Integrated research platforms enabling patient-centric drug development

The action aims to address these issues by creating, in a collaborative rather than competitive manner, a reusable and modular approach for the design and execution of patient-centric platform trials.

It will create best practices, tools and guidelines for establishing multi-company platform trials by leveraging, extending and improving concepts from previous pioneering multi-company platform trials to new disease areas.

The proposal is divided into (i) a set of common foundational elements applicable to all disease areas, (ii) clinical networks and networks of patient-level data and (iii) disease-specific integrated research platforms in several disease areas: major depressive disorder (MDD), tuberculosis (TB), non-alcoholic steatohepatitis (NASH) and neurofibromatosis (NF).

Despite good progress in many areas of healthcare, it seems that clinical development rather than discovery research is the limiting factor for innovative new products and treatment options to reach patients.

Root causes of this include (i) siloed and competitive development process focussed on single compounds with transient clinical trial infrastructures, (ii) insufficient collaboration among industry and between industry, not-for-profit product developers and academia, (iii) limited patient-centric alignment of stakeholders, and, (iv) in general, the limited focus on individual patient-tailored treatments.

As a result of these, there is a clear shortage of (i) investigators & investigational sites for phase 2-3 clinical trials, (ii) patients for enrolment in such trials, (iii) sharing of insights and use of real-world data, and (iv) investigations of combination treatments, in particular from different sponsors.

Developing the proposed reusable IRP and platform trial approach in Europe will deliver a tangible advantage for developing innovative new medicines, and for advancing fundamental and applied medicines research in general, in academia and industry. This includes four platform trial protocols fully ready for execution for diseases of high unmet need in scope of the World Health Organisation (WHO) priority medicines list.

This will allow patients to benefit quicker from medical innovations, both through accelerating new medicines development in general but also through faster enrolment in clinical trials with a lower chance of being randomised to the placebo, and potentially a higher likelihood of being allocated to the most promising treatments for individual patients, including multi-company combinations of medicines.

It will also increase participation of patients in the design of clinical trials and in the development of predictive biomarkers and trial endpoints that are clinically meaningful and approved by regulators.