1 / 21

EMA -HTA scientific advice: the first 5 years

EMA -HTA scientific advice: the first 5 years. Jorge Martinalbo, Scientific advice Human Medicines R&D Support Division. Moscow, 2 nd Nov 2015. outline.

cleta
Download Presentation

EMA -HTA scientific advice: the first 5 years

An Image/Link below is provided (as is) to download presentation Download Policy: Content on the Website is provided to you AS IS for your information and personal use and may not be sold / licensed / shared on other websites without getting consent from its author. Content is provided to you AS IS for your information and personal use only. Download presentation by click this link. While downloading, if for some reason you are not able to download a presentation, the publisher may have deleted the file from their server. During download, if you can't get a presentation, the file might be deleted by the publisher.

E N D

Presentation Transcript

  1. EMA-HTA scientific advice: the first 5 years Jorge Martinalbo, Scientific advice Human Medicines R&D Support Division • Moscow, 2nd Nov 2015

  2. outline the views expressed are the personal views of the presenter and may not be understood or quoted as being made on behalf of or reflecting the position of the EMA or one of its committees or working parties. I have no financial relationships to disclose. • background • HTA-EMA scientific advice • procedure • experience, profile of requests • frequent topics & examples

  3. EMA structure & functions ACCESS DEVELOP • decentralisedbody EU • headquarters scientific secretariat and coordination (London > 1995) • network of national agencies from 28 EU countriesand > 5000 experts internal & external – scientific committees (multidisciplinary) & working parties

  4. EMA ‘regular’ scientific advice Regulators CTAs = national ph1 phase 2 phase 3 MAA EMA scientific advice & qualification • SAWP: multidisciplinary expert group (n>50) • focus phase 3 RCTs - also exploratory, SATs • population – all therapeutic areas • comparator, blinding • endpoints, SAP • safety • filing strategy: single pivotal, CMA • (post-authorisation safety & efficacy studies, pragmatic trials, registries, meta-analyses)

  5. effective access1 regulators – EU MA HTAs & payers – national P&R (5-30m) (1-10m) (4-19m) (5-19m) +1 year +2 year 1median times (+ range, months) from EC MA decision to final HTA recommendation / P&R decision and outcomes based on basket of 16 oncologic drugs authorised in EU 2012-2014

  6. differences?

  7. HTA & P&R frameworks CONTROLS hospital (in- vs. outpatient) vs. ambulatory • PRICE • (ex-)‘free’ • value-based • external referencing REIMBURSEMENT selective public financing NHS: positive lists + regional mechanisms (HTA) relative effectiveness added therapeutic value mixed cost-effectiveness economic budget impact

  8. early dialogue & dangerous liaisons

  9. multi-stakeholder dialogues EMA-HTA to discuss/align evidence requirements early in development so drug developerscan address information needs of both regulators and payers/HTAs 2 HTA, payers 1 Regulators P&R ph1 phase 2 phase 3 MAA EMA scientific advice & qualification + FDA 2013- SEED(Shaping European Early Dialogues) EMA-HTA scientific advice 2010- adaptive pathways pilot 2014- +10 0 +2y +4 +6 +8

  10. procedures N=57 SEED 2 2

  11. HTA involvement n HTAs/procedure median = 3 excl. SEED

  12. profile requests indications N=57 products

  13. procedural

  14. ‘70-day’ procedure pre-validation assessment HTA1 HTA2 ) ( HTA3 SAWP LoI BRIEFING DOC DISCUSS -2m -1m +1m +2m +3m START

  15. FAQs I population definition • in/exclusion criteria (homogeneous <confounders, variability vs. heterogeneous co-morbidities, ECOG PS2, co-meds) internal vs. external validity, representative • feasibility: orphan, stratified medicine comparators • vs. active control(s)? • ‘basket’ investigator’s choice? – global • effective use – medical diversity, GLs • posology recomm. variability • licensing & reimbursement status - availability

  16. FAQs II endpoints • patient-benefit clinical outcomes, threshold clinical relevance • surrogates, biomarkers • PROs / HRQoL – choice instruments, collection, analysis • short- vs. longer-term effects; time horizon – IAs, follow-up statistical methodology • SAP, adaptive designs, multiplicity / error control • adjustment for OS confounding next-line therapies/cross-over HTA-only • cost / resource utilisation, economic modelling, utilities

  17. squamous adenocarcinoma NSCLC stratification e.g. lung cancer ORR, DoR? PFS? OS? PROs? BM- BM+ chemotherapy chemotherapy targeted - coDx BM+ cis/carboplatin paclitaxel, docetaxel gemcitabine vinorelbine irinotecan etoposide not pemetrexed cis/carboplatin paclitaxel, docetaxel gemcitabine vinorelbine irinotecan etoposide + pemetrexed + bevacizumab + nintedanib • EGFRm erlotinib, gefitinib, afatinib • ALK+ crizotinib, ceritinib emerging off-label - emerging • BRAFm: vemurafenib, dabrafenib • HER2m: trastuzumab, pertuzumab • ROS1: crizotinib, ceritinib • RET: cabozantinib, vandetanib • MET: crizotinib, cabozantinib • […] immunotherapy targeted nivolumab + emerging

  18. Adaptive pathways pilot project oncology/total start 3/2014 as non-binding, safe-harbor pilot, prospectively planned systems approach to lifecycle incl. P&R and utilisation existing regulatory framework process/tools Criteria for candidate selection • preliminary evidence PoC, unmet need, but not necessarily a breakthrough • adaptation: early stage of development, plan includes iterative phases of evidence collection – evaluation – adapt license • RWDreal-world data (registries, post-A S&E studies, observational trials…), also ‘unconventional’ designs e.g. basket • stakeholders’ input (+HTAs/payers)

  19. pros & cons EMA-HTA SA? • dialogue: Sponsor-R/H, R-H, H-H as equal partners to identify and ideally solve critical divergences • experience procedure EMA SA (>3000, 20 years) • expertise: external clinical experts, patient involvement • flexibility choice of HTAs, topics for discussion, no eligibility restriction • confidentiality • comprehensive &constructiveF2F discussion 4h • commitment: written outcome • incentives: orphans, SME, ATMPs, paediatrics (fee reductions/waivers) • parallel vs. convergent? • no harmonised, joint written advice • constraints normative and methodological frameworks, interpretation • logistics, coordination, 4h F2F – advance booking • need for > plurality broader HTA representation • improve patient involvement • impact assessment pending

  20. Summary EMA benefit / risk HTAs & payers costs / relative E SINGLE standard for MAA evaluation approval decision EU + EFTA NATIONAL P&R 28+ methods, criteria, decisions dialogue – align evidence access faster and predictable conditional Acc EMA-HTA scientific advice scientific advice & qualification + FDA adaptive pathways pilot PRIME (BTD-like)

  21. jorge.martinalbo@ema.europa.eu

More Related