Presentation on theme: "Senior Scientific Project Manager"— Presentation transcript:
1Senior Scientific Project Manager From IMI to IMI2Hugh LavertySenior Scientific Project ManagerVilnius, 11th September 2014
2Declining R&D productivity The way in which pharmaceutical companiesdevelop new medicines is changingPharmaRegulatorsEU PricingGenericsHC ReformRising R&D costDeclining R&D productivityPatent cliff
3New approaches needed“Deciphering the complexity of human diseases and finding safe, cost-effective solutions that help people live healthier lives requires collaboration across scientific and medical communities throughout the health care ecosystem.Indeed, we must acknowledge that no single institution, company, university, country, or government has a monopoly on innovation.”
4Innovative Medicines Initiative: Joining forces in the healthcare sector The biggest public/private partnership in Life Science aiming to:Make drug R&D processes in Europe more innovative and efficientEnhance Europe’s competitivenessAddress key societal challengesFeatures:1:1 funding, joint decision makingAll EU funds go to SMEs, academia, patient organisations and regulatory agenciesLarge pharmaceutical industry, represented by EFPIA, contributes in-kind
5How it works 18 weeks 9 weeks 6 weeks SMEs Academic research teams EFPIAIMITopic Definition& LaunchApplicant ConsortiaEoI Submission1st ranked EoI + EFPAFull Project Proposal SubmissionGoverning Board approvalSignatures & project kick-offHow it worksSMEsAcademic research teamsHospitalsStep 1Step 2Step 3Step 4Step 5TheConsortiumRegulatory authoritiesPatients’ organisationsCall Launch1st ranked EoI SelectionGB approval of 1st ranked FPPnegotiations startSignatures & project kick-off18 weeks9 weeks6 weeks
6714 academic & research teams The IMI communityCalls 1-846 projects> 6000 researchers23 patient org.410EFPIA teams14 regulators714 academic & research teams135 SMEsregulators on board of12projects50% of projects have regulatory authorities representatives in Scientific Advisory Boards61% of projects reported some form of PATIENT involvement
11Implementation of project results inside industry Making a differenceImplementation of project results inside industryProjectAreaResults descriptionIMIDIAdiabetesThe human beta cell line EndoC BetaH1 has been validated by Endocells and 3 pharma partners confirming their initial insulin secretion capacity. These cells have been successfully transferred as a research tool for drug discovery to industrial partners.DDMOREknowledgemanagementSeveral drug/disease models identified by DDMORE are adopted or further developed inside the industry.eTRIKSAdoption of the eTRIKS results, TransMART system deployments in 5 pharmaceutical companies.EUROPAINChronic painPreclinical model of spontaneous pain in rodents has been developed, standardized, validated, and is already used for internal decision making in the drug development process.The ultraviolet B (UVB) pain model has also started to be used for in house R&D.
12Impact on regulatory framework ProjectAreaResults descriptionPROactiveCOPDQualification Advice completed at the EMAEU-AIMSautismStarted EMA formal scientific advice procedure for qualification of 5 biomarkers in ASDeTOXdrug safetyProvided an update on the eTOX database and the prediction system to the CHMP Safety Working Party (SWP) at EMA. Scientific Advice Procedure was initiated.MARCARcancerHas developed new biomarkers, technologies, and alternative test systems that help explain or predict animal and/or human carcinogenic pathways and mechanisms for non-genotoxic carcinogenesis. This will provide enhanced scientific rationale for Carcinogenicity Assessment Document (CAD) submissions, with potential impact for ICH S1 carcinogenicity testing guideline revisions.Safe-TDeveloped and now progressed towards an aligned EMA/FDA qualification a set of novel safety biomarkers for drug-induced kidney, liver, and vascular injury.DDMOREknowledgemanagementIn May 2012 an advisory meeting with EMA and FDA representatives was held. Through a Modelling Review Group , DDMoRe is in regular contact with both the EMA and FDA regarding the qualification of the content of the project’s Model Library.
