TERMIS PAP: Session 2 — Building an Evidence Base
The principle of “evidence base” comes from the clinical background, from evidence based medicine. All products/therapies offered to patients must have a strong evidence base supported by well-designed and -conducted research.
To provide a strong evidence base for an advanced therapeutic product the following must be performed:
1. The proof of concept must be demonstrated both in vitro and in vivo. What sort of experimental model is used must be carefully considered. The appropriate model for the proposed effect may have to be developed.
2. The safety of each component in the therapeutic must be tested (risk assessment, possible side effects, stability).
3. Efficacy of each component must be tested (mechanism of action, added value to the overall system must be investigated).
4. The ideal parameters of each component (e.g. concentration of bioactive agents) and how it will affect the final results must be understood.
5. Fundamental research must be performed to understand the underlying science of the proposed mechanism of the therapeutic.
6. A systematic review of scientific literature: Where does your technology stand with respect to the current state of the art?
7. Meta-analyses: What kind of proof does your experimental work provide in light of the analysis of the aggregate results in the relevant field?
8. Randomised control trial: What is the clinical and pre-clinical scenario for your product/idea? What are the measures to prevent bias?
A product will only be authorised when safety, efficacy and quality can be demonstrated. In Europe to obtain authorisation an advance therapeutic must be assessed by the Committee for Advanced Therapies (CAT) of the European Medicines Agency (EMA). It is recommended to study the materials provided by this agency.
It is also worth noting that the Committee for Advanced Therapies is happy to answer the questions of those seeking authorisation for their products.
It is also important to mention the Enterprise Europe Network, the world’s biggest technology transfer organisation. The Network helps small and medium-sized enterprises (SMEs) make the most of business opportunities in the EU and beyond. This organisation can help your business find European cooperation partners and funding possibilities for the commercialisation of your technology/therapy.
Committee for Advanced Therapies (CAT): http://www.ema.europa.eu/ema/index.jsp?curl=pages/about_us/general/general_content_000126.jsp&mid=WC0b01ac05800292a5
Enterprise Europe Network: http://een.ec.europa.eu/