European Medicines Agency fostering development of innovative medicines

European Medicines Agency – fostering development of innovative medicines Bio. Capital Europe 2019, Amsterdam 14 March 2019 Presented by Prof Guido Rasi, Executive Director An agency of the European Union

These Power. Point slides are copyright of the European Medicines Agency. Reproduction is permitted provided the source is acknowledged. 1

Medicines Regulatory Agencies: protector & promoter of public health Gatekeeper Enabler From the EMA mission statement: 3 “We support research and innovation to stimulate the development of better medicines”

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 4

Pharmaceutical R&D in Europe Early Research Non-clinical and First in Man Clinical exploratory Clinical confirmatory Authorisation and postlicensing • A medicinal product takes an average of 12 -13 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 (Di. Masi 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. 5

Trends in clinical success rates for medicines over past 2 decades 6 (Smietana et al, Nature Reviews Drug Discovery, 2016);

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 7

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 8

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 9

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