1318.4% 15% SME participation in IMI projects (up to 8th Call) Total IMI commitment€ 723 millionTotal received by SMEs€ 133 million% SME18.4%Total IMI participations886Total SME participations13515%
14SME success stories SME involved in SAFE-T project “Thanks to IMI our company went from 6 to 50 employees.Now we are ready to go to further expand.”SME involved in IMIDIA project –“1st product released to the market in 2013 – IMI was instrumental in validation of the first cell line product, 2nd product release planned this year, 3rd diagnostic product in development.In preparation: a new patent filing to protect technologies for the creation of third generation human beta cell lines.SME involved in PharmaCog project“We are developing a blood panel for AD for diagnosis, stratification and companion diagnostics in AD. The Panel was tested on 300 patients in IMI project”SME involved in eTOX project“We have developed in silico models for predicting toxicity, which were validated by pharmas in eTOX. Now we have signed a contract with one of the companies to use our models in house.”
15Promoting patient involvement IMI makes efforts to enhance patient centric approachPatient dedicated workshopsInvolving patients at all levelsProviding forum for discussionIMI best practice examples:EUPATIU-BIOPREDPROactive
16For patient-centric R&D more trained patients are needed Competent authoritiesPolicy makersTrial protocol design, informed consent, ethical review, marketing authorization, value assessment, health policyPublicResearch subjectInfo providerAdvisorReviewerCo-researcherDriving forceClinical ResearchResearch Ethics CommitteesHTA agencies & committees
17Paradigm shift in empowering patients on medicines R&D Key European initiative to provide objective, credible, correct and up-to-date public knowledge about medical research Will build competencies & expert capacity among patients & public Will facilitate patient involvement in R&D to collaborate in academic research, industry research, authorities and ethics committees
18Key collaborative activity areas: CollaborationKey collaborative activity areas:Diabetes, CNS disorders, Tuberculosis, Patient Reported Outcomes, Cancer, Preclinical Safety and Education & Training.IMI signed horizontal agreements with:Critical Path, Juvenile Diabetes Research Foundation as well as with Clinical Data Interchange Standards Consortium.
19Better Science = Better Decisions The measures of successSUCCESSNew models developed & publishedSetting new standardsIn house implementation by industryImpact on regulatory guidelinesBetter Science = Better Decisions
20www.europeanleadfactory.eu nd4bb-enable.eu IMI’s drug discovery platformsTargetscreeningHit-to-leadLead-to-candidatePreclinicalPhase IPhase IIPhase IIIEuropean Lead Factory Focus:identification of new hitsELF Budget:€92.0m EFPIA in-kind€80.0m IMI JU‘Qualified’hitEuropean Lead FactoryND4BB Drug Discovery PlatformLeadClinicalCandidatePhase 1-readyInsert web addresses for applicantsnd4bb-enable.euENABLE Budget:€26.0m EFPIA in-kind€58.9m IMI JUENABLE Focus: to move promising hitsinto early clinical development
22The Evolution of IMI: From bottlenecks in industry – to bottlenecks in Industry and Society Make Drug R&D processes in Europe more efficient and effectiveand enhance Europe’s competitiveness in the Pharma sectorIdea generationBasic researchand non-clinicaltestingHuman testingRegulatoryApprovalHTA andPharmacovigi-lanceDailyMedicalpracticeOriginally designed as a technology platform to resolve scientific bottlenecks in drug discovery and development to reduce attrition rate, the research agenda of IMI evolved to also address main challenges for society.IMI agenda was broadened to include all stages of medicines development to translate more effectively invention into innovation.As a consequence, IMI directly addresses a number of European and national priorities/policies:Active and Healthy AgeingAntimicrobial Resistance and infectious diseases (diagnostics and treatment)PharmacovigilancePersonalised MedicinesImpact of chronic diseasesMental healthBeyond resolving scientific bottlenecks and contributing to resolve significant healthcare challenges, the endavour of IMI2 is to translate scientific results into standards of care to have a real beneficial impact on patients.Primary focus ofearly IMI calls2007 SRAShift to also addressing challenges in in society and healthcare2011 SRAIMI 2includes real lifemedical practice2013 SRA
23The Vision for IMI2 – The right prevention and treatment for the right patient at the right time effectiveeffectivenot effectiveAdverse eventsTrial and ErrorDxTestBnot effectiveBiologically heterogeneous patient populationCadverse eventse.g. biomarkerInformation based treatment decisionsvsGraphic adapted from C. Carini, C. Fratazzi, Eur. Pharm. Rev. 2008, 2, 39-45
24Science is driving advances in diagnosis: breast cancer is actually 10 different diseases Thursday April“A landmark study has reclassified the country’s most common cancer in breakthrough research that could revolutionise the way we treat breast tumours… scientists found breast cancer could be classified into 10 different broad types according to their common genetic features.”
