Real world evidence studies: An integrated part of drug development Helsinki Pål Hasvold Medical Evidence Scientific Leader CV AstraZeneca

Transcription

1 Real world evidence studies: An integrated part of drug development Helsinki Pål Hasvold Medical Evidence Scientific Leader CV AstraZeneca Nordic-Baltic

2 The need for real world data in different phases in the life cycle of a drug How global RWE programs are set up within AstraZeneca Areas for development in the Nordic countries 2

3 Need for real world data in drug development? Preclinical studies Clinical trials Safety: (Ad hoc) monitoring PASS Pharma cology/ chemistry IND* Phase I Phase II Phase III NDA** Phase IV *Investigational New Drug **New Drug Application 2 4 years 2 6 months 3 6 years 1 3 years

4 Need for real world data in drug development and the life cycle of a drug? Preclinical studies Clinical trials Pharma cology/ chemistry IND* Phase I Phase II Phase III NDA** Phase IV *Investigational New Drug **New Drug Application RWE study program 2 4 years 2 6 months 3 6 years 1 3 years

5 RWE should be a fully integrated part of drug development Disease insights and Treatment reality How large is this patient population? How are they treated? Outcome/risk? Which are the high risk groups? What are the cost of treating/not treating them? RCT results into clinical perspective An RCT population vs. real life Who would benefit most of treatment? Drug utilization? Who are treated? For how long? Switch pattern? Disturbing signals? Outcome in a real world setting? Pathophysiology studies

6 Antiplatelet therapy in acute coronary syndrome Acute coronary syndrome: Myocardial infarction or unstable angina pectoris A P2Y 12 inhibitor is recommended, in addition to aspirin, for 12 months unless there are contraindications such as excessive risk of bleeds

7 Ticagrelor is indicated for the prevention of thrombotic events (for example stroke or heart attack) in people with acute coronary syndrome

8 Brilique (ticagrelor) Clinical trial program ACS Stable CHD with prior MI PAD Acute ischaemic stroke or TIA Type 2 diabetes at high risk of CV events n=18,624 n=21,162 n=13,887 n 9600 n 17,000

9 Reimbursement dossier Burden of disease xx is associated with a level of CV risk that is greater than in xx and carries a high burden due to CV and. More than 1 in x patients with xx suffer a CV event and almost 1 in y patients die within a year of diagnosis Current xx management and unmet needs There is a significant unmet medical need for effective xx therapy Economic burden of disease Key cost drivers in xx 9

10 APOLLO study programme: 5 individual studies in 4 countries encompassing >150,000 patients Countries USA UK Sweden France Databases Medicare HealthCore CPRD MINAP HES National registries EGB PMSI Details Demographics and health insurance claims Linked to death registry Age >65 years n=53,909 1 Demographics and health insurance claims Commercially insured years 2 n=13,492 2 Three linked datasets Longitudinal data Primary and secondary care n= Nationwide Longitudinal data Hospital discharge data linked to prescription register and death registry data n=77,976 1 Sample of national healthcare insurance data Hospital discharge data linked to death registry n=1764 1) Rapsomaniki E, et al. ESC Late Breaking Registry abstract 2014: In press; 2) DeVore S, et al. ISPOR poster 2014 CPRD, Clinical Practice Research Datalink; EGB, Échantillon Généraliste de Bénéficiaires (General Sample of Beneficiaries); HES, Hospital Episode Statistics; MINAP, Myocardial Ischaemia National Audit Project; PMSI, Programme de médicalisation des systèmes d'information (French National Hospital Database) *This differs from the N in the 4-country analysis (Rapsomaniki E, et al) due to harmonisation of the data

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12 Post myocardial insights: European Society of Cardiology 2015

13 PRECLUDE-MI study Cooperation between Swedeheart/UCR and AstraZeneca The aim of this study was to provide a registry-based analysis of recurrent MIs and whether these were associated to a previously stented lesion (culprit) or not (non-culprit)

14 Cumulative incidence at 8 years for first recurrent myocardial infarction related to non-culprit, culprit and indeterminate lesions Varenhorst et al. AHA 2016 Higher risk for non-culprit related recurrent MIs than culprit related recurrent MIs

15 Brilique (ticagrelor) Clinical trial program ACS Stable CHD with prior MI PAD Acute ischaemic stroke or TIA Type 2 diabetes at high risk of CV events n=18,624 n=21,162 n=13,887 n 9600 n 17,000

16 Do we need more or other types of documentation? Only two pages of inclusion and ex criteria's Wallentin L et al. N. Engl. J. Med. 2009;361:

17 ACS treatment in real life?

18 Initiation and persistence of dual antiplatelet therapy after acute coronary syndrome National hospital register National cause of death register National drug register ACS How are patients treated: Type of antiplatelet treatment? For whom? For how long? Are they switched? 18 months

19 Patients < 75 years Halvorsen et al. BMC Cardiovascular Disorders 2016 May 31;16:115

20 Halvorsen et al. BMC Cardiovascular Disorders 2016 May 31;16:115

21 Challenges in the Nordics

22 Requests for the future: 1) Drug treatments in hospital 2) Primary care data in good, qualitative and accessible format 3) Randomized trials with register follow-up

23 Primary care data All patients prescribed x or y between 71 Primary Care Centers in Sweden 43,575 eligible patients Swedish National Discharge Register 1,2 15,990 included patients Swedish Cause of Death Register 1. Martinell M et al. Ups J Med Sci 2012;117: Janson C et al. BMJ ;346:f3306

24 Conclusions RWE data/studies are a fully integrated part of drug development programs in AstraZeneca Important data for patients, clinicians, payers and AstraZeneca We are in the middle of the smørgåsbord However