Examples of RWE-projects conducted by Medaffcon
Study to determine predictive potential of an algorithm for earlier diagnosis of Gaucher disease, utilizing linkage to Auria biobank samples in Finland (SPOT-GD -study), Shire Finland Oy
Gaucher disease (GD) is a lysosomal storage disease, caused by deficiency of the enzyme β-glucocerebrosidase. This leads to accumulation of glucosylceramide in reticuloendothelial cells of spleen, bone marrow and liver. Delay in GD diagnostics is a major challenge and can lead to dysfunction and damages of the target organs. The international Gaucher Earlier Diagnosis Consensus (GED-C) initiative has established a point-scoring system for clinical parameters to promote earlier diagnosis of the disease. The aim of this study was to locally adjust and apply the point-scoring system to health record data available in Auria Biobank, and by using this approach, identify potential undiagnosed patients with Gaucher disease.
A biobank and health register analysis of burden of disease in severe asthma or COPD patients, GSK Finland
This study surveys the status and characteristics of patients with severe asthma or COPD, diagnosed in the hospital district of Southwest Finland during 2004-2015. Information regarding treatment, health care resource use and associated costs, as well as mortality will be explored.
Multiple Myeloma, Amgen AB Finland
Multiple myeloma (MM) is the second most common hematological cancer representing 10-15% of all hematological malignancies. MM results from the clonal expansion of monoclonal plasma cells in the bone marrow, leading to increased secretion of abnormal immunoglobulin into urine and / or peripheral blood. Overproduction of the immunoglobulin fragment causes several deleterious systemic effects, leading to symptoms typically affecting bones and renal function. While MM still remains mostly incurable, the overall survival of MM patients has improved significantly due to the development of more effective therapies.
The aim of this biobank and registry -based study is to survey the status and characteristics of the MM patient cohort diagnosed in Southwest Finland during 2004-2016.
Eosinophilic asthma, AstraZeneca
Asthma is a heterogeneous disease, often characterised by chronic inflammation of breathing organs. New treatment options have been created for the treatment of serious asthma, however, further information is needed on the biological markers that could be used in the diagnosis and follow-up of patients as well as in the evaluation of the prognosis of different phenotypes and endotypes. The aim of this non-interventional retrospective register study is to examine the significance of eosinophil levels in difficult asthma by assessing the characteristics of these patients and their use of healthcare resources.
Read the full article by Mäkelä et al. Health care resource utilization and characteristics of patients with eosinophilic asthma in secondary health care in Finland. Eur Clin Respir J, vol 5, 1458560, 2018.
The special characteristics of chronic lymphocytic leukaemia in the Hospital District of Southwest Finland in 2004–2013, AbbVie
Chronic lymphocytic leukaemia (CLL) is the most common haematological cancer in Western countries. It is typically found as a coincidental finding in connection with other examinations. While asymptomatic CLL typically is not treated, commonly used treatment options in the most difficult forms of the disease include chemoimmunotherapy and other new cancer-specific drugs. The treatment of CLL has developed immensely during the past ten years and therefore the prognosis has improved in all variants of the disease. This study determines the current situation of CLL, e.g. characteristics of the patient population, used treatment regiments and associated outcomes. With the information obtained, it will be possible to evaluate treatments needed in the future and how much resources they require from the healthcare.
Examining the epidemiology, treatment, comorbidites and prognosis of rare immunological haematological diseases in the Hospital District of Southwest Finland in 2003–2014, Novartis Finland Oy
Chronic immunological thrombocytopenic purpura (ITP) and aplastic anaemia (AA) are rare autoimmune-based blood disorders. The exact causes of these diseases are not fully known. The prognosis of aplastic anaemia, in particular, was very poor 40 years ago as estimated survival of a patient with serious AA was only 1–2 years from the diagnosis. This study examines the prevalence, prognosis and complications of ITP and AA, related costs and treatments used. The study provides also valuable information of the impact of the treatments on prognosis of the patients.
Study on the epidemiological prevalence of melanoma, treatment effectiveness and the use of healthcare resources in the Hospital District of Southwest Finland, Novartis Finland Oy
Metastatic melanoma is a rapidly progressing disease with poor prognosis. Only in recent years, with the introduction of new effective therapies, improvements in the overall survival have been achieved. In particular, BRAF inhibitors and immunotherapy provide new treatment options for a subset of patients. Further information is, however, warranted to clarify the value of different biomarkers indicating the likely responders that would benefit from these different therapies. This study provides information on the use of different treatment options for patients with melanoma, effectiveness of the treatments used, observed comorbidities as well as utilization of healthcare resources.
Study on the epidemiological prevalence of polycythemia vera, Novartis Finland Oy
Polycythemia vera (PV) is a slowly progressing disease which can further develop into myelofibrosis or acute leukaemia in a subset of PV patients. This study examines how PV patients are divided into groups of low and high risks, and which risk factors influence the progression of polycythemia vera into either myelofibrosis or acute leukaemia. This study increases knowledge on the epidemiology of polycythemia vera and the potential factors impacting its prognosis.
Study on the epidemiological prevalence and postoperative treatment of acromegaly in the Hospital District of Southwest Finland, Novartis Finland Oy
Acromegaly is a rare disease resulting from excess growth hormone. In most cases, the disease is caused by a pituitary tumour. This study provides information on the proportion of acromegaly patients whose growth hormone levels do not normalize after the removal of the pituitary tumour and proportion of patients that need somatostatin analogue treatment in order to obtain clinical response. In addition, the comorbidities between patients with instable growth hormone levels and those with recovered hormone levels will be compared. This study provides information for more optimized targeting of treatments for patients with acromegaly.
Study on the epidemiological prevalence of myelofibrosis, polycythemia vera and essential thrombocythemia and the overall survival of patients with myelofibrosis in the Hospital District of Southwest Finland, Novartis Finland Oy
Myelofibrosis (MF) is a slowly progressing disease with poor prognosis. While improvements in overall survival of patients with MF have been achieved, a proportion of MF patients develop subsequently acute leukaemia. This study assesses the proportion of MF patients whose disease transforms to leukaemia and the putative significance of JAK2V617F gene mutation in this process. This study increases knowledge on the epidemiology of myelofibrosis and the potential prognostic factors of the disease.