25Objectives of IMI2 – what the Regulation says increase the success rate in clinical trials where possible, reduce the time to reach clinical proof of concept in medicine developmentdevelop new therapies for diseases for which there is a high unmet need and limited market incentivesdevelop diagnostic and treatment biomarkers for diseases clearly linked to clinical relevance and approved by regulators;reduce the failure rate of vaccine candidates in phase III clinical trials through new biomarkers for initial efficacy and safety checks;provide support for the development of tools, standards and approaches to assess efficacy, safety and quality of regulated health products.
26The premisesAlignment with Horizon 2020 objectives of the Health challengeAddressing healthcare priorities identified by the WHO 2013 report on priority medicines for Europe and the world Strategic Research Agenda aimed at progressing the vision of personalised medicines, for both prevention and treatmentCollaboration across sectors to harness all knowledge and technologies which can contribute to IMI2 vision - diagnostics, imaging, IT, medical devices, …
272013: WHO report on priority medicines for Europe and the World: societal challenge reflected in the IMI2 SRATherapeutic Areas in IMI2 SRA(no priority order)
28Drive change in delivery of medical practice IMI2: Major Axes of ResearchBiomarker identification/validation(precision medicine)Innovative methodologies toevaluate treatment effectReclassification of diseaseby molecular meansInnovative clinical trial paradigmsTarget & Biomarker Identification(safety & efficacy)Adoption of innovative clinicaltrial designsTarget Identification andvalidation(human biology)European HealthPrioritiesBenefit/Risk AssessmentDeterminants of drug /vaccineSafety and efficacyInnovative MedicinesPatient tailored adherence programmesHealthcare delivery: focus on the treatment programmes not just the medicineInnovative drug deliverymethodologiesManufacturing for personalisedmedicinesDiscovery and Developmentof novel preventative andtherapeutic agentsInnovative adherenceprogrammesDrive change in delivery of medical practice
29Strategic Research Agenda Comprehensive framework for a 10-year programmePrepared with input from 80+ organisations (internet and targeted)Project ideas from industry and third parties will be screened against it
30IMI2 - Broad participation to be able to set ambitious goals IMI is evolving, with a stronger focus on the needs of patients and society and with simpler rules and procedures Evolution in scientific focus • Stronger focus on needs of patients and society, including unmet needs • Increased emphasis on improving patient access to innovative medicines (in addition to medicines development) • Focus on personalised medicine (the right treatment for the right patient at the right time)
31IMI2 - Broad Participation to achieve ambitious goals: Bigger budget: 3,45 Billion Euro, equally shared by EU and industryNot limited to EFPIA members: open for other industries / companies, which can contribute to the PPP goals (Healthcare IT, medical devices,…) giving them the opportunity to establish their own projectsThe principle of large companies providing an inkind contribution matched by IMI funding for public beneficiaries will be retained.
