Relative effectiveness assessments of oncology medicines for pricing and reimbursement decisions in European countries.

BACKGROUND: There is a debate on the added clinical value of new, expensive, anticancer treatments. Among European decision makers, the relevance of commonly used end points in trials, especially overall survival (OS), progression-free survival (PFS) and quality of life (QoL), varies, leading to the available evidence being valued differently. This research studies the extent to which the value of end points for cancer medicines differs among European decision makers. METHODS: We compared guidelines and relative effectiveness assessments (REAs) of medicines for pricing or reimbursement decisions in England, France, Germany, The Netherlands, Poland, and Scotland. Anticancer medicines that received marketing authorization in Europe between 2011 and 2013 with at least four available national REAs were evaluated. A total of 79 REAs were included. RESULTS: Health technology assessment (HTA) guidelines indicate a preference for clinically and patient relevant end points such as OS and QoL above surrogate end points. Most guidelines do not specify whether PFS is considered a surrogate or patient-relevant end point. The number of REAs included per jurisdiction varied between 7 (The Netherlands) and 18 (Germany). OS data were included in all REAs and were the preferred end point by HTA agencies, but these data were not always mature or robust. QoL data are included in only 54% of the REAs, with a limited impact on the recommendations. PFS data are included in 70% of the REAs, but the extent to which HTA agencies find PFS relevant varies. CONCLUSIONS: European decision-making on relative effectiveness of anticancer medicines is affected by a gap in requested versus available clinical evidence, mainly because the regulator is willing to accept some degree of clinical uncertainty. A multi-stakeholder debate would be essential to align concrete robust evidence requirements in oncology and a collectively shared definition for relevant clinical benefit, which will benefit patients and society in general.

Does conditional approval for new oncology drugs in Europe lead to differences in health technology assessment decisions?

An early access pathway of conditional approval for potentially beneficial medicines is available within the European regulatory framework. However, marketing authorization does not necessarily result in recommendations for public funding by health technology assessment (HTA) agencies. As conditional approval goes along with less than complete data on benefits and risks of a treatment option for a high medical need, this raises the question how HTA decision-making is affected by these uncertainties.

Poland health system review.

Since the successful transition to a freely elected parliament and a market economy after 1989, Poland is now a stable democracy and is well represented within political and economic organizations in Europe and worldwide. The strongly centralized health system based on the Semashko model was replaced with a decentralized system of mandatory health insurance, complemented with financing from state and territorial self-government budgets. There is a clear separation of health care financing and provision: the National Health Fund (NFZ) the sole payer in the system is in charge of health care financing and contracts with public and non-public health care providers. The Ministry of Health is the key policy-maker and regulator in the system and is supported by a number of advisory bodies, some of them recently established. Health insurance contributions, borne entirely by employees, are collected by intermediary institutions and are pooled by the NFZ and distributed between the 16 regional NFZ branches. In 2009, Poland spent 7.4% of its gross domestic product (GDP) on health. Around 70% of health expenditure came from public sources and over 83.5% of this expenditure can be attributed to the (near) universal health insurance. The relatively high share of private expenditure is mostly represented by out-of-pocket (OOP) payments, mainly in the form of co-payments and informal payments. Voluntary health insurance (VHI) does not play an important role and is largely limited to medical subscription packages offered by employers. Compulsory health insurance covers 98% of the population and guarantees access to a broad range of health services. However, the limited financial resources of the NFZ mean that broad entitlements guaranteed on paper are not always available. Health care financing is overall at most proportional: while financing from health care contributions is proportional and budgetary subsidies to system funding are progressive, high OOP expenditures, particularly in areas such as pharmaceuticals, are highly regressive. The health status of the Polish population has improved substantially, with average life expectancy at birth reaching 80.2 years for women and 71.6 years for men in 2009. However, there is still a vast gap in life expectancy between Poland and the western European Union (EU) countries and between life expectancy overall and the expected number of years without illness or disability. Given its modest financial, human and material health care resources and the corresponding outcomes, the overall financial efficiency of the Polish system is satisfactory. Both allocative and technical efficiency leave room for improvement. Several measures, such as prioritizing primary care and adopting new payment mechanisms such as diagnosis-related groups (DRGs), have been introduced in recent years but need to be expanded to other areas and intensified. Additionally, numerous initiatives to enhance quality control and build the required expertise and evidence base for the system are also in place. These could improve general satisfaction with the system, which is not particularly high. Limited resources, a general aversion to cost-sharing stemming from a long experience with broad public coverage and shortages in health workforce need to be addressed before better outcomes can be achieved by the system. Increased cooperation between various bodies within the health and social care sectors would also contribute in this direction. The HiT profiles are country-based reports that provide a detailed description of a health system and of policy initiatives in progress or under development. HiTs examine different approaches to the organization, financing and delivery of health services, and the role of the main actors in health systems; they describe the institutional framework, process, content and implementation of health and health care policies; and highlight challenges and areas that require more in-depth analysis.

Poland: health system review

The Health Systems in Transition (HiT) country profiles provide an analytical description of each health system and of policy initiatives in progress or under development. They aim to provide relevant comparative information to support policy-makers and analysts in the development of health systems and reforms in the countries of the WHO European Region and beyond. The HiT profiles are building blocks that can be used: to learn in detail about different approaches to the financing, organization and delivery of health services; to describe accurately the process, content and implementation of health reform programmes; to highlight common challenges and areas that require more in-depth analysis; and to provide a tool for the dissemination of information on health systems and the exchange of experiences of reform strategies between policy-makers and analysts in countries of the WHO European Region. This series is an ongoing initiative and material is updated at regular intervals.

TLC fractionation and visualization of selected phenolic compounds applied as drugs.

Sixteen visualizing agents, thirteen of which were a group of alkacemetric indicators were used for the detection of selected drugs (being phenolic compounds): adrenaline, dopamine, phenylephrine, metaraminol, fenoterol, and bithionol. Visualizing effects for the drugs investigated after their TLC separation were estimated on silicagel (A), mixture silica gel/kieselguhr (B), and on polyamide (C). The best separations and the most positive visualising effects were obtained on adsorbent A, and the least on absorbent C. On adsorbent A, the most profitable detectability equal to 100 ng, was obtained also for adrenaline, dopamine and fenoterol with the application of basic solution of bromocresol green and brilliant cresyl blue as visualizing agents.