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A REVIEW OF PATIENT REGISTRIES IN HEART FAILURE ACROSS EU-5 COUNTRIES Jatin Gupta, Manu Sehgal, Palvi Gupta PAREXEL 9th November 2015 CONFIDENTIAL © 2015 PAREXEL INTERNATIONAL CORP. AGENDA A REVIEW OF PATIENT REGISTRIES IN HEART FAILURE ACROSS EU-5 COUNTRIES © 2015 PAREXEL INTERNATIONAL CORP. / 2 • Background and objective • Approach and methodology • Results • Conclusion and limitations CONFIDENTIAL BERLIN, GERMANY BACKGROUND AND OBJECTIVES © 2015 PAREXEL INTERNATIONAL CORP. / 3 CONFIDENTIAL BACKGROUND HEART FAILURE BURDEN OF HF REGISTRIES • Heart failure (HF*) is a complex clinical syndrome of symptoms and signs that suggest the efficiency of the heart as a pump is impaired. It is caused by structural or functional abnormalities of the heart (NICE CG108) • HF is a global public health problem affecting an estimated 26 million worldwide (Ambrosy 2014). The prevalence of HF in Europe is ~1-2% (France 2.2%, UK 1.3%) (Cowie 2015) • Registries are prospective, observational cohort studies of patients who have a particular disease and/or are receiving a particular treatment or intervention. It involves prospective data collection of clinical, economic, and PRO* information that rely on realtime data capture (Garrison 2007) • HF is a chronic condition predominantly affecting people over the age of 50 years and its incidence and prevalence increases with age (NICE TA314) • Acute HF is complex clinical syndrome characterized by a rapid change in the signs and symptoms of HF, resulting in a need for urgent therapy, and is often life-threatening (Dahlstrom 2010) • Chronic HF is associated with high mortality and morbidity rate, decreased quality of life, and rising healthcare costs in Europe and globally (Dahlstrom 2010) • HF accounts for 1-3% of all US and European hospital admissions (Cowie 2015) • Almost 25% hospitalised patients are re-hospitalised for HF within the 30- day post discharge period, respectively (O’Connor 2010) *HF: Heart Failure; PRO: Patient Reported Outcomes © 2015 PAREXEL INTERNATIONAL CORP. / 4 CONFIDENTIAL • Patient registries are a good source of evidence for clinicians, disease organizations, health technology agencies, health insurers/payers, regulatory authorities requiring realworld data for evidencebased decision making (Garrison 2007) RATIONALE FOR THIS REVIEW RCTs* are regarded as the gold standard for clinical decision making purposes. However, data from RCTs often have limited external validity (generalisability) for the following reasons: • Highly selected patient populations • Often limited to specific geographic settings • Most data pertain to hospitalised patients • Registries and surveys in patients with acute and chronic HF* can provide a longitudinal view of the whole clinical story of patients with HF including the acute episodes and the consequent changes in the clinical conditions and in the management strategies • Many registries with real-world data on HF patients exist but a comprehensive review to help understand the scope, availability, and quality of collected data is currently lacking The objective of this review was to systematically identify and assess key characteristics of HF registries reporting patients’ data across the EU-5 countries *HF: Heart Failure; RCTs: Randomised Controlled Trials © 2015 PAREXEL INTERNATIONAL CORP. / 5 CONFIDENTIAL BERLIN, GERMANY APPROACH AND METHODOLOGY © 2015 PAREXEL INTERNATIONAL CORP. / 6 CONFIDENTIAL HF REGISTRIES WERE IDENTIFIED THROUGH A SYSTEMATIC REVIEW OF THE PUBLISHED LITERATURE AND INTERNET SEARCH Searching of databases • HF* registries were identified through a systematic search of Embase® and