1. Slide 1 Encouraging innovation: the regulators perspective Bringing the best to healthcare through partnership and innovation IPHA, 1 st December 2011 Pat O’Mahony Chief Executive Irish Medicines Board

2. Slide 2 Some Key Challenges/Opportunities and Current Shared Topics  Balance of benefit and risk (harm) – Pharmacovigilance Regulations  New science and new therapies and Need to improve research and development and regulatory processes  Globalisation of the market – International regulatory collaboration  Demographics, disease burden and unmet needs - Scarce resources for health budgets – HTA?  Medicines Availability  Transparency, communication & information – Patient Engagement - Prescription Status Consultative Panel

3. Slide 3 New Science and New Therapies  New science and new therapies and Need to improve research and development and regulatory processes  Biomarkers – Critical Markers of Disease  Surrogate endpoints  Personalised Medicines  Nanotechnology - EU has decided on a definition of a nanoparticle - first region to do so

4. Slide 4 EMA Qualification of Novel Methodologies for Drug Development (1/8)  Common understanding important Scientists in universities, consortia and companies need to discuss with regulators early to achieve a common understanding on what is needed to qualify a novel methodology also in terms of regulatory approval The regulatory requirements should be up to date with the progress in science Question: Can this novel methodology be used as inclusion criterion or as an endpoint in a clinical trial?

5. Slide 5 EMA Qualification of Novel Methodologies for Drug Development (2/8)  Biomarkers – Briefing meetings since 2001 with EMA Innovation Task Force and the CHMP Pharmacogenomics Working Party  Novel methodologies – CHMP qualification June 2008: draft EMA guidance published for consultation on the qualification of novel methodologies including biomarkers, imaging techniques, new non-clinical models, new statistical approaches via the Scientific Advice Working party of the CHMP January 2009: Final guidance published

6. Slide 6 EMA Qualification of Novel Methodologies for Drug Development (3/8)  Qualification Advice and Qualification Opinion CHMP Qualification Advice on future studies and methodologies for further method development towards qualification, based on the evaluation of the scientific rationale and on preliminary data submitted. CHMP Qualification Opinion on the acceptability of a specific use of the proposed novel method in non-clinical or clinical studies, based on the assessment of submitted data obtained in studies already performed, not necessarily specific to one product.

7. Slide 7 EMA Qualification of Novel Methodologies for Drug Development (4/8)  Qualification team One Coordinator (SAWP or CHMP member) Minimum 4 experts, one a statistician (not bound to Coordinator country). We target inclusion of at least one external to the regulatory system, a key opinion leader One EMA Scientific Administrator

8. Slide 8 EMA Qualification of Novel Methodologies for Drug Development (5/8)  Other regulatory agencies Applicant is encouraged to apply in parallel to the EMA and FDA. The confidentiality agreement between the FDA and EMA makes it possible to have the procedure ongoing at the same time in both agencies. The agencies will communicate the assessment and meet with the Applicant together. It is envisaged to reach the same conclusions and experience so far confirms this. Applicant still has the choice to apply to one agency only.

9. Slide 9  Today and the future 21 procedures started 2 Qualification Opinions finalised (public after agreement with the sponsor at www.ema.europa.eu) One non-clinical: biomarkers for nephrotoxicity. One clinical: biomarkers in the cerebrospinal fluid as inclusion criteria for clinical trials in early Alzheimer’s disease. 4 Qualification Advices finalised (confidential to Applicants). EMA Qualification of Novel Methodologies for Drug Development (6/8)

10. Slide 10 EMA Qualification of Novel Methodologies for Drug Development (7/8)  What is new? – What is the ultimate goal? Scientific Advice Working Party / CHMP early involvement in the design of the strategy towards qualification of novel methodologies. Scientific Advice Working Party / CHMP commitment to evaluate the data obtained from the agreed studies and to provide a Qualification Opinion regarding the use of the method in R&D. Qualification Opinion part of the future Marketing Authorisation Application to support use of the novel methodology in the context of the development of a new product. Goal: speed up drug development, contribute to public health.

11. Slide 11 EMA Qualification of Novel Methodologies for Drug Development (8/8)  Reference document: Review titled New pathway for qualification of novel methodologies in the European Medicines Agency Authors: Efthymios Manolis, Spiros Vamvakas and Maria Issac (European Medicines Agency, London) Published in Journal Proteomics Clinical Applications. 2011, 5,1-8

12. Slide 12 Conference Critical Markers of Disease (CMOD) May 1 st and 2 nd 2012

13. Slide 13 New Science and New Therapies Advanced Therapies Much happening on regulatory front IMB is running a joint scientific conference with PDA (Parenteral Drug Association) Ireland Chapter, on Advanced Therapy Medicinal Products in July 2012, entitled “Making gene and cell therapy a reality” (subtitle “An efficient partnership between authorities and industry in Europe”) The conference will run for two days from July 10 th -11 th 2012, and will form a satellite event immediately before the prestigious ESOF 2012 multidisciplinary scientific congress on 11 th - 15 th July ESOF( European Science Open Forum) is the centerpiece of Dublin being designated as European City of Science for 2012

14. Slide 14 Some Key Challenges/Opportunities and Current Shared Topics  Globalisation of the market - International regulatory collaboration  How to ensure both strength and depth of expertise in regulators and efficient processes  Various actions - ICH, Summit Themes (2011 - Performance, Community Expectations, Transparency, Medical Devices, HTA engagement)  Demographics, disease burden and unmet needs - Scarce resources for health budgets – HTA?  Personalised Medicines and Drug device combination products  Medicines Availability  Transparency, communication & information – Patient Engagement - Prescription Status Consultative Panel

15. Slide 15 Encouraging innovation: the regulators perspective THANK YOU! Bringing the best to healthcare through partnership and innovation IPHA, 1 st December 2011 Pat O’Mahony Chief Executive Irish Medicines Board