Original article
In 2011, 30 new agents were approved in the USA and 22 in Europe, including 8 and 7 for the treatment of oncological indications, respectively (1). This trend towards an increasing proportion of oncological drugs, which has already been observed for several years, is continuing in 2012, as current figures from the US Food and Drug Administration (FDA) show: one third – 13 of the 39 newly approved active substances – relate to oncological areas of application (2). Approval in oncology often takes place as part of accelerated approval procedures (“conditional marketing authorization” or “accelerated approval”) and/or as an “orphan drug” for rare diseases. The main reasons given for the significant increase in oncological active substances are: progress in the molecular-biological characterization of tumor growth, development of a large number of new "targeted" active substances, great need for more effective drug therapy options for advanced tumor diseases, demographic development, expiry of patents for blockbusters on Treatment of common diseases and a very lucrative market for oncological drugs.
24.01.2013
When discussing surrogates and endpoints, one quickly comes to the question of which treatment results and characteristics are actually relevant for the patient. The appropriate expression “patient-relevant endpoint” is anchored in the legal basis for early benefit assessment according to Section 35a SGB V.1 While in other places there is explicit talk of a “therapy-relevant higher benefit” or “therapy-relevant side effects” (cf. e.g. § 35 Para. 1b SGB V), is clearly aimed at the patient. In the associated dossier specifications for Section 35a SGB V, module 3.3.6 mentions patient preferences as a data source to be taken into account.2 Thus, the importance of patient-relevant endpoints and patient preferences has increased with the AMNOG (Law on the Reorganization of the Medicines Market), but what role they play in concrete terms plays is still to be clarified. Nevertheless, the IQWiG (Institute for Quality and Efficiency) commissioned pre-AMNOG for two pilot studies on preference measurement (Conjoint Analysis, Analytical Hierarchy Process), one of which has already been published. IQWiG also focuses on patient preferences in its current method paper (see IQWIG (2013); IQWIG (2010b); IQWIG (2011)).
24.01.2013
The German Medical Association defines health services research (1) as the scientific study of the provision of health-related products and services to individuals and the population (2) under everyday conditions. For this purpose, health services research examines how access to health and health care, how its quality and costs and ultimately how our health and well-being are influenced by the financing systems, by social and individual factors, by organizational structures and processes and by health technologies ( 3). The observation units include individuals, families, populations, organizations, institutions, municipalities, etc. In addition to this definition of content, the German Medical Association offers a methodological/functional definition of health services research, describes the general objective of health services research and names the systematics/components (input-throuput- Output-outcome) of health services research (1). In our article we would like to expand this characterization of health services research to include five further tasks: the distinction between care and research, the economic definition of over- and under-care, the description of the data and methods required for health services research, and the distinction between economically complete and incomplete health care analyses and ultimately the formal medical (in addition to the commercial) assessment of effort and return, including a method for identifying patient-relevant endpoints.
24.01.2013
What man wouldn't be terrified if they were diagnosed with prostate cancer? However, whether a panic attack is justified depends on the stage of the illness. If bone metastases already appear, life expectancy is low. Tumors in the prostate often spread to the bones. Metastases in other areas of the body also further shorten the lifespan of patients.
24.01.2013
Germany is a rich country, despite the financial crisis and its global consequences. It offers the third most expensive healthcare system in the world - after the USA and the Netherlands1. Nevertheless, people here are not getting any older than in countries with much cheaper systems, such as that in Sweden. Life expectancy at birth was 80.3 years in Germany in 2009; it was higher in 13 European countries, with the highest in Switzerland at 82.3 years2. It is undisputed that progress costs money and innovations do not always have to make sense in the long term - at least at the beginning of the introduction this cannot always be estimated precisely. But here the problem is completely different: It is not the innovations that make the system so expensive, although that is often claimed and is therefore viewed particularly critically, but rather the uncontrolled use of techniques and processes that are often already outdated.