MEDLINE® databases using embase.com interface Key inclusion criteria • Registries with data on HF patients from EU5* countries (France, Germany, Italy, Spain, and the UK*) and patient counts ≥1000 were included Analysing evidence from registry websites/publications • Registry website and publications in peer-reviewed journals were assessed for availability of information on basic registry details, patient characteristics, disease management, resource use, and treatment outcomes *EU5: European Union Countries; HF: Heart Failure; UK: United Kingdom © 2015 PAREXEL INTERNATIONAL CORP. / 7 CONFIDENTIAL FOLLOWING DATA VARIABLES WERE SOUGHT FOR EACH IDENTIFIED REGISTRY Category Data variables Basic registry information Region, acronym, full name, website, country, year established and data collection period, current status (active or inactive), patient group, objective, funding agency Patient characteristics Age, gender, race/ethnicity, comorbidities Disease management Diagnosis and staging of HF*, diagnostic procedures and tests, treatment procedures, pharmacological interventions Resource use Hospitalisations, ER* visits, outpatient visits, primary care visits, length of stay, cost Treatment outcomes Laboratory results, quality of life (scale/instrument used), adverse events reported, survival data/all-cause/HF-specific mortality rates *ER: Emergency Room; HF: Heart Failure © 2015 PAREXEL INTERNATIONAL CORP. / 8 CONFIDENTIAL BERLIN, GERMANY RESULTS © 2015 PAREXEL INTERNATIONAL CORP. / 9 CONFIDENTIAL REVIEW IDENTIFIED 23 REGISTRIES WITH >1000 PATIENTS Number of included patients varied from 1037 to 25,000 with the average follow-up ranging from 3-53 months No. of HF patients Name Acronym Acute Heart Failure Global Registry of Standard Treatment Clinical Practice Research Datalink Disease management program in heart failure in Lorraine (Insuffisance Cardiaque en Lorraine) Epidemiology of Acute Heart Failure in Emergency Registry EuroHeart Failure Survey I EuroHeart Failure Survey II European Society of Cardiology-Heart Failure Long-Term Registry European Society of Cardiology-Heart Failure Pilot Survey Evidence based Treatment in Heart Failure registry German Competence Network Heart Failure database Global Registry of Acute Coronary Events Global Registry of Acute Coronary Events 2 HELUMA heart failure registry Italian Network on Heart Failure Outcome registry Italian Survey on Acute Heart Failure Realise Atrial Fibrillation Registry Schlahf registry ScreenIng of todays patIents with Chronic systolic heart failure Spanish National Registry on Heart Failure Spanish REDINSCOR registry (Clinical and Preclinical Heart Failure Research Network Registry) The prospective observational Longitudinal RegIstry of patients with stable coronary artery disease Trieste Registry of CV Diseases VIDA-IC study ALARM-HF CPRD ICALOR 4,953 8,404 25,000 EAHFE-1,2,3 EHFS I EHFS II ESC Heart Failure ESC-HF Pilot EVITA-HF CNHF GRACE GRACE 2 HELUMA IN-HF IS-AHF RealiseAF SchlaHF INDICATE RICA REDINSCOR 5,845 10,701 3,580 19,211 5,118 3,082 12,756 >5,000 >5,000 2,318 5,610 2,807 4,790 8,341 15,148 >4,000 2,263 CLARIFY 4,943 VIDA-IC 2,217 1,037 © 2015 PAREXEL INTERNATIONAL CORP. / 10 CONFIDENTIAL MULTIPLE HF REGISTRIES EXIST FOR EACH EU-5 COUNTRY • A total of 43% registries were multinational while 57% were local or nationally representative patient registries • Data was collected for patients with acute HF* only (31%), chronic HF only (43%), and both acute and chronic HF (26%) in the identified registries • Twelve registries each collected data from Spain, Germany, and Italy and eight and six registries from France and the UK*, respectively 60% Percentages of registries 