V HTA Symposium in Krakow – Session Recap I
Arcana Institute hosted the V HTA Symposium in Krakow on 29th-30th May 2017, which provided an in-depth view of the many important topics taking place in Poland in the realm of systematic changes in healthcare related to the development of clinical registries and planned introduction of amendments to reimbursement regulation concerning rare diseases and medical devices.
HTA & Real World Data
Speaker’s Panel: Prof. Rafał Niżankowski, Dominik Dziurda, Dr Andrzej Śliwczyński, Dr Wojciech Wysoczański, Izabela Pieniążek
Moderator: Jacek Walczak
Real World Data
Data that is analyzed for clinical effectiveness of interventions should come from reliable clinical studies found at the top of the hierarchy for evidence (i.e. systematic reviews, RCT). Furthermore, such data should be supported and supplemented with data from trusted and reliable payer and other entity databases, including patient registries.
Real World Data (RWD), such as data originating from patient registries, allows for conclusions to be made on the effectiveness of a technology in real-world clinical practice. It is important to state that there is an increasing importance of RWD, in particular for the treatment of rare diseases and for medical devices, for which the availability of RCTs may be limited. Access to RWD data provides an important source of information for a specific subject matter and is supplementary to data obtained from clinical trials carried out the by the manufacturer of a medical technology. Prior to the collection and analysis of large data sets, it is necessary to define the purpose, the scope of data to be gathered and how collection of such data may be used in practice, which may include the continuous monitoring of results from a health program or to compare the effectiveness of various therapeutic interventions for a given indication (e.g. chemotherapy vs. biological drugs). The process for collecting data should be carefully planned by identifying reference points, which allows to introduce correction of data.
In regards to the amount of available registries by country, Italy comes on top with the highest number of registries available. In Poland, there is an ever-increasing sentiment for the need to maintain reliable registries as a key source of data to determine treatment effectiveness in everyday clinical practice. Registries are often considered a tool for advice and support, allowing clinicians to obtain information and to verify if the currently used therapy is the best available therapeutic option for a given patient. Nevertheless, it should be noted that in Poland, the biggest hindrance for the creation of various databases / registries is the issue of mentality, where entities, such as hospitals, are not willing to share and publish certain information, such as number of deaths or infections. Additionally, the collection of reliable data is associated with increased bureaucratic work by clinicians.
During the Symposium, it was highlighted that the basic benefits package makes up part of the system for quality management and availability of services. A careful analysis of the recommendations of diagnostic and therapeutic procedures creates the shape of the basic benefits package. Determining the benefits tariffs by the Agency for HTA and Tariff System in Poland (AHTAPol) allows for market regulation of providers of medical technologies. Analysis of the recommendations for diagnostics and therapeutics, based on ICD-9 and ICD-10, helps to define the eligibility of certain groups of patients for publically funded benefits. Furthermore, taking into account the organizational and structural standards allows for effective management of quality and availability of benefits.
Health outcome-based risk-sharing agreements (RSS)
In Poland, the importance of risk-sharing schemes (RSS) based on health effects is becoming increasingly recognized by the Ministry of Health (MoH) and National Health Fund (NFZ, Payer). It is important to note that for certain medicines, such as those used in rare indications or medical devices, RSS may be the only option for obtaining reimbursement. Despite the willingness of the Payer to adapt RSS, settlement of risk-sharing schemes based on health outcomes comes with its difficulties. The availability of databases allows for the obtainment of information on patient treatment stages in order to infer the occurrence of a health effect. However, few European countries make use of RSS based on health effects for the simple fact that it is not possible to identify the occurrence of the effect. Assessing a health effect may be problematic in the case when a patient has various medications and procedures at a given time.
Statistical significance and probabilistic analysis
When analyzing statistical significance in clinical studies, it should be noted that the selection of the statistical test may influence the obtainment of statistically significant results. Consequently, it is important to approach such significant results with caution, in particular those bordering statistical significance. A new drug should have clinical effectiveness greater than that which would allow it to qualify as a generic or biosimilar. The difference should be clinically identifiable.
New guidelines for conducting HTA analyses have been in force since August 2016. One of the key changes in the section for economic analysis is the introduction of the need to carry out a Probabilistic Sensitivity Analysis (PSA). By adopting such a requirement, AHTAPol follows other HTA institutions in Europe and around the world. A similar requirement can be found in the guidelines of NICE, AWMSG, NCPE, ZN, or CADTH. The PSA is an optional analysis in the guidelines of HAS, IQWIG, PBAC and PHARMAC. Looking at the reimbursement process in Poland, which began after the new AOTMiT guidelines came into force, most of the HTA reports fulfil PSA requirements under the economic analysis, whose results are taken into account both at the verification phase and at the recommendation of the President of the Agency. Unfortunately, results of the PSA are often confidential, making it difficult to draw a clear conclusion on the relationship between results of an analysis and the nature of the recommendation. Given the available data, there is a tendency of receiving a negative recommendation by the President of AHTAPol for medicines for which the probability of cost effectiveness did not exceed 30%.
