Regulatory Science and Innovation from the EU perspective

Transcription

1 Regulatory Science and Innovation from the EU perspective Pekka Kurki Finnish Medicines Agency 11/6/2014 Regulatory science and innovation 1

2 Disclaimer This presentation reflects only personal views of the author and does not necessarily represent the position of any regulatory authority, scientific committee, or working party. EMA EMA Fimea 11/6/2014 Regulatory science and innovation 2

3 Regulatory Science and Innovation from the EU perspective EU regulatory network Evolution of the network Increasing role of the European Medicines Agency The role of the Finnish Medicine Agency Innovation and emerging science Innovation and regulatory science Cell-based products and the translational gap Biosimilars a regulatory innovation? 11/6/2014 Regulatory science and innovation 3

4 European Union 500 million users of medicinal products 27 EU Member states European regulators European Medicines Agency > 45 national competent authorities > European Experts 11/6/2014 Regulatory science and innovation 4

5 Evolution of the European regulatory network: Steps towards centralisation 1965: 1975: 1987: 1993: 1995: : 2007: 2008: X National Marketing Authorisation in Finland Harmonised requirements for MA, CPMP Concertation-procedure (biotechnology) Centralised and mutual recognition procedures Euroopan Medicines Agency (EMEA) established Finland joins the EU-network Orphan drug regulation De-Centralised procedure Scope CP widens Pediatric regulation Regulation for advanced therapies medicinal products Scope of CP further widened New pharmacovigilance directive (Clinical trials regulation) 11/6/2014 Regulatory science and innovation 5

6 EU Regulatory Network 11/6/2014 Regulatory science and innovation 6

7 ehdotus European network of regulatory agencies EU Commission: legislation, administration EMA (European Medicines Agency) Centralised MA procedure Committees to solve EU-wide scientific issues National agencies Scientific expertise for EMA Mutual recognition procedure De-centralised procedure National procedures 11/6/2014 Regulatory science and innovation 7

8 Functions of EMA Centralised MA procedure Referrals related to safety of medicinal products Arbitration of disputes between the Member States Co-ordination of inspections (related to CP) Good Manufacturing Practise Good Laboratory Practise (e.g. toxicology) Good Clinical Practise Mutual recognition agreements Co-ordination of sampling and testing Safe limits for residues of veterinary medicines used in food-producing animals Common IT-solutions for the network (portal for applications) Registries (adverse effects, clinical trials, licensed medicinal products) etc 11/6/2014 Regulatory science and innovation 8

9 Strategy of Fimea Active involvement in the committees, working parties and working groups of the EU-Commission and the EMA Special emphasis on biologicals Optimal balance between safety and access 11/6/2014 Regulatory science and innovation 9

10 EMA committees and their working parties PgWP SAWP BWP Cell and gene therapy Tissue engineering SWP BMWP CHMP Chair: Tomas Salmonson CAT Chair: Dr. C.Schneider Oncology Rheumatology & immunology PRAC Chair: June Raine 11/6/2014 Regulatory science and innovation 10

11 Number Human resources of European national regulatory agencies DE FR UK DK SE ES RO BE PT PL CZ AT IE EL NO NL HU FI BG LV LT SI EE IS SK IT MT CY LU Human or Joint PEI Veterinary 11/6/2014 Regulatory science and innovation Pekka Kurki 11

12 Arachnophobia! Spiders generally use elaborate courtship rituals to prevent the large female from eating the small males before fertilization, except where the male is so much smaller that he is not worth eating (Wikipedia). 11/6/2014 Regulatory science and innovation 12

13 Regulatory Science and Innovation from the EU perspective EU regulatory network Evolution of the network Increasing role of the European Medicines Agency The role of the Finnish Medicine Agency Innovation and emerging science Innovation and regulatory science Cell-based products and the translational gap Biosimilars a regulatory innovation? 11/6/2014 Regulatory science and innovation 13

14 RRRRRRRREGULATION Does regulation suppress innovation?

15 New methods and technology need for regulatory science omics Predictive pharmacology Personalised medicine Nanotechnology Predictive toxicology Biomarkers Adaptive study designs Modelling and simulation Bio-farming Risk management Advanced therapies Therapeutic vaccines Biosimilars 11/6/2014 Regulatory science and innovation 15

16 Potential targets for cell-based therapies Experience of product classifications in CAT Examples degeneration of the cells of the retina extended corneal lesions traumatic spinal cord injury severe dermal lesions chronic myocardial ischemia various cancer types autoimmune diseases inborn errors of liver metabolism Cell-based therapies may satisfy unmet medical need in many serious and life-threatening diseases. 11/6/2014 Regulatory science and innovation 16

17 Advanced Therapies Medicinal Products in EU % of the ATMPs under clinical development were gene therapy products and 78% cell-based products. The majority of sponsors were charities and academics and 40% were commercial enterprises. Almost all companies were small or medium-sized. Most products are being developed for various cancers, cardiovascular diseases, hematological and musculoskeletal conditions as well as for infections. Maciulaitis et al., Molecular Therapy 20: 479, /6/2014 Regulatory science and innovation 17