50% 40% 30% 57% 52% 20% 43% 43% 34% 31% 26% 26% 10% 0% Multinational National Setting Spain, Germany, and italy France Countries *HF: Heart Failure; UK: United Kingdom © 2015 PAREXEL INTERNATIONAL CORP. / 11 CONFIDENTIAL UK Acute HF Chronic HF Patient type Both acute and chronic HF MAJORITY OF THE REGISTRIES REPORTED DATA FOR PATIENT DEMOGRAPHICS, CLINICAL CHARACTERISTICS, MANAGEMENT, AND OUTCOMES Data was available for patient demographics (100%), comorbidities (87%), diagnosis and disease classification (78%), mortality (78%), hospitalisation (96%), prescribed drugs (100%), and patient-reported outcomes (17%) Percentages of registries reporting data 120% 100% 80% 60% 100% 100% 87% 40% 78% 96% 78% 20% 17% 0% Demographics Comorbidities Diagnosis and disease classification Mortality Prescribed drugs Hospitalisation PRO It appears from the published data that there is less emphasis on patient reported outcomes PRO: Patient Reported Outcomes © 2015 PAREXEL INTERNATIONAL CORP. / 12 CONFIDENTIAL Specific treatment procedures Specific diagnostic procedures and test results REGISTRIES REPORTED DATA FOR SPECIFIC DIAGNOSTIC PROCEDURES, TREATMENT, AND RESOURCE USE Blood pressure 91% Blood tests 87% Renal function 70% Imaging (Echo) 70% Electrocardiogram 65% Biomarkers (NT-proBNP, BNP) 44% Coronary revascularisation 57% Cardiac rhythm device 52% Heart catheterization 39% Heart transplant 13% Electrophysiology mapping 9% Resource use Hospitalisations 96% Length of stay 57% Outpatient visit 39% Emergency room visit 22% Cost PRO AE Primary care visit 13% Any AE or specific AE 17% Patient reported outcomes 17% Direct or indirect 13% 0% 10% 20% 30% 40% 50% 60% 70% 80% AE: Adverse Event; BNP: Brain Natriuretic Peptide; NT-proBNP: N-terminal pro-brain natriuretic peptide © 2015 PAREXEL INTERNATIONAL CORP. / 13 CONFIDENTIAL 90% 100% OVER THE LAST DECADE THERE HAS BEEN A SHARP INCREASE IN THE NUMBER OF HF REGISTRIES 6 CLARIFY ESC-HF Pilot EVITA-HF Realise AF Trieste Registry Number of registries 5 EAHFE IN-HF SchlaHF REDINSCOR 4 3 GRACE GRACE 2 EHFS II IS-AHF VIDA-IC ESC Heart Failure ALARM-HF ICALOR 2 HELUMA INDICATE 1 CPRD 0 1985 EHFS I 1990 © 2015 PAREXEL INTERNATIONAL CORP. / 14 1995 2000 Inception year CONFIDENTIAL CNHF RICA 2005 2010 2015 OVER THE LAST DECADE THERE HAS BEEN A SHARP INCREASE IN THE NUMBER OF HF REGISTRIES 15 out of 23 registries initiated post-2005 6 CLARIFY ESC-HF Pilot EVITA-HF Realise AF Trieste Registry Number of registries 5 EAHFE IN-HF SchlaHF REDINSCOR 4 3 GRACE GRACE 2 EHFS II IS-AHF VIDA-IC ESC Heart Failure ALARM-HF ICALOR 2 HELUMA INDICATE 1 CPRD 0 1985 EHFS I 1990 1995 2000 Inception year CNHF RICA 2005 2010 2015 Note: Text in red font indicates registries supported by industry-funding; Black text indicates registries supported by non-profit organizations /universities/ government agencies or where funding information was not clear Inception year for 15 out of 23 registries was post-2005 i.e. majority of the registries initiated in last 10 years reflecting increase in use of real-world data in the recent years © 2015 PAREXEL INTERNATIONAL CORP. / 15 CONFIDENTIAL MAJORITY OF REGISTRIES WERE SPONSORED BY INDUSTRIES Majority of published registries were sponsored by pharmaceutical companies (61%); while 30% of registries were sponsored by non-profit organisations, universities or government agencies Not specified 9% Other 30% Industry funded 61% Rise in the number of heart failure registries in recent years and industry funding indicates increased interest of stakeholders in these registries’ data © 2015 PAREXEL INTERNATIONAL CORP. / 16 CONFIDENTIAL BASED ON DATA VARIABLES REPORTED, TWO REGISTRIES PROVIDED MOST COMPREHENSIVE