Abstrakti

Četvrta međunarodna konferencija
Donošenje odluka zasnovano na dokazima u zdravstvu centralne i istočne Evrope

Krzysztof Łanda, M.D., HTA Audit

HTA for decision making in pricing – the Australian IHPA and the Agency for Tariffs planned in Poland

Considering cost accounting of health services and the Pricing Agency cannot be done without the understanding of several primary issues which have significant implications in this regard: One should be aware of what valuation is and what purpose it serves and one must clearly differentiate between cost of services and valuation of services. Valuation is always arbitrary. Prices make transactions either possible or impossible. In exchange for payment, somebody waives their right to certain goods or provides services. If the main aim of setting forth prices is ensuring the adequate supply of certain services, then valuation does not have to be performed on the basis of a cost analysis (although in some cases - ca. 10-15% of services - such an analysis will be used; for the majority of services however, it is neither necessary nor possible to be performed - see information below). One must be aware of the subject matter we are dealing with, that is:
The number of services in the guaranteed benefits package, the variability of the package's content and the variability of service costs over time, and consequently the variability in interrelations of health care service costs. Only that can depict the possibilities for cost accounting of health care services.
Before preparing a draft act on the Pricing Agency, a feasibility study for several possible scenarios of its functioning should be performed. Among others, one should pose a question of whether performing functions of the Pricing Agency by different institutions would not impair the basic functions of those institution which they were primary established to perform, and most certainly the function of valuation should not be performed jointly with appraisal for the purpose of reimbursement decisions (i.e. decisions on including a technology in the guaranteed benefits package). Establishing the Pricing Agency within AHTAPol does not seem to be the best permanent solution, it can however be considered a temporary one.

Mark Parker, MSc. Health Economics Unit, University of Liverpool Management School, United Kingdom

Decision Criteria

Decision Making within a healthcare system is a simple matter of life and death, at the population level. Within a constrained budget healthcare decision makers face the very real problem that choosing to allocate resources to one area necessarily means those resources are not available to another.
To address these problems Health Economists have equipped decision makers with an array of tools to enable them to make informed and transparent choices, providing a podium for discussion whereby each interested stakeholder can ensure their interests are considered, and place them within the context of the healthcare system.
Budget Impact, Cost Effectiveness/Utility Analysis and now Value Based Pricing and Multi Criteria Decision Analysis have emerged as the tools of choice to summarise concisely complex decision problems, emerging from various legal battles between patients, the pharmaceutical industry and health insurance organisations, Health Technology Assessment Agencies are quickly emerging as a means to keep these problems out of the courts, submissions to the various different agencies across the world effectively now form legal briefs, which lay out in intricate detail what is meant by "Best Practice", and by inference the harm caused to a countries population should healthcare deviate from this practice.

Vanesa Benkovic, HTA, Health research consultant, Croatia

Market access in CEE countries

There is an increased demand for healthcare, owing to the ageing of the population. The rate of unemployment is quite high in CEE countries, which reduces the numbers of people making contributions for the healthcare system. However, as in many countries, slower GDP [gross domestic product] growth has affected the availability of money for spending in the healthcare sector. As a result, there is a lot of pressure on healthcare services, and on prices. Each of the CEE countries has different profile of health financing percentage from the GDP as well as the share of public financing, which all comes back to the amount of money reserved for drug and devices budget. Issues of referencing, pricing and models of reimbursement make similar hurdles with more-less same level of complexity. On the other hand, state regulation of market access for drugs and devices is present in CEE countries with many variations. Restrictions are increasingly being placed on reimbursement; drugs are being approved for smaller target populations, or only following the failure of other agents. Risk sharing and other forms of managed entry agreements are also becoming more frequent, in relation to the uncertain outcomes of the use of some products. Under such agreements companies are required to monitor the performance of their products and reimbursement will only be granted for responsive patients. The development of an electronic health system may come as a transparent solution for managing prescribing, enabling better overview and control, and may serve as real-time info on consumption, unmet needs and proper follow up of clinical guidelines.

Tarik Catic, MSc., ISPOR Bosnia and Herzegovina President

Patients registers as a tool to access new medicines

Access to innovative medicines is limited currently limited in CEE countries and different policies have been set to combat this. The main obstacle is budget limitations but also a lack of HTA implementation in decision making. Recently some improvements have been made except most payers base their decision criteria on budget impact.
New approaches in reimbursement of new/innovative and expensive therapeutic options should be high priority in order to improve therapeutic outcomes and patient satisfaction. Development of risk-sharing schemes, conditional reimbursement and follow up on therapeutic outcomes could be the chance to increase access to novel therapeutics.
Patient data and registries are collections of secondary data related to patients with a specific diagnosis, condition, or procedure, and they play an important role in post marketing surveillance of pharmaceuticals. Most frequently registries vary in sophistication from simple spreadsheets that only can be accessed by a small group of physicians to very complex databases that are accessed online across multiple institutions. Even when some epidemiology data exists in CEE countries, the data collected does not meet the requirements to follow up product performance and outcomes. Mostly, these data are general indexes, but through introduction of disease/product registries more detailed analysis could be performed. Registries can be associated with pay-for-performance agreements. In order to implement such registries, authorities could develop partnerships with industry, taking care of data confidentiality, ethical and technical issues.