24.01.2013
Improving oral health and intensifying dental care for residents in nursing homes and facilities for the disabled have been the subject of numerous publications (1) and the subject of political initiatives (2) for many years. The Federal Dental Association (BZÄK) and the National Association of Statutory Health Insurance Dentists (KZBV) presented a joint concept in 2010 (3). Almost all authors assume that dental care in these facilities is inadequate. In 2006, the dental service of the Bremen Public Health Department examined the residents of two nursing homes in Bremen and determined the existing need for dental treatment. In over 30% of the residents examined, dental intervention would have been urgently required at that time (4).
24.01.2013
The benefits of statutory health insurance include the prevention, early detection and treatment of illnesses as well as aftercare. All services provided at the expense of the GKV are subject to the cost-effectiveness requirement according to §12 SGB V. The population-oriented distribution of morbidity (approximately 20% of the insured persons tie up around 80% of the services) means, conversely, that approximately 80% of the insured persons actually receive no or only very few services claim. A financing model must take this into account (false price signals) as well as the politically responsible design of an effectiveness-based performance framework. This highlights the need to examine which services should be collectively financed. Medical procedures, products and medicines must be evaluated with regard to their effect (ideally the patient-relevant benefit) in order to include them in the GKV's catalog of services or, if necessary, exclude them.
24.01.2013
Based on the findings from health services research, decisions can be made about benefits in statutory health insurance (GKV) and health care can be improved. Corresponding analyzes are made easier by extensive, reliable and comprehensible data. In order to improve the data situation in Germany, the legislature has created the conditions with the Statutory Health Insurance Care Structure Act to enable data from the morbidity-oriented risk structure adjustment (Morbi-RSA) to also be used for health care research purposes. The German Institute for Medical Documentation and Information (DIMDI) is responsible for preparing and making this data available. In addition to joint self-administration, institutions such as the Federal Joint Committee, the Institute for Quality and Efficiency in Health Care and universities are also authorized to use the data. Whether the intended improvement in health care research can actually be realized must be critically questioned once the data transparency process has begun. The aim of this work is to provide an overview of the new process.
24.01.2013
Biopharmaceuticals are medicines whose active ingredients are obtained as part of a genetic engineering manufacturing process using DNA recombination methods. This manufacturing process is fundamentally different from the synthesis of the active ingredients of chemical drugs. While a chemical synthesis route can be copied identically, the production of drugs from genetically modified organisms is much more complex and results in the active ingredient remaining unique and can only be imitated by another manufacturer, but not reproduced identically. For this reason, copycat biopharmaceuticals are referred to as biosimilars and not as generics, as is the case with chemical medications. In the next few years, a number of biopharmaceuticals will lose their exclusivity, so new biosimilars are likely to complement the biosimilar market, which is currently limited to three drug groups. Even if this can result in cost savings for the payers, it is not possible to validly forecast market developments.
24.01.2013
Clinical registries are increasingly being used to gain knowledge in healthcare (Bertoletti 2013, Lesko 2013, Majek 2013, Veiby 2013, Weiss 2013). Proponents of registers see the advantage of being able to use a well-maintained register to answer a large number of questions that were not yet known at the time the register was initiated. Since regularly and largely complete data sets are collected to record the services provided and to determine the remuneration for services, it is obvious that these data should also be used to answer relevant questions in the benefit assessment of medicines and care management. In addition, this data can be merged with data sets from various sources, e.g. clinical data with data from insurance companies or pharmacies. Skeptics of registers fear that reliable answers cannot always be provided with register data. This argument cannot be dismissed out of hand because it seems possible that a register can, for example, record all the data that is generated during the operation of a technical device. If it is not an isolated technical device but a device in a specific environment, e.g. a car in traffic, which is exposed to complex processes such as the behavior of all road users, it will be considerably more difficult to combine all conceivable variables a priori in one To map registers that ultimately influence the outcome of a process, in the case of a car, reaching the destination. If registers are to be used to describe patient-relevant problems, it can be predicted that the complexity of these bio-psycho-social processes will be so high that the limit of feasibility may be reached. These two perspectives allow us to predict that registries will undoubtedly be useful in health services research, but will also be subject to certain limitations - just like clinical studies. We would therefore like to present theoretical considerations as well as practical experiences on the opportunities and risks of registries and clinical studies and discuss recommendations for their optimized use.