VIEW OF THE HF PATIENTS IN REAL-WORLD SETTINGS • Identified HF registries were assessed for percentage availability of information on the key data variables 14 Number of registries 12 10 8 6 12 4 8 2 0 2 1 ≤25% 26-50% 51-75% >75% Percentage availability of data on key variables A total of 63% registries reported data on >50% of the assessed data variables © 2015 PAREXEL INTERNATIONAL CORP. / 17 CONFIDENTIAL BERLIN, GERMANY CONCLUSION AND LIMITATIONS © 2015 PAREXEL INTERNATIONAL CORP. / 18 CONFIDENTIAL CONCLUSION • Rise in the number of HF* registries and industry funding in recent years indicate increased interest of stakeholders in these registries’ data • Majority of the registries collected data on key clinical outcomes like hospitalisations and mortality in HF patients demonstrating concordance with the clinical studies • However, only a few registries collected data on the use of biomarkers (NTpro-BNP*, BNP*, troponin), treatment procedures, patient-reported outcomes, adverse events, and costs of care • Long-term follow-up data from registries could help to identify the prognostic factors predisposing HF patients to rehospitalization and sudden death and to understand the impact of interventions on patients’ quality of life, thus, complementing the clinical efficacy demonstrated in relatively short-term, controlled clinical trials • Further, applicability and acceptance of HF registries’ data to inform access and reimbursement decisions and to determine cost-effectiveness of different interventions in clinical practice need to be evaluated * BNP: B-type natriuretic peptide; HF: Heart Failure; NTpro-BNP:N-terminal pro-brain natriuretic peptide © 2015 PAREXEL INTERNATIONAL CORP. / 19 CONFIDENTIAL LIMITATIONS OF THE REVIEW • All literature reviews are limited by publication bias with respect to the available articles. Also, only English language articles were considered for this review • This review was limited to registries with more than 1000 patients and at least one of the data collection center in EU-5 countries © 2015 PAREXEL INTERNATIONAL CORP. / 20 CONFIDENTIAL REFERENCES NICE CG108 (2010) Management of chronic heart failure in adults in primary and secondary care. Available at: http://www.nice.org.uk/guidance/cg108/resources/guidance-chronic-heart-failure-pdf NICE TA314 (2014) Implantable cardioverter defibrillators and cardiac resynchronisation therapy for arrhythmias and heart failure. Available at: https://www.nice.org.uk/guidance/ta314 Dahlstrom U et al. (2010) Heart Failure Pilot Protocol. CardioPulse Article. European Heart Journal. 31:2184-2186 Ambrosy AP et al. (2014) The global health and economic burden of hospitalizations for heart failure: : lessons learned from hospitalized heart failure registries. Journal of the American College of Cardiology. 63 (12):1123-1133 Cowie MR (2015). The global burden of heart failure. European Society of cardiology presentation Garrison LP et al. (2007). Using real-world data for coverage and payment decisions: The ISPOR real-world data task force report. Value in Health. 10 (5):326-335 O’Connor CM et al. (2010) Causes of death and rehospitalization in patients hospitalized with worsening heart failure and reduced left ventricular ejection fraction: results from Efficacy of Vasopressin Antagonism in Heart Failure Outcome Study with Tolvaptan (EVEREST) program. American Heart Journal. 159 (5):841-849 © 2015 PAREXEL INTERNATIONAL CORP. / 21 CONFIDENTIAL QUESTIONS? Jatin Gupta Senior Consultant, Evidence Evaluation PAREXEL International [email protected] www.PAREXEL.com © 2015 PAREXEL INTERNATIONAL CORP. / 22 CONFIDENTIAL THANK YOU © 2015 PAREXEL INTERNATIONAL CORP. / 23 CONFIDENTIAL