Tanja Novakovic, MSc. Pharm President of the Pharmacoeconomics Section (SFE), Galenika a.d., Serbia

Big data in Health Care

Clinical data from randomised controlled trials represents the gold standard in evidence for medical decision making, however, these trials represent just a small subset of the data available, and are not necessarily applicable to real life practice.
Furthermore cost data often represents the most uncertain aspects of any health technology assessment, either because such data was not collected during these trials, or the trials were conducted in countries with a different healthcare structure, and therefore not applicable in other countries.
Data collected from ongoing clinical practice however is often cumbersome in size, difficult to interpret due to the lack of control groups and subject to deep privacy concerns. Realising the value of this data and overcoming these problems will soon help all healthcare providers better manage their budgets and greatly improve the quality and quantity of services they provide, by providing a sounder and more current information base for their decisions, and real time monitoring of the outcomes of their decisions.
Achieving this will require potentially difficult collaborations between the healthcare and pharmaceutical industries, but turning the emerging and massive streams of incomprehensible data into valuable information will revolutionise the way healthcare is delivered.

Janko Samardzic MD, PhD. Institute of Pharmacology, Clinical Pharmacology and Toxicology Medical Faculty, University of Belgrade, Serbia Health Insurance Fund, Serbia

Clinical effectiveness in decision making

In most countries of Europe, physicians have the exclusive right to prescribe medicines. Therefore, they play a crucial role in determining whether medicines are consumed rationally or not. However, in reality, physicians face different pressures, such as staying objective in the choice of treatment, being up-to-date with innovations in therapy, keeping the budgets, earning an income, etc. Thus, there are a number of measures to improve physicians' prescription behavior, such as prescription monitoring, prescription guidelines, requirements for prescribing by the international non-proprietary name (INN), financial, educational and information tools.
At least 24 EU Member States already have prescription guidelines. Guidelines consist of clinical prescription protocols based on evidence and may include financial considerations. In Austria, Belgium, Germany and Hungary these guidelines are compulsory, which are expected to be implemented in Serbia soon. Of course, combining different measures, such as electronic prescription monitoring and guidelines linked with electronic systems appears to be one the most effective way. The guidelines are mostly based on evidence gathered from randomized controlled clinical trials from phases I to III. These trials are very good at assessing efficacy, but it should be taken into account that these trials are not without substantial biases, such as selective and information bias. Furthermore, efficacy is not the same as effectiveness. Efficacy means if the drug works in ideal circumstances, and it could be assessed by clinical trials of phases II and III. Effectiveness means if the drug works in real life and cannot be evaluated in controlled trials, but through observational studies into phase IV. On the other hand, according to the procedures of Health Insurance Fund, pharmacological report represents a part of application file for reimbursement lists. The report has to illustrate the completed pharmacological, therapeutic and social value of drug and it should be based on clinical data and guidelines, in order to improve the clinical effectiveness of the new medicine vs. comparator.

Eldon Spackman, PhD, Research Fellow, Centre for Health Economics at University of York, United Kingdom

Should off-label agents be used as comparators in Health Technology Assessments?

HTA bodies provide guidance and advice to improve health of the population. Their remit is to compare clinical and economic evidence toprovide recommendations on reimbursement of new technologies.The value of a new technology is dependent on the costs and outcomes of current practice. To improve overall health a new technology must improve health more than all the treatments it will displace due to its costs.
In practice, treatment alternatives to the new technology may be off-label. As an example, twenty-four NICE technology appraisals from 2008-2012 included an off-label comparator and in 8 (33%) of these appraisalsthe new technology was rejected. Some question whether new technologies should be compared to off-label agents since if the new technology is found to compare unfavourably, then it will be rejected in favour of the off-label agent. NICE does not have remit to recommend off-label agents however rejection of a new treatment may be considered an indirect recommendation for use of the off-label agent.
Cost-effectiveness principles suggest that all possible comparators, even off-label agents, should be included in an evaluation. Pragmatically there may be some issues: some HTA bodies may be restricted by remit, off-label agents may have less developed evidence and there may be some risk or liability involved in an indirect recommendation. Cost-effectiveness methods are ideal for understanding these issues.