32IMI2 - Broad Participation to achieve ambitious goals: Specified Budget: 225 million Euros reserved for non-EFPIA led projects (to be matched by inkind contributions)Objectives, deliverables and timelines determined by the company(ies) proposing the projectInkind contribution determined by the company(ies)Once approved by IMI’s Governing Board the Programme Office will launch a call for proposals to identify public partners for the projectThe call process and review of submitted proposals will be independent of the company(ies)
33The Role Of The Programme Office A neutral broker:To implement programmes and activities in the common interest of all stakeholdersTo monitor the use of public funds and industry investmentTo guarantee fair and reasonable conditions for optimal knowledge exploitation and disseminationTo facilitate the interaction between stakeholders, including Intellectual Property agreementsTo actively communicate and promote IMI and its activities
34Submission date: 12 November 2014 IMI2: The First CallTwo topics:Translational approaches to disease modifying therapy of type 1 diabetes mellitus (T1DM)Discovery and validation of novel endpoints in dry age-related macular degeneration and diabetic retinopathySubmission date: 12 November 2014
35Translational approaches to disease modifying therapy of type 1 diabetes mellitus (T1DM)Vilnius, 11th September 2014
36Translational Approaches To T1DM: Background A chronic disease affecting worldwide around 17 Million people and with highest incidence rate in Europe ( ~ 22 / / year), with major regional differences.The incidence of childhood T1DM is reported to be rising rapidly worldwide, especially in the under 5 year old age group.T1DM is generally seen today as an autoimmune disease, but its cause is unknown (genetic susceptibility, diabetogenic trigger(s) and/or exposure to a driving antigen).The disease is currently not preventable and no cure is available. The only available pharmacotherapy for T1DM patients is the lifelong injection of insulin.
37Translational Approaches To T1DM: Aims and Objectives Better Disease Biology and Translational Medicine (Target & Biomarker Identification)Generation of a high quality and comprehensive European network of clinical and translational research centres (providing a prospective clinical trials database for T1DM) including at risk and early T1DM patients.Establishment of systematic large-data repository enabling extensive cross functional data mining and integrated data analysisPhenotypical characterization (in silico based on medical records as well as active through experimental medical studies)Systematic prospective and retrospective launch of broad “–omics” characterization of human biological samplesDevelopment and characterization of the most appropriate preclinical T1DM model(s) for discovery of novel clinical therapies.
38Translational Approaches To T1DM: Aims & Objectives Innovative clinical trial paradigms for preventative and disease modification trials in T1DM.Development of standardized entry criteria and endpoints for T1DM trials (both metabolic and immune profiles) with participation of patient advocacy groups, and regulatory authorities.Implementation of the use of electronic data capture devices to collect an array of “real world data”Testing and development of novel bio-statistical methodologies applicable to new compositions of relevant end points for T1DM clinical trials.Evaluation of novel mono- and combination approaches (i.e. combining multiple immune modulatory approaches, immune cell migration modification, immune tolerance inducers, β-cell enhancing therapeutics) in people with T1DM.
39Translational Approaches To T1DM: Key Deliverables An improved understanding of the immunological and beta cell biology aspects of T1DM to disentangle its heterogeneity both in at risk and early diagnosed patients and for staging participants in future T1DM clinical trials.The development of novel and relevant endpoints & readouts for T1DM clinical trial based on clinical & standardised molecular “real world data” obtained from T1DM patients, and on the application of novel bio-statistical methodologies.Pre-clinical T1DM models with improved translational value.Improved understanding of the human T1DM disease biology and optimised clinical trial setting to allow testing novel mono- and combination approaches in T1DM.
