Legemiddelindustriens bruk og behov av Real World Data Steinar Østerbø Thoresen MD PhD Leder FOU LMI og Medisinsk Direktør Abbvie
Agenda Disclosure Politiske signaler om samarbeid offentlig/industri RWE-hva er det? Registerdata i ulike faser av medikament utvikling Biomarker Koblinger til andre registre Konklusjoner og veien videre
Disclosure 2013- Medical Director Abbvie Norway 2013- Head R&D Group The Pharma Trade Org Norway 2006-2013 Head of Clinical Research, Medical Director Norway Denmark and Iceland GSK 1999-2013 Professor University of Bergen 1992-2006 Head National Cervical- and Breast-Cancer Screening, The Cancer Registry of Norway 1990-1991 Visiting scientist Harvard Medical School, Boston 1985-1990 Head of Registration, The Cancer Registry of Norway 1979-1984 Pathologist PhD Haukeland University Hospital
Viktige initiativer Helsedata som nasjonalt fortrinn
Den nye legemiddelmeldingen 2015
Viktige initiativer Komi t e e n anmoder regjeringen om å ta initiativ til et møte mellom aktuelle aktører for å drøfte interesse, behov og muligheter, herunder rammer og betingelser, for å legge til rette for helsedata som grunnlag for næringsutvikling. K omi t e e n støtter også at dette området innen helsefeltet har et ubrukt potensial og ønsker at dette skal være et næringspolitisk satsingsområde. Stortinget ber regjeringen utrede modeller og en infrastruktur for et trygt og etisk forsvarlig samarbeid mellom norske offentlige biobanker, helseregistre og industrielle aktører.
REAL WORLD EVIDENCE Evidence collected outside of the controlled constraints of conventional randomized clinical trials used to understand burden of illness, treatment patterns, patient behaviors and product performance in settings and populations that are representative of routine clinical practice
Helsedata er avgjørende for morgendagens legemiddelutvikling Ny behandling blir mer målrettet
Drug development
Why RWE? As the gold standard of clinical evidence, randomized clinical trial (RCT) data is used to inform decision making, however RCTs do not answer all questions, especially the ones related to real world clinical effectiveness and health economics 1 Real world data is important to adress relevant medical questions raised by medical community, regulators and pharma industry and is a unique opportunity to detect treatment events/outcomes in the real world setting. Health Technology Assessment (HTA) analysis of the pharmaceuticals is increasingly in need of real-world data to address aspects of clinical effectiveness and economic evaluation 2 Access with Evidence Generation (AEG) or conditional reimbursement models as well as new pricing schemes needs to be supported by real world data
Incidence/prevalence and survival Incidence and prevelance data are already collected by the Cancer Registry on high level. From research perspective there is a need to capture incidence and prevalence of disease for clinically relevant subgroups and also, divided by biomarker status and by region/hospital Outcome - overall survival, relapse free survival in general are in the registry but there is a need for further details about clinical subgroups and also, divided according to treatment regiment and/or biomarker status. HEOR/HTA related; opportunity to extract a matched patient subgroup as control group representing standard of care to use as comparator in HTA analyses when marketing permit is based on early phase data and soft endpoints
Treatment data Primary focus from an industry perspective is on drug based treatment but data on other treatments like surgery and radiotherapy on a less detailed level is off course of interest For drug based treatment central info will be: Drug name (INN) and brand Dosing scheme Treatment line: standard of care for patient groups (1.st, 2nd, 3de line etc) Drug survival: time and date start/end treatment Reasons for ending / changing treatment (pre-set categories e.g. loss of efficacy, side effects, type and level of side effects) Response to treatment For patients included in clinical studies study name, drug and other relevant info on treatment. Will enable long term follow-up of clinical study patients. For drugs used before marketing permit, e.g. in compassionate use programs, off label use, named patient basis registration of name of drug, response to therapy, side effects and treatment duration
Eksempel fra Italia; early access Treatment related data improving access in innovation AIFA Registry: a part of Italian reimbursement landscape Monitoring appropriate use of drugs according to approved therapeutic indications Assessing and tracking patient eligibility Evaluating utilization in clinical practice Collecting epidemiological data including safety profile Introduction of the AIFA system and Managed Entry agreements has massively improved patient access in Italy. Russo P. Mennini F.S., Siviero P., Rasi G. Time to market and patient access to new oncology products in Italy: a multistep pathway from European context to regional health care providers - Annals of Oncology 21: 2081 2087, 2010
SACT, UK The SACT initiative, is a national data collection programme designed to optimize patient outcomes and service provision for cancer chemotherapy in England. Key facts: 138 of the 142 Trusts are providing data returns in 2014 132,000 individual patient records, which cover chemotherapy treatment for all solid and hematological malignancies, including those in clinical trials SACT dataset is used for commissioning, benchmarking, individual appraisal and clinical research in real life setting. Dataset used for identification of patient flows, differences between supplied and administered drug volumes (data quality of SACT submissions and to engage manufacturers regarding optimizing usage to reduce wastage), capacity planning, and captures data to support the administration of Patient Access Schemes (to reduce the administrative burden in calculating financial rebates).
Biomarkers Possibility to quickly include markers based on new drugs under development in the registry Data on biomarkers relevant for diagnostic, prognostic or treatment related decisions HEOR/HTA - An overview of accessible tissue or datasets accessible for analyses as part of collaborative projects could be useful.
Combination of data across registries Short term: linkage of the cancer registry to other registries or data sources would be an important next step Long term: central data to support data from the cancer registry includes: -national bio-banks -national prescription registry -national patient discharge registry -price/tender results Examples of central registries of potential interest
Helsedata sin betydning for legemidlers utvikling og bruk Preklinisk Forskning Sykdomsmekanismer Molkylær biologisk innsikt Identifisering og validering av nye targets Forskning Identifisering av pasient sub-populasjoner Kliniske data muliggjør identifisering av subpopulasjoner av pasienter og forskning på deres specifikke karakteristika Utvikling Registerbaserte randomiserte kliniske studier Registerdata muliggjør store studier til lave kostnader og under mer realistiske forhold enn tradisjonelle kliniske studier Regulatorisk Monitorering gjennom legemiddelets livsløp Kontinuerlig monitorering av elektroniske pasientjournaler muliggjør tidlig oppdagelse av bivirkninger Markedsadgang Datagrunnlag for prisforhandling og refusjon Registerdata er basis for kunnskapsgrunnlag som basis for prisforhandlinger og refusjon
Konklusjon Industrien og offentlig myndigheter har alle behov for RWE, men vi må være klar over begrensningene Kreft (med verdens beste kreftregister) peker seg ut som et satsningsområde for Norge De politiske signaler er klare! Hva hindrer oss? Burde ikke industrien, Kreftregisteret, HF og HOD (SLV) gå sammen og lage den optimale RWE model?