Organ transplantation is in many cases the only effective therapy for end-stage organ failure. However, it is estimated that only 10% of the global organ needs are covered. Traditionally, only organs obtained in optimal conditions have been considered for transplantation. However, organ lack is leading to the increased use of suboptimal grafts (e.g. non-heart beating donors), which poses new challenges. The most important hurdle preventing the clinical use of suboptimal organs is the limitation of static cold storage (SCS). SCS is well suited for optimal grafts, but not for suboptimal ones since (i) it allows short preservation times (2-20 h), (ii) it does not provide information about the organ viability and (iii) it additionally damages the organ due to ischemia-reperfusion.
The solution proposed by EBERS is a new portable medical device based on normothermic perfusion, capable of maintaining human organs in a viable state for sustained periods under physiological conditions. The NORMOPERF project will offer the clinical community a new device for organ preservation that will allow for a x2-3 increase of the current transplant rates, will enable extended preservation times, will improve the clinical results thanks to the use of enhanced preservation conditions and will provide objective real-time information on the quality and function of the organ.
Our technology has already been proven in preclinical tests based on a porcine transplant model. The NORMOPERF project focuses on the industrialization of the technology, on obtaining the regulatory approval as a new medical device and on the execution of a multi-center clinical trial. The work plan is divided in six work packages, as shown in detail below.
|Industrial manufacturing of the commercial version of the product
|Quality assurance and regulatory
|Clinical study for validation of the commercial version of the device/consumables
|Plan for commercial readiness and market access
|IP, dissemination & communication
|Operations, financial and technical project management
Industrial manufacturing of the commercial version of the product
The aim of WP1 is to adapt the manufacturing process of the device and its consumables to make it compatible with large scale production and the regulatory requirements in the EU (CE marking) and in a post-project stage in the USA (FDA). The obtained final version of the product will be suitable from a technical, production and regulatory perspective to be employed in subsequent quality & regulatory activities (WP2) and the clinical trial (WP3). The main steps to give in this WP are as follows:
Quality assurance and regulatory
The goal of WP2 is to obtain the regulatory approval for our technology as a medical device, which involves (i) managing successfully the achievement of the CE-marking for commercialization of NORMOPERF in EU, and (ii) establishing the strategy for future access to the USA market, including a post-project plan to achieve the FDA approval. The following tasks will be carried out:
Clinical study for validation of the commercial version of the device/consumables
In WP3 we will demonstrate the efficacy of our device in kidney transplantation procedures. This will be done by means of a multi-center clinical study in kidney transplantation. In this clinical trial the performance of the technology will be evaluated vs. the gold standard method (static cold storage, SCS) by comparing results achieved by using NORMOPERF (experimental group) with results from SCS (control group). This WP will be performed in collaboration with principal investigators (PIs) of seven Spanish transplant centers. Furthermore, the study monitoring will be done by a subcontracted CRO. The following steps will be taken in this WP:
Plan for commercial readiness and market access
WP4 is devoted to define a precise plan for market access in the EU first and later in the US. Thus, the main objective of this WP is to prepare and implement a market access plan for reimbursement of NORMOPERF technology in the EU countries selected as priority in EU for commercialization in the first selling years. To achieve this goal, we will perform the following tasks:
IP, dissemination & communication
The main goal of WP5 is to provide a wide visibility to the NORMPERF project and our company among the clinical community, key stakeholders and the general public. Moreover, this WP will ensure an efficient and appropriate management of our intellectual property, including the international extension of our IP and competitive intelligence activities.
Operations, financial and technical project management
The overall objective of WP6 is to ensure that all aspects of the NORMOPERF work plan are appropriately managed throughout the duration of the project and all administrative aspects are undertaken and delivered according to EC requirements. More specifically: