Biologics registers in RA: methodological aspects, current role and future applications

The beginning of the 21st century saw a biopharmaceutical revolution in the treatment of inflammatory rheumatic diseases, particularly rheumatoid arthritis. The fast-evolving use of biologic therapies highlighted the need to develop registers at national and international levels with the aim of collecting long-term data on patient outcomes. Over the past 15 years, many biologics registers have contributed a wealth of data and provided robust and reliable evidence on the use, effectiveness and safety of these therapies. The unavoidable challenges posed by the continuous introduction of new therapies, particularly with regard to understanding their long-term safety, highlights the importance of learning from experience with established biologic therapies. In this Perspectives article, the role of biologics registers in bridging the evidence gap between efficacy in clinical trials and real-world effectiveness is discussed, with a focus on methodological aspects of registers, their unique features and challenges and their role going forward

The Danish National Chronic Myeloid Neoplasia Registry

Marie Bak,1 Else Helene Ibfelt,2 Thomas Stauffer Larsen,3 Dorthe Rønnov-Jessen,4 Niels Pallisgaard,5 Ann Madelung,6 Lene Udby,1 Hans Carl Hasselbalch,1 Ole Weis Bjerrum,7 Christen Lykkegaard Andersen1,7 1Department of Hematology, Zealand University Hospital, University of Copenhagen, Roskilde, 2Research Centre for Prevention and Health, Rigshospitalet Glostrup, University of Copenhagen, Glostrup, 3Department of Hematology, Odense University Hospital, Odense, 4Department of Hematology, Vejle Hospital, Vejle, 5Department of Surgical Pathology, Zealand University Hospital, University of Copenhagen, Roskilde, 6Department of Surgical Pathology, Zealand University Hospital, University of Copenhagen, Næstved, 7Department of Hematology, Rigshospitalet, University of Copenhagen, Copenhagen, Denmark Aim: The Danish National Chronic Myeloid Neoplasia Registry (DCMR) is a population-based clinical quality database, introduced to evaluate diagnosis and treatment of patients with chronic myeloid malignancies. The aim is to monitor the clinical quality at the national, regional, and hospital departmental levels and serve as a platform for research. Study population: The DCMR has nationwide coverage and contains information on patients diagnosed at hematology departments from January 2010 onward, including patients with essential thrombocythemia, polycythemia vera, myelofibrosis, unclassifiable myeloproliferative neoplasms, chronic myelomonocytic leukemia, and chronic myeloid leukemia. Main variables: Data are collected using standardized registration forms (so far up to four forms per patient), which are consecutively filled out online at time of diagnosis, after 2-year and 5-year follow-ups, and at end of follow-up. The forms include variables that describe clinical/paraclinical assessments, treatment, disease progression, and survival – disease-specific variables – as well as variables that are identical for all chronic myeloid malignancies. Descriptive data: By the end of 2014, the DCMR contained data on 2,690 patients with an inclusion rate of ~500 patients each year. Since the registry was established, annual reports have shown consistently high national coverage and data completeness, ≥90% and ≥88%, respectively. Conclusion: The DCMR is a national database used for monitoring the quality of patient care in patients with chronic myeloid malignancies, but until validation has been conducted, the data must be used with caution. However, the DCMR is a valuable data source accessible to clinicians and researchers. Keywords: myeloproliferative disorders, database, treatment, health care quality assurance, outcome assessment, epidemiology, researc

Validity and completeness of rheumatoid arthritis diagnoses in the nationwide DANBIO clinical register and the Danish National Patient Registry

Else Helene Ibfelt,1 Jan Sørensen,2,3 Dorte V Jensen,4,5 Lene Dreyer,5,6 Berit Schiøttz‑Christensen,7 Pia H Thygesen,8 Ada Colic,9 Johnny L Raun,10 Natalia Manilo,11 Anne Rødgaard,4 Uta E Poulsen,12 Claus Rasmussen,13 Torben Hansen,14 Babara Unger,15 Randi Pelck,16 Anita Kincses,17 Henrik Nordin,18 Tove Lorenzen,19 Ali Theibich,20 Inger Marie Jensen Hansen,21 Jakob Espesen,22 Jolanta Grydehøj,23 Mette Holland-Fischer,24 Anne Gitte Loft,25 Merete Lund Hetland4,26 1Research Centre for Prevention and Health, Capital Region of Denmark, Rigshospitalet – Glostrup, Denmark; 2Centre of Health Economics Research, Institute of Public health, University of Southern Denmark, Odense, Denmark; 3Healthcare Outcome Research Centre, Royal College of Surgeons in Ireland, Dublin, Ireland; 4DANBiO and Copenhagen Center for Arthritis Research, Center for Rheumatology and Spine Diseases, Rigshospitalet – Glostrup, Glostrup, 5Center for Rheumatology and Spine Diseases, Gentofte University Hospital, Hellerup, 6The Parker Institute, Frederiksberg and Bispebjerg Hospital, Frederiksberg, 7Spine Centre of Southern Denmark, Hospital Lillebaelt, Middelfart, 8Department of Rheumatology, Odense University Hospital, Odense, 9Department of Rheumatology, Sydvestjysk Sygehus, Esbjerg/Grintsted, 10Department of Internal Medicine and Rheumatology, SLB – Fredericia Hospital, Fredericia, 11Department of Rheumatology, Frederiksberg Hospital, Copenhagen, 12Department of Rheumatology, Gigthospital Gråsten, Gråsten, 13Clinic of Internal Medicine, Rheumatology, Regionshospital Nordjylland, Hjørring, 14Department of Rheumatology, Holbæk sygehus, Holbæk, 15Department of Internal Medicine/Rheumatology, Hospitalsenheden Horsens, Horsens, 16Department of Rheumatology, Zealand University Hospital, Køge, 17Department of Rheumatology, Nordsjællands Hospitaler, Hillerød, 18Center for Rheumatology and Spine Diseases, Centre of Head and Orthopedics, Rigshospitalet, Copenhagen, 19Department of Rheumatology, Silkeborg Regional Hospital and University Clinic, Silkeborg, 20Department of Rheumatology, Slagelse Hospital, Slagelse, 21Department of Rheumatology and University of Southern Denmark, Svendborg Hospital, Odense, 22Department of Internal Medicine, Vejle Hospital, Vejle, 23Department of Rheumatology, Regional Hospital, West Jutland, Herning, 24Department of Rheumatology, Aalborg University Hospital, Ålborg, 25Department of Rheumatology, Aarhus University Hospital, Aarhus, 26Department of Clinical Medicine, Faculty of Health and Medical Sciences, University of Copenhagen, Copenhagen, Denmark Objectives: In Denmark, patients with rheumatoid arthritis (RA) are registered in the nationwide clinical DANBIO quality register and the Danish National Patient Registry (DNPR). The aim was to study the validity of the RA diagnosis and to estimate the completeness of relevant RA cases in each registry.Study design and setting: Patients registered for the first time in 2011 with a diagnosis of RA were identified in DANBIO and DNPR in January 2013. For DNPR, filters were applied to reduce false-positive cases. The diagnosis was verified by a review of patient records. We calculated the positive predictive values (PPVs) of the RA diagnosis registrations in DANBIO and DNPR, and estimated the registry completeness of relevant RA cases for both DANBIO and DNPR. Updated data from 2011 to 2015 from DANBIO were retrieved to identify patients with delayed registration, and the registry completeness and PPV was recalculated.Results: We identified 1,678 unique patients in DANBIO or in DNPR. The PPV (2013 dataset) was 92% in DANBIO and 79% in DNPR. PPV for DANBIO on the 2015 update was 96%. The registry completeness of relevant RA cases was 43% in DANBIO, increasing to 91% in the 2015 update and 90% in DNPR.Conclusion: DANBIO held a high proportion of true RA cases (96%) and was found to be superior to the DNPR (79%) with regard to the validity of the diagnosis. Both registries were estimated to have a high completeness of RA cases treated in hospital care (~90%). Keywords: rheumatoid arthritis, validity, incidence, clinical registry, Denmar