40Translational Approaches To T1DM: EFPIA PARTICIPANTS AND ASSOCIATED PARTNERSSanofi (coordinator), Juvenile Diabetes Research Foundation (JDRF) (co-coordinator), Novo Nordisk, Eli Lilly, GSK, Helmsley Charitable Trust.DURATION OF THE PROJECTThe indicative duration of the project is 84 month (7 years).BUDGETEFPIA and associated partners: EURIMI2 JU: EURTotal: EUR
41Translational Approaches To T1DM: APPLICANT CONSORTIUMAcademic endocrine clinics and associated supporting departmentsBasic, translational, and clinical researchers from the fields of T1DM autoimmunity and β-cell biologyDrug discovery and medical staff in Pharmaceutical Industry and Small and Medium size EnterprisesHands-on data base specialists and big data managersPatient organizations/representativesExperts in regulatory science and health technology assessment preferably representing European health authorities.The project will be expected to establish a T1DM Patient Advisory Committee
42Translational Approaches To T1DM: Suggested Work Plan A plan for interactions with Regulatory Agencies/Health Technology Assessment bodies with relevant milestones and appropriate resource allocation should be includedSynergies with other EU and global initiatives, including IMI projects
43Discovery and validation of novel endpoints in dry age-related macular degenerationand diabetic retinopathyVilnius 11th September 2014
44Novel Endpoints For Retinal Diseases Retinal diseases among leading causes of blindness worldwideAge-related macular degeneration (AMD): Early form reported to occur in 30% of the population of 75 years and above (over 50% by age 80); late form in % of the population over 70 years Approximately 93 million affected by diabetic retinopathy (DR) in 2010Limited treatment options for dry form of AMD or DRMajor development hurdles: lack of suitable endpoints for early exploratory and pivotal clinical trials, lack of predictive markers and models
45Novel Endpoints For Retinal Diseases: Aims & Objectives To evaluate novel endpoint candidates for dry AMD and DR:technical, medical and health economic appropriatenessbridging preclinical and clinical studies.Methods in scope:Visual function testing beyond Best Corrected Visual Acuity (BCVA)ElectrophysiologyImaging methods to assess retinal structureSoluble and genetics biomarkersPatient reported outcome tools and Quality of Life-related endpointsA combination of these methods
46Novel Endpoints For Retinal Diseases: Key Deliverables Generation of robust data resulting from retrospective and/or prospective studies as basis for discussion of regulatory acceptability of the endpoints for future clinical programmes.It is expected that the proposed research program delivers data on: Technical evaluation of methods (validity, repeatability, reliability, interpretability, translatability and acceptability by patients)Development of novel methods and toolsClinical validation of methods/tools in patient studies for dry AMD & DRCollection of biomarkers for selection of high risk populationsSynergies between dry AMD and DR vs condition-specific aspects
47Novel Endpoints For Retinal Diseases: EFPIA PHARMA PARTICIPANTS AND OTHER PARTNERSBayer HealthCare (coordinator), Sanofi, Novo Nordisk, ZeissDURATION OF THE PROJECTThe indicative duration of the project is 60 month (5 years).BUDGETEFPIA and associated partners: EURIMI2 JU: EURTotal: EUR
48Novel Endpoints For Retinal Diseases: Setting-up & running of studies required to meet topic’s objectivesMultidisciplinary applicant consortium with a track record ofClinical expertise in ophthalmologyClinical research experienceAccess to patients and databasesPublic health expertiseHealth economic expertiseUnderstanding of pre-clinical models in ophthalmologyBiomarkersData managementRegulatory, ethics, patients and project management
49Novel Endpoints For Retinal Diseases: Suggested Work Plan Architecture for the full proposal to be suggested by the Applicant consortiumIntention to set-up of an Advisory panel to the Consortium comprising payers, regulatory agencies and other relevant expert advisorsPlan for interactions with Regulatory Agencies/Health Technology Assessment bodies expectedSynergies with other EU and global initiatives, including IMI projects
50IMI2 Info Day Crowne Plaza Hotel, Brussels, Tuesday 30 September 2014 Workshops and presentations of topics by the topic writersOverview of IMI 2 funding and intellectual property (IP) rulesTips on applying for funding under IMI 2Networking opportunitiesIMI staff on hand to answer questionsWe warmly encourages small and medium-sized enterprises, mid-cap businesses, patient organisations, regulatory authorities, academic teams, industry, hospitals and other organisations50