>> “The German healthcare system is probably one of the best in the world, but we still have a great need for innovation, including in our processes,” said Prof. Dr. Alexander Schachtrupp from the B. Braun Foundation at the start of the 4th plenary event entitled “From innovation to standard care: benefit assessment and decision-making”, which was organized jointly by the Neubrandenburg University of Applied Sciences and the B. Braun Foundation on June 6th in Berlin became. The questions arise as to how medical innovations reach the patient and whether this can be achieved in a reasonable time. Prof. Dr. Axel Mühlbacher emphasized the opportunities to promote innovation in the sense of using new options for action through adaptive benefit assessment. The health economist from the Neubrandenburg University of Applied Sciences and co-organizer of the plenum is certain that a balance must be achieved between high investments in new technologies, patient benefits and the costs of the system.
Medical technology innovations get into the system faster: Post-market data is helpful
Harald Kuhne from the Federal Ministry for Economic Affairs and Energy pointed out the economic importance of the healthcare industry with a gross value added of around 350 billion euros in 2017. At 93 percent, the industry structure of medical technology companies is dominated by small and medium-sized companies. The innovation cycles are significantly shorter than in the pharmaceutical sector: a third of sales come from products that are no older than three years. You have to keep this in mind when you talk about procedures that are imposed on medical technology manufacturers in order to be eligible for reimbursement. “If the same criteria apply to medical technology as to the pharmaceutical industry, it could be dangerous for the German innovation system,” noted Kuhne critically. Using the example of an implant for individualized neurostimulation to lower blood pressure, Dr. Michael Lauk, CEO of neuroloop GmbH, the time periods to be taken into account: Basic and preclinical research takes around 10 years. The first applications on humans will be followed by a 2-year multi-center study, so the market launch is planned for 2022. Access to the reimbursement system takes many years. Therefore, in Lauk's opinion, “financial and defined paths for the introduction of new therapies and methods are needed, especially for start-up companies, after safety and basic effectiveness have been demonstrated, for example by focusing on post-market data.
Early benefit assessment for a quick start and late for safety
The importance of randomized controlled trials when it comes to proving the causality of clinical effects is largely undisputed. “Nevertheless, it makes sense to discuss the extent to which we can create additional evidence with innovative study designs and registries,” explained Mühlbacher. In his opinion, instead of problematic all-or-nothing decisions, conditional pricing and continuous evidence generation over the product life cycle could be designed as an adaptive benefit assessment. In connection with the early benefit assessment of drugs, Prof. Dr. Bernhard Wörmann, Berlin, points out that although almost all new preparations are available early with this process, only relatively immature data can be available at this point. This is also critical with regard to certain side effects that can only occur years later. That is why the medical director of the German Society for Hematology and Medical Oncology (DGHO) suggests an additional late benefit assessment for sustainable determinations. In order to compensate for the deficits in the early benefit assessment, standardized data would have to be collected in the meantime, for example from registers.
Professor Wörmann also addressed another important topic, namely the relevance of endpoints, and asked the question: “Are the endpoints that are specified by the Health Technology Assessment or that we use in guidelines really close to the patient?” In oncology, for example, there is currently a discussion for immunotherapies as to whether the mean survival time or the rate of long-term survivors is more important. Which endpoint has the higher value? According to Wörmann, the assessment of endpoints in very heterogeneous study groups is particularly difficult. For example, for patients over 80 years old with Hodgkin's lymphoma, is the endpoint survival time just as relevant as for young patients or is quality of life more relevant? Progression-free survival is also an important assessment criterion, as the hematologist and oncologist explained: “Many diseases become chronic diseases, patients do not live disease-free, but progression-free.”
Patient preferences are also playing an increasing role here. Who is qualified to make value judgments when determining whether the benefits of a method outweigh the risks, asked Juan Marcos Gonzalez, a professor at Duke University in Durham, USA. Traditionally these were doctors and politicians. “But fundamentally, the patient is the best expert when it comes to his or her well-being,” emphasized Gonzales. In the USA there is a development towards patient-centered healthcare and thus the increasing importance of patient preferences. The Food and Drug Administration (FDA) fundamentally advocates the approach of involving patients in the entire process of medical device development. Gonzalez reported on a pilot study as part of the FDA's Center for Devices and Radiological Health (CDRH) Patient Preferences Initiative in which the scientist was involved.
Prof. Dr. warned against overestimating apparently objective data from the measurement of clinical effects. Karl H. Wehkamp, University of Bremen. Patients judge medical innovations in their respective, heterogeneous roles with changing interests and perspectives. The importance of measuring patient preferences was confirmed by PD Dr. Stefan Sauerland, head of department for non-drug procedures at the Institute for Quality and Efficiency in Health Care (IQWiG). However, results on patient preferences are not regularly required for the IQWiG reports on benefit assessment, as the usual data situation is clear, says Sauerland. <<

by: Matthias Manych