaborative platform trials in which different treatments can be tested at the same time or consecutively for a single disease. These tools and materials are being designed in collaboration with patient representatives, to ensure that the patient perspective is embedded in the design of these trials, and seeking feedback from regulators to discuss the characteristics of the master protocols being designed. Both, MDD and NF, as clinical progressive disorders, include an observational study (longitudinal natural history study, LNHS) as part of their new framework design, helping to understand the long-term course of the diseases and allowing the identification of suitable participants to timely enroll them in the platform trial. Tools to build site capacity and assess their readiness are also being developed within the project.

Tools and materials are being designed in collaboration with patient representatives, to ensure that the patient perspective is embedded in the design of these trials.

The different disease specific teams first identified, together with the medical and patient community, the main challenges to be addressed in the execution of multi-drug platform trials in their specific disease areas (e.g.: the selection of the optimal trial phase and trial endpoints, the use of biomarkers and biopsies, the testing of combination regimens, statistical and regulatory considerations, etc.). For MDD, EU-PEARL has developed the criteria to help identify people who do not obtain sufficient benefit from current antidepressant treatment, classifying a minimal response to antidepressant treatments (treatment resistant depression, TRD) or a partial response (partial response depression, PRD).

The different disease specific teams first identified, together with the medical and patient community, the main challenges to be addressed in the execution of multi-drug platform trials in their specific disease areas.

Additionally, the disease-specific teams have been participating in dissemination activities to create awareness and community engagement around the platform trials being set up. They are also creating an extensive network of clinical trial sites and research infrastructures, with the aim of building four disease-specific integrated research platforms (IRPs) to be sustained after the life of the project, that will make any platform trial designed widely accessible over Europe.

Together, we are shaping the future of clinical trials; once the project is completed and funding is secured, a reusable infrastructure for screening, enrolling, treating and assessing participants in adaptive platform trials in each of the four diseases could be a reality.

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