York Zöllner, Hamburg University of Applied Sciences, Germany

Merging theory and practice: Germany’s approach to evidence-based decision-making

Since the advent of the new drug pricing & reimbursement law from AMNOG in 2011, the German pharmaceutical market has ceased to be a “free pricing” market for patented drugs. While there were earlier attempts to regulate drug pricing, these were highly debated and relatively short-lived.
In order to portray and interpret the current paradigm in pricing and reimbursement decision-making for newly launched drugs, and discuss whether and how the former can claim to be a reasonable compromise between scientific judgement and business practicalities:

• Market figures (size, trends) will be presented and interpreted (price/volume decomposition, international comparison).
• Regulations will be classified into structural and process-based elements, incentive (dysincentive) based measures to foster innovation (discourage me-too’s) as well as straightforward interventionist measures to contain or cut expenditures.
• The record of payor decisions taken so far including the underlying reasoning will be examined; similarly, as manufacturer reactions thereto will be interpreted.
• The role of health economic evaluation as well as other elements of economics – e.g. negotiations, embedded into a game theory framework – will be addressed.
• An outlook regarding potential future domestic developments as well the scope for European harmonization will be given.

Authors: Alin Liviu Preda, MD, Health Outcomes Specialist, GSK Romania Ciprian-Paul RADU, MD, PhD, Market Access Manager, Roche Romania

HTA System in Romania – From Past to Future

The transition from a Semashkoto Bismarck model health care system began for Romania after 1989, but, in practice, “the big reform” started in 1998 with the development of the social health insurance scheme and the organization of National Health Insurance House. Although in 1992, following a study financed through World Bank, it was identified the need for developing a Health Technology Assessment (HTA) system in Romania, no practical decision was taken because of lack of involvement at political level. During the period 1992-2002 some form of mentorship offered by Alberta Heritage Foundation Canadawas performedin this area and HTA was brought in the attentionof the authorities (without any initiation of a Governmentalprogram), but HTA remained a subject onlyfor the academic and research purposes.Very little words are to be said about HTA in the silent period of 2002-2011, then,in 2011, NICE International (contracted by the Romanian MoH) made a report evaluating the basic health care package, where among many other recommendations included a strong point on building HTA capacity and guidelines specific for Romania environment. Consequently in June 2012, MoH published a new HTA methodology to be used for approval of new drugs or new indications, considering the fact that no new drugs /indication were approved in Romania from 2008. The Romanian HTA system is based on a scoring card system, taking into consideration 6 aspects: (1) HTA reports from France and (2) UK; (3) number of EU countries where the submitted drug is reimbursed; (4) relative effectiveness; (5) relative safety and (6) relative Patient Reported Outcomes. Beside this, a simple Budget Impact Model is required as an orientation instrument used for imposing restriction for reimbursement. The dossiers evaluation is done by the new HTA Unit created within the Ministry of Health, the Specialty Commissions within the MoH and the National Commission for the coordination of the Specialty Commissions, which in summarizing the provided evidence for the decision making used some of the EVIDEM Collaboration tools. The first wave of HTA dossiers was submitted in June and July 2013, arriving at over 200 dossiers by the end of 2013. By February 2014, most of the dossiers submitted have been evaluated by the HTA Unit (167 HTA dossiers) and the MoH has made public results for these submitted dossiers, which include both new molecules and new indications. There has been an acceptance rate (score above 6 points) of about 80% and the draft of the new reimbursement list was published for public debate.The final publishing of the List in theOfficial Gazette is still pending because of the changes in the political environment and because the authorities and Pharma industry are still discussiong on the alignment of the new List with the clawback taxation and price legislation update in Romania. Finally, we can appreciate that even the HTA process is working in Romania from the technical perspective, there are still a lot of things to be done in terms of health policy and decision making based on HTA, because the public reimbursement remains a highly political dependent process which leads to large delays in the access of patients for new therapies. Without a strong commitment from the political officials to integrate the reimbursement process with the entire health care reform, there is a risk for the patients to continuously lack access to better technologies.

Brian Godman, Institute for Pharmacological Research ‘Mario Negri’, Milan, Italy Prescribing Research Group, University of Liverpool Management School, Liverpool, UK Division of Clinical Pharmacology, Department of Laboratory Medicine, Karolinska Institutet, Karolinska University Hospital, Huddinge, Stockholm, Sweden

Evidence requirements in pharmaceutical policies across Europe: implication for the future

