Herausforderungen an die klinische Evaluation und Gesundheitsökonomie auf dem Weg zur personalisierten Medizin

Abstract

Healthcare systems across the globe are currently challenged by aging populations, increases in chronic diseases and the difficult task of managing a healthcare budget. In this health economic climate, personalized medicine promises not only an improvement in healthcare delivery but also the possibility of more cost-effective therapies. It is important to remember, however, that personalized medicine has the potential to both increase and decrease costs. Each targeted therapy must be evaluated individually. However, standard clinical trial design is not suitable for personalized therapies. Therefore, both scientists and regulatory authorities will need to accept innovative study designs in order to validate personalized therapies. Hence correct economic evaluations are difficult to carry out due to lack of clear clinical evidence, longitudinal accounting and experience with patient/clinician behavior in the context of personalized medicine. In terms of reimbursement, payers, pharmaceutical companies and companion diagnostic manufacturers will also need to explore creative risk-sharing concepts. Germany is no exception to the challenges that face personalized medicine and for personalized medicine to really become the future of medicine many health economic challenges first need to be overcome. The health economic implications of personalized medicine remain unclear but it is certain that the expansion of targeted therapies in current healthcare systems will create a host of challenges.

Cost-effectiveness of precision medicine: a scoping review

“…There is still a lack of clinical evidence to support PM technologies, particularly of “real-world” data on clinical utility, which is not model based or derived from selective trials (Akhmetov and Bubnov 2015; Berm et al 2016; Brüggenjürgen et al 2012; Phillips et al 2014; Terkola et al 2017). There is an even larger uncertainty with pharmacogenomic tests, since they have no direct influence on patient outcomes, as they do not treat patients directly, but rather improve a clinician’s decisions about treatment (Akhmetov and Bubnov 2015).…”

Personalized medicine: biomarkers and companion diagnostics

“…In this perspective, particularly in oncology, it is now essential that future clinical studies are designed so as to match the therapy to a specific molecular makeup [18,19].…”

“…In fact, the trend of the world Health Services to reimburse high-complexity laboratory tests is gradually declining, also as a result of the remodeling of the reimbursement methodology implemented by the National and Regional Authorities which, in an attempt to maintain the sustainability of the system, consider more favorably low-price diagnostic tests which involve, among other things, the use of a smaller amount of manpower. On the issue of reimbursement, payers, pharmaceutical companies and manufacturers of CDx will have to explore new risk-sharing patterns [18].…”