The healthcare biotechnology sector offers huge business and commercial opportunities; however it also requires heavy and risky investments which are often lacking in Europe, hampering the development of the industry.
The challenge includes either:
a) Cell technologies in medical applications (phase 1 only for 2016 deadlines and phase 2 for all deadlines in 2016 and 2017)
Cell technologies include cell manufacturing (culture, multiplication, scale-up and automation), preservation, banking and transport; identification, cell sorting and delivery, imaging, tracking, process and quality control; genetic engineering and gene editing; production of therapeutic biomolecules. The medical applications of cell technologies include diagnostics and biosensors; cell and gene therapy, tissue engineering, bio-artificial organs, haematology, immunotherapy, and vaccine and antibody production; predictive toxicology, synthetic biology, and modelling development and disease processes.
However, the diversity, complexity and variability of living cells pose challenges for bringing safe, reliable, regulatory-compliant and cost-effective products to the market and to the patient. SMEs developing cell-based products and processes have limited financial resources to take the critical steps to move from proof of concept to practical application while at the same time addressing considerations such as scale-up/scale-out, automation, logistics, regulatory pathways and business models.
Particular attention should be given to dialogue with regulators and compliance with safety and regulatory requirements, such as those pertaining to cell procurement, GMP, ethics, clinical trials, ATMPs and medical devices.
The challenge addresses cells from any eukaryotic source though their eventual application must be to human medicine.
b) Clinical research for the validation of biomarkers and/or diagnostic medical devices (only at the first cut-off date in 2017 and for phase 2 applications - phasing out of the topic PHC-12-2014/2015 introduced in the Work Programme 2014-2015)
Biomarkers are used in clinical practice to indicate both normal and pathological conditions. They are also used for predictive or prognostic purposes. They are being used increasingly in medicine and many potential new biomarkers are proposed every year. However, only a few of these have been validated for clinical use. To achieve validation a robust analytical method is required and a link to a pertinent clinical process or endpoint needs to be demonstrated.
This validation process should provide evidence for high analytical value, appropriate sensitivity and specificity, and clinical validity. Particular attention should be given to validation of biomarkers with potential for rapid uptake into clinical practice. Both in vivo and in vitro potential biomarkers are eligible. Priority is given to the validation of disease-related biomarkers (i.e. diagnostic, susceptibility/risk, monitoring and prognostic biomarkers). Validation of the clinical performance of new diagnostic devices can also be supported, either in combination with the biomarker validation or against existing standards.