18 Novel ATMPs in clinical trials / EU IMPs * GTMP sctmp TEP * Cumulative number of new applications (report is suggestive and should not be considered exhaustive) This report has been generated from the EudraCT Data Warehouse. 11/6/2014 Regulatory science and innovation 18

19 Support to innovation by the EMA Innovation think tank Innovation Task Force & SME office Scientific Advice CAT CHMP working parties (guidelines) Innovative Medicines Initiative (IMI) US Critical Path Institute initiatives 11/6/2014 Regulatory science and innovation 19

20 Strategy of Fimea Active involvement in the relevant committees, working parties and working groups of the EU- Commission and the EMA Special emphasis on biologicals Optimal balance between safety and access Development of medicinal products Increased regulatory and scientific advice Dialogue with relevant stakeholders 11/6/2014 Regulatory science and innovation 20

21 Clinical-trial phases of scientific-advice requests ( ) 100% 90% 80% 70% 60% 4% 5% 61% 57% 62% 1% 50% 40% 30% 20% 10% 0% 21% 21% 24% 14% 18% 13% Phase I Phase II Phase III Phase IV 11/6/2014 Regulatory science and innovation 21

22 National vs. EMA/CHMP scientific advice? EMA/CHMP scientific advice Best regulatory/scientific expertise Predictive for the CHMP/CAT opinions Formal and expensive Questions concern mainly the late clinical develoment (Phase 3) 11/6/2014 Regulatory science and innovation 22

23 EMA scientific advices in 2012 co-ordinated by Fimea experts Biosimilars Immunomodulators ATMP Immunotherapy (cancer vaccines) Cytokine blocker 3 SMEs 11/6/2014 Regulatory science and innovation 23

24 National vs. EMA/CHMP scientific advice? National scientific advice (in Fimea) Wide scientific /regulatory view Affordable Less formal, more interactive Represents a national view 11/6/2014 Regulatory science and innovation 24

25 National scientific advice 2012 Biotech. proteins Chemicals Combination product ATMPs Blood product From phase 1 to 4, one SME 11/6/2014 Regulatory science and innovation 25

26 Drug development and Fimea Labor atory Preclinical Clinical trials Marketing authorisation application Idea translational gap National scientific advice EMA/CHMP scientific advice 11/6/2014 Regulatory science and innovation 26

27 Baby medicines welfare clinic a pilot project A new form of scientific&regulatory advice is needed for small companies companies from outside the ICH countries the very early phase of drug development baby medicines welfare clinic Discussion meeting without briefing books Short minutes, not to be used as a reference A follow up meeting is possible 11/6/2014 Regulatory science and innovation 27

28 Drug development and Fimea Labor atory Preclinical Clinical trials Marketing authorisation application Idea Medicines welfare clinic National scientific advice EMA/CHMP scientific advice 11/6/2014 Regulatory science and innovation 28

29 Medicines welfare clinic 2012 ATMP severe burns cell therapy - University ATMP cancer gene therapy - SME Novel radiotherapy cancer - SME ATMP - cancer gene therapy SME Biologicals biosimilar concept global pharmaceutical company Biological prophylactic immunotherapy - SME ATMP - stem cell therapy - SME Six domestic companies, one global pharmaceutical company All projects in very early phase 11/6/2014 Regulatory science and innovation 29

30 Future of European Scientific Advice EMA Earlier involvement of the Rapporteurs (scientific advice?) Adaptive or staggered licensing and scientific advice? National scientific advice CHMP/EMA scientific advice Product development 11/6/2014 Regulatory science and innovation 30

31 Biosimilar products in EU 19 Marketing Authorisation Applications (MAAs) reviewed (14 positive, 4 withdrawn, 1 negative) 12 biosimilars currently hold a valid marketing authorisation (1 somatropin, 5 epoetins, 6 filgrastims) Biosimilar MAAs currently under review (1 follitropin alfa, 2 infliximabs, 1 filgrastim) 11/6/2014 Regulatory science and innovation 31

32 Regulatory environment for biosimilars in EU Dir 2003/63 GL for comparability Dir 2004/27 Overarching GL Quality and Nonclinical/clinical GL Product-specific GLs GL on mabs First epoetin First somatropin First filgrastim 11/6/2014 Regulatory science and innovation 32

33 EU guidelines for biosimilars Defines principles Under revision General guidelines Quality Under revision Quality / Safety Efficacy Under revision Nonclinical Clinical In preparation: Follitropin alfa IFN-β Product class specific data requirements LMMH Insulin Somatropin GCSF Epoetin IFN-α Under revision Under revision mabs Nonclinical Nonclinical Nonclinical Nonclinical Nonclinical Nonclinical Nonclinical Clinical Clinical Clinical Clinical Clinical Clinical Clinical

34 Collaboration between EMA and FDA Good afternoon! We disagree! It is morning! 11/6/2014 Regulatory science and innovation 34

35 Click to edit Master title style Conclusion: Regulators, even the small agency in the North can support innovation! Thank you for your attention! Thanks to Dr Paula Salmikangas and Dr. Niklas Ekman for help in preparing this presentation 11/6/2014 Regulatory science and innovation 35