'Pharmaceutical expenditure has been rising steadily across Europe during the past decade. It has recently fallen following the economic crisis, but due to rise again due to ageing populations and the continued launch of new premium priced products. This has necessitated ongoing reforms and initiatives across Europe to help ensure the ideals of equitable and comprehensive healthcare. Measures include new models to optimise the utilisation of new drugs incorporating horizon scanning pre-launch as well as critical evaluation of the health gain alongside requested prices given that only a minority of new drugs are truly innovative. In addition, evaluation of potential managed entry agreements to enhance reimbursement as well as post launch follow-up activities. These include monitoring the prescribing of new drugs against agreed clinical guidance, as well as assessing their effectiveness and safety in routine clinical practice. Initiatives for established drugs include measures to improve their rational use with the help of formularies such as the ‘Wise List’ launched by the regional Drug and Therapeutics Committee in Stockholm, Sweden, combined with extensive communication and education programmes. In addition, encouraging INN prescribing to reduce patient confusion, especially if patients are dispensed different branded generics on each occasion, as well as enhance their use versus originators once multiple sources become available. This alongside transparency in the pricing of generics to obtain lower prices. These measures, together with numerous examples from across Europe, will be discussed in the presentation. The objective is to try and ensure sustainable universal access for healthcare across Europe including Serbia

Ruth Lopert, PhD, Department of Health, Principal Adviser, Therapeutic Goods Administration, Australia

Pharmaceutical expenditure in Serbia - current and future scope for HTA

In Serbia, although per capita spending is low, pharmaceuticals consume a high proportion of health expenditure. A review of current reimbursement lists and expenditure patterns sought to identify opportunities to improve value for money.
While in the short term major gains are possible through improving procurement mechanisms for off-patent, ‘essential’ medicines, better value for money from patented ‘specialty’ medicines may also be achieved through adapting the results of health technology assessments (HTAs) conducted in other, selected jurisdictions. Rather than setting wholesale prices by external reference pricing, the application of ‘de facto’ HTA - referencing prices and listing conditions in another jurisdiction with established HTA processes, and determining a cost-effective price by adjusting for relative per capita GDP - could guide both listing decisions and price negotiations. For drugs yet to be listed, the de facto HTA method could be applied to set conditions of listing and as a basis for declining listing of drugs for indications not considered cost-effective or clinically appropriate there.
Although specialty medicines currently represent a small proportion of overall expenditure, utilization is growing rapidly. Over the medium term building Serbian HTA capacity will be increasingly important as high cost drugs become a growing proportion of the total drug budget, particularly as off-patent prices are reduced. The development of robust, evidence-based HTA processes will be important to improve Serbia’s capacity to:

1. Assess value for money in the selection, listing and pricing of medicines and
2. provide a means of prioritizing expenditure in a resource limited environment.

Dragana Baltezarević, M.D., Health Insurance Fund, Serbia

Reimbursement of medicines in Serbia

The Serbian healthcare system is based on the principles of equality and solidarity. Citizens pay health insurance contributions as a percentage of their income while healthcare services are used according to their needs. Serbia as the poorest country in Europe, disposing of only 300€ per capita for funding the total healthcare system, spends only 50€ per capita for medicines. The Serbian Reimbursement List is still an integral part of the Rulebook on Reimbursement list which is published in the Official Gazette. On the other hand, the new “Rulebook on criteria for listing” expects to be published in May 2014.
The general criteria for listing are: The RFZO financial plan. Public health importance. Pharmacotherapeutic and pharmacoeconomics aspects.
For the first time the “Rulebook on criteria” recognizes special agreements and shorter application procedure for generic medicines. The newly established Central Committee for medicines determines which medicines will be listed, based on the opinion of the Republic Expert Committees and the Committee for evaluation of pharmacoeconomics reports. In addition, the current structure of the Serbian Reimbursement List, as well as the new “Rulebook on criteria” enable price reduction, cost control and adoption of the pharmacoeconomic priniples during the decision-making regarding the reimbursement of medicines. The main goal is still budget reallocation and savings, for listing more new medicines.

Biljana Kozlović, M.D., Assistant Director for medicines, Health Insurance Fund, Serbia

Outcomes of the Central Public Procurement of Medicines in Serbia

As a part of the World Bank Second Serbia Health Project preparation, the Republic Health Insurance Fund (HIF), as central purchasing body, has successfully completed centralized procurement of the most expensive medicines for hospitals and primary health care centers with generated savings of close to 3 billion dinars (25 million EUR). This system, introduced as a pilot project in 2013, has helped to reduce prices on drugs by an average of 27%, compared to the previous system. In the first and the largest centralized public procurement of medicines from the Drug List B, 22 bidders competed for 183 lots of medicines. Framework agreements were concluded with the total of 15 suppliers and manufacturers that offered the lowest prices of medicines and which, based on defined prices of medicines, are concluding individual supply contracts with health care facilities till the end of 2014. The framework agreements were concluded after open tender procedure, with one supplier for each separate lot. Their enforcement is mandatory for all health care facilities, and the framework agreements have a validity period of 12 months. This is only the beginning of the public procurement reform in the health sector that will be the backbone of the new World Bank Second Serbia Health Project.