Public private partnerships in support to regulatory sciences

Transcription

1 Public private partnerships in support to regulatory sciences The Innovative Medicines Initiative example M. Chlebus 3 October

2 Summary: Partnering on regulatory sciences Progress much slower when working in isolation/silos IMI pre-competitive scope, funding model and processes to manage conflicts of interest allow private and public parties (including patients and regulators) to collaborate IMI objectives contribute to regulatory science evolution Regulatory science questions are part of project objectives Regulatory affairs expertise in projects provided primarily by companies; engagement with regulators in capacity as advisors or project project partners or through EMA qualification advice Multiple companies share data and test new tools and solutions in real life development projects in parallel Examples: safety biomarkers, patient reported outcomes, platform trials, modelling and simulation, pragmatic trials, surrogate markers, patient preference elicitation

3 The Innovative Medicines Initiative: the largest public-private partnership for health research worldwide In total 5 billion until 2024 half from EU FP7 & Horizon 2020 R&D funding, half from Industry NO PUBLIC FUNDING FOR INDUSTRY EU FUNDING GOES ONLY TO PUBLIC PARTNERS 3

4 End-to-End approach: Accelerating Alzheimer s Research

5 IMI impact on pharma value chain DISCOVERY New lines of enquiry in drug development Identification of new hits and leads First human Beta cell line for diabetes research EARLY DEVELOPMENT Regulatory qualified safety biomarkers In silico predictive models Novel epigenetic mechanisms and early biomarkers for non-genotoxic carcinogenesis Normalised and structured data of about 8,000 legacy GLP toxicology reports about 100 predictive algorithms LATE DEVELOPMENT Development of clinical biomarkrs/endpoints, e.g.; for autism, sarcopenia, asthma, pain Development of antibiotics and Ebola vaccines and diagnostics Clinical trial networks and fast-fail cohorts (pediatrics, antimicrobial resistance, autism) PATIENT ACCESS & USE Continued dialogue on merits of outcomes based and adaptive models Integration of patient voice in benefit risk evaluations Definition of evidentiary standards for pragmatic trials Methodological standards in pharmacovigilance

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7 SPRINTT example Qualify biomarkers for the specific conditions of Physical Frailty & Sarcopenia (PF&S) Validate and implement a multimodal (diet + exercise) intervention against PF&S, to prevent clinical consequences Achieve scientific and regulatory consensus on target population, state and efficacy biomarkers, and clinical trial methodologies Develop a health economic model evaluating the PF&S intervention Realise an infrastructure enabling the creation of a Clinical Knowledge Hub (CKH) and patientcentered remote service delivery 7

8 To improve outcomes in Hematologic Malignancies, we are teaming with leading institutions across Europe

9 Regulatory impact examples: EU-AIMS (Autism) received several letters of support in Dec 2015 for clinical outcomes and 4 patient stratification methods SAFE-T received 2 letters of support in September 2016 for DILI and DIKI biomarkers PROACTIVE qualification for PROs capturing patients experience with physical activity (COPD) MARCAR evidence for ongoing ICH S1 guidance on carinogenicty discussion EUROPAIN evidence for Guideline on clinical development of medicinal (including patient stratification PROTECT - SmPC-ADR Database, Inventory of drug consumption databases, Integration of recommendations on Good Signal Detection for pharmacovilance and pharmacoepidemiology

10 Evolution of Real World Data in IMI Electronic Health Records for Clinical Research European Medical Informatics Framework GetReal pragmatic trials Big Data for Better Outcomes Next: biosensors/digital endpoints

11 For more information or to submit an idea Please contact ontent/ongoing-projects Submit your idea at: THANK YOU! T I T L E O F T H E P O W E R P O I N T 11