Task 1: Evaluation of regulatory path for hospital exemption for CardAP cells.
Subcontracting of specific regulatory expertise: Applying for Hospital exemption for Advanced Therapies Medicinal Products Guideline (ATMPs): implementation of Art 28(2) of Regulation 1394/2007.
The management of CellServe talked to regulatory experts about the strategy to apply for a hospital exemption. Experts think that the hospital exemption could be applied for, if the clinical data of the phase I study will be convincing and demonstrate that patients will have a clear benefit. The problem using this instrument is the administrative efforts necessary to get a reimbursement because contracts have to be negotiated with hospitals and health insurances.
Task 2: Evaluation of regulatory path and development costs until European market authorisation
CellServe has calculated the costs of the product development until market authorization.
Evaluation of detailed regulation path for European approval and market authorization according ATMP-guideline.
CellServe started with the development of the CardAPcells several years ago. The company considered the ATMP regulation during the whole development process and involved external experts to fulfil all relevant regulations. The last steps in this development process are the clinical studies, which will demonstrate the safety and efficacy of the CardAPcells and the GMP production, which was established by Fraunhofer Institut IZI (FHG-IZI).
Task 3: Development of a business plan for the CardAP cells project.
Regulatory and marketing strategy, financial planning, calculation of financial requirements, time schedule of the development, subcontracting.
In close collaboration with ConsulTech and the subcontractors the management of CellServe developed a business plan, which was integrated into the phase 2 project application. This business plan includes the development strategy, the marketing plan and the financial plan, including the plan how to raise venture capital from private and public investors.
Task 4: Evalution of CardAP manufacturing costs.
CellServe will reduce costs and investments in manufacturing by a cooperation with FHG-IZI as a well established contract manufacturer with a GMP-production.
Task 5: Evaluation of reimbursement in countries such as UK, France, Spain and Italy.
In each country the specific regulations have to be evaluated.
https://ct.catapult.org.uk/sites/default/files/Market-access-case-study-0814_v3.pdf(odnośnik otworzy się w nowym oknie)CellServe decided to start marketing under hospital exemption in Germany and not in other European countries, because the German market with 45,000 patients is very large and the time to achieve the full market authorization will be 2023/2024. In this time period many rules could change and it seems to be more reasonable to evaluate the reimbursement systems in more detail in 2019.
Task 6: FTO analysis
A FTO analysis was performed by ConsulTech in December 2016. The report showed that there are hundreds of patents and patent applications with claims concerning cell therapy of heart muscle. None of these describes a cell type with the same properties of CardAP cells. Most of the publications describe the use of stem cells or induced pluripotent stem cells (IPSC). The patent of CellServe/Charité does not infringe any other IP.
Task 7: Completion of the clinical study protocol.
The clinical study protocol was finished and will be up loaded on the EC server and submitted as part of the phase 2 application.
Task 8: Marketing study showing the potential of the CardAP cells.
The marketing study was updated. CardAP cells will have the potential to create a billion $ market, if the clinical results will demonstrate safety and efficacy and if CellServe will collaborate with other companies to market the product in all relevant markets.
