1. BioCapital Europe 2019, Amsterdam
30 May 2019
European Medicines Agency – fostering development of innovative medicines
BioCapital Europe 2019, Amsterdam
14 March 2019
Presented by Prof Guido Rasi, Executive Director

2. These PowerPoint slides are copyright of the European Medicines Agency
These PowerPoint slides are copyright of the European Medicines Agency. Reproduction is permitted provided the source is acknowledged.

4. Medicines Regulatory Agencies: protector & promoter of public health
Gatekeeper
Enabler
From the EMA mission statement:
“We support research and innovation to stimulate the development of better medicines”

5. Regulatory challenges of ‘innovative products’, examples
Advanced therapies (gene, cell, tissue based); some truly ‘personalised’; benefit-risk “context dependent”; single intervention – long-term outcome
Precision medicine: radio-theranostics; stratification criterion often incompletely understood; small patient numbers
Early disease interception: extremely long observation periods for benefit-risk assessment
Personalised treatment combinations: combinatorial complexity; small patient numbers; clinical indication difficult to define

6. Pharmaceutical R&D in Europe
30 May 2019
Pharmaceutical R&D in Europe
Clinical confirmatory
Clinical exploratory
Non-clinical and First in Man
Authorisation and post-licensing
Early Research
A medicinal product takes an average of years to reach the market from discovery;
The cost of R&D for a new chemical or biological entity was estimated at € 1,926 million in 2013 (DiMasi et al, Journal of Health Economics, January 2016);
Attrition rate is high, on average only 1 to 2 of every 10,000 substances synthesised in laboratories will successfully pass all stages of development required to reach the market.

7. (Smietana et al, Nature Reviews Drug Discovery,2016);
30 May 2019
Trends in clinical success rates for medicines over past 2 decades
(Smietana et al, Nature Reviews Drug Discovery,2016);

8. What can regulators do to enable innovation?
30 May 2019
What can regulators do to enable innovation?
Provide regulatory guidance and support to medicines developers
Provide supportive scientific environment and standards:
Adapt evidence standards to specific products and feasibility of studies
Contribute to the progress of regulatory science
Qualification of scientific methods
Collaborate with HTAs to define data requirements for market access

9. EMA Regulatory Science to 2025
Aim to build a more adaptive regulatory system that will encourage innovation in human and veterinary medicines
guidance on modernised medicines developments
facilitate the optimisation of regulatory science
assess benefits and risks of innovative therapies and diagnostics based on new technologies
Under public consultation until 30 June 2019

10. Closing remarks:
Regulators are at the crossroads of science, values and law, which allows to enable innovation
Experience shows that regulators can successfully guide medicines development hence supporting to avoid failures
EMA has and will contribute to regulatory science, in collaboration with external partners
Developers are encouraged to engage early with EMA and continue dialogue throughout the medicines lifecycle

11. Thank you for your attention
30 May 2019
Thank you for your attention
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