Towards a 21 st Century Regulator s role EMA Early Access Toolbox

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1 Towards a 21 st Century Regulator s role EMA Early Access Toolbox Bulgarian Presidency Conference on Health as the Real Winner: Presidency Conference on Options to Provide Better Medicines for All 6 March 2018, Sofia Zaide Frias Head of Human Medicines Evaluation Division, EMA An agency of the European Union

2 21 st Century Regulator s Role is about connecting the dots and building right links Facilitate development and access to medicines Protects human and animal health Building a common Vision 1 Evaluates applications for Marketing authorisation Path towards a 21st Century Regulator's role; AESGP Conference with Heads of EU Medicines Agencies, Zaide Frias Provides information to healthcare professionals and patients

3 Facilitating research and development EMA Early access toolbox Medicines & Technology Horizon scanning Innovation Task Force Business Pipeline Research & Development support your name here SA /Parallel HTA Orphan /Paediatric ATMP/SME EU Regulatory pathways PRIME/Adaptive pathways/best evidence generation Cost of Development Incentives/Innovative Medicines Initiative (PPP)/ Horizon 2020/ 2 Towards a 21st Century Regulator's role; Health as the Real Winner, Zaide Frias

4 EU Regulatory Pathways PRIME PRIORITY MEDICINES Adaptive Pathways Multiple Sources >160 eligibility requests 34 granted Single Source 3 Towards a 21st Century Regulator's role; Health as the Real Winner, Zaide Frias Eichler HG et al. Clin Pharm & Ther 2012

5 Facilitate access to medicines Time difference vs. EC approval date (months) INN (trade name) Indication NICE SMC GBA HAS AIFA axitinib (Inlyta) RCC vismodegib (Erivedge)* BCC pertuzumab (Perjeta) mbc HER ziv-aflibercept (Zaltrap) CRC enzalutamide (Xtandi) CRPC bosutinib (Bosulif) CML regorafenib (Stivarga) CRC cabozantinib (Cometriq)* MTC ponatinib (Iclusig) CML, ALL Ph pomalidomide (Pomalyst) MM T-DM1 (Kadcyla)* mbc HER Ra 223 Cl2 (Xofigo)* CRPC dabrafenib (Tafinlar) MEL BRAFm afatinib (Gilotrif) NSCLC EGFRm obinutuzumab (Gazyva) CLL *first in class median Towards a 21st Century Regulator's role; Health as the Real Winner, Zaide Frias Martinalbo J et al. Ann Oncol. 2015

6 Experience with parallel regulatory/hta discussion on evidence generation plans Number of parallel advice + Level of alignment N * + advice through parallel scientific advice and early dialogue/seed * to September 2017 Level of agreement (HTABs vs. regulators; N = 31 procedures): full agreement partial agreement disagreement. British J Clin Pharm, Volume 82, Issue 4, pages Towards a 21st Century Regulator's role; Health as the Real Winner, Zaide Frias 5 October 2017

7 The Digital Revolution in Medicine is taking place now From Chatbots to Artificial Intelligence From Big data to the Health Cloud New role for regulators

8 Science and technology is advancing at exponential pace Modelling & Simulation Extrapolation Data Digital health and wearable technology Personalised medicines/ Biomarkers Gene therapy/ cells and tissues based products

9 Technologies which could change our lives: potential impacts & policy implications (Source EP STOA s) Wearable technologies: From physical electronic devices to new types of 'smart fabrics', the reasons for wearing our clothes are changing. How will this change our data-sharing habits and the way healthcare is delivered? Health Apps and their evaluation and regulation CT data capturing software / home reporting programs Smart home technologies: The Internet of Things now increasingly includes electronic devices operating in our homes. How will our everyday behaviours and personal relationships change as a result? (ENISA) Integrated Health and Health Care reporting / monitoring early warning signals to prevent longer / severe hospitalisation Towards a 21st Century Regulator's role; Health as the Real Winner, Zaide Frias

10 Scientific advancement and changing scene of medicines development New competences are required for regulatory and public health systems, both for evaluation and delivery to patients.

11 Borderline products (MP-MD) / Novel technologies 10 Towards a 21st Century Regulator's role; Health as the Real Winner, Zaide Frias

12 From Regulatory Optimisation to Digital transformation Novel enabling technology development Utilisation for Regulatory Processes

13 Need to widen the collaborations horizon 12 Path towards a 21st Century Regulator's role; AESGP Conference with Heads of EU Medicines Agencies, Zaide Frias

14 Pursues regulatory optimisation Path Towards a 21 st Century Regulator s role Embraces digital transformation Develops new competences 13 Path towards a 21st Century Regulator's role; AESGP Conference with Heads of EU Medicines Agencies, Zaide Frias Interacts with new players

15 Further information Zaide Frias, Head of Human Medicines Evaluation Division European Medicines Agency 30 Churchill Place Canary Wharf London E14 5EU United Kingdom Telephone +44 (0) Facsimile +44 (0) Send a question via our website Follow us

16 Back up slides: PRIME builds on existing Regulatory tools 15 Prime and support to Early Access; DIA EuroMeeting 2016; Zaide Frias

17 Scientific advice Sponsors prefer early interactions Earlier SA is associated with higher MAA success rate Compliance with SA recommendations on clinical trial design associated with o Higher MAA success rate o Less major objections o Shorter MAA procedure 16 Hofer M.P., Jakobsson C., Zafiropoulos N., Vamvakas S., Vetter T., Regnstrom J., Hemmings R.J., Regulatory watch: Impact of scientific advice from the European Medicines Agency, Nature Reviews Drug Discovery, Vol 14(5), pp

18 Accelerated assessment 25 Accelerated assessment requests In 2017, 58% of Accelerated assessment timelines were maintained till Opinion Robust decision making under accelerated timelines requires a mature submission, which should be subject to pre-filing discussions Requests for accelerated assessment rejected Requests for accelerated assessment accepted 6 10 Reasons for reverting to standard timelines during the MAA evaluation, include: Major clinical objection questioning the clinical relevance of the effects Numerous major objections including need for reanalysis of efficacy data Major objection on adequacy of extrapolation Significant quality major objection Critical GCP issues identified in inspections Need for a GMP inspection 17

19 Conditional Marketing Authorisation Overview of Conditional marketing authorisations by year of granting and current status Importance of early dialogue and prospective planning 18 DLP 31 Dec 2015

20 Regulatory and HTA Interactions 19 Eichler HG et al. Nat Rev Drug Disc 2010; 9: