The 7th Krakow Symposium HTA is another edition of the event on the subject of systemic changes in the field of health care in Poland.

We present you with a full summary of individual speeches.
(click on the button to move to the session)

DAY I

DAY II

DAY I: SESSION I

WHAT NEW DEVELOPMENTS IN REIMBURSEMENT CAN WE EXPECT?

Speakers: Roman Topór-Mądry, Krzysztof Kopeć, Magdalena Cysewska, Katarzyna Kolasa
Chairman: Tomasz Bochenek

AOTMiT’s role in the healthcare system

In his presentation, the President of the Agency for Health Technology Assessment and Tariff System (AOTMiT) summarised the Agency’s goals and plans for the future. A total of 124 recommendations and 50 opinions of the Agency’s President on terms of Emergency Access to Medicines (RDTL) were issued by 31 December 2018. The Agency which serves as an expert institution for the Minister of Health within the system undertakes actions concerning the reimbursement decision-making process. In 2018, a department for health services was developed in the institution and some new remote branches were created in Krakow and Wrocław. Since last year, they have begun to provide opinions on clinical guidelines, which is both the current and future main goal of the Agency. Health policy programmes which function in the form of health programme recommendations and which have been successfully continued for over 1.5 year also constitute a new element. Among current challenges, the latest one is connected with a law amendment enabling the Agency to perform analyses and examinations in terms of the HTA Tariff System and health services as well as joint clinical assessment on the international level.

ATOMiT experts point to the following challenges/projects which the Agency faces:

• HTA:
  • a joint project with EUnetHTA, the goal of which is to specify and implement sustainable and balanced cooperation model in the field of HTA on the European level.
  • “Impact HTA” in cooperation with London School of Economics. The project analyses method proposals, tools and guidelines streamlining decision-making
  • “Challenges in the HTA” workshops,
  • “Fair and Affordable Pricing” project, the aim of which is to improve and simplify the access to effective and safe health technologies while preserving cost-effectiveness,
  • developing a scheme which would simplify international cooperation and enable joint pricing negotiations in terms of reimbursement of medicinal product and exchange of information.
• Health needs, the main initiative for which is Global Burden of Disease in cooperation with Health Metrics Evaluation Washington University. The project pertains to the use of the tool for quantitative determination of health deterioration as a result of a disease, injury and risk factor (Disability-adjusted Life Years ratio – DALY), in order to improve the healthcare system and eliminate disproportions.
• Treatment cost analysis. One of the elements of this project is cost assessment versus patient’s clinical condition.
• Numerous research activities in terms of development of methods for choosing effective and economical solutions in healthcare.
• Expanding the scope of actions pertaining to public health.
• Training courses.

Perspective of reimbursement change in the context of the National Drug Policy (PLP) and the Reimbursement Mode for Development (RTR)

President of The Polish Union of Pharmaceutical Industry Employers presented the intended schedule of work in terms of the “National Drug Policy” document, together with the Union’s proposals concerning amendments in the Act on reimbursement. From the experts’ point of view, introducing scientific advice in drug marketing authorisation process by the first quarter of 2019 appears to be of great importance. It will improve the marketing authorisation process through establishing the role of scientific advice on the drug development planning stage (including designing clinical trials) and in manufacturing of medicinal products (fine-tuning the marketing authorisation process for medicinal products). For the second quarter of 2019, the possibility of assessment of reimbursement applications in English is planned, as part of international projects aiming at simplifying and accelerating international cooperation. It seems to be significant concerning the document and changes in the reimbursement system that there be a change in frequency of reimbursement lists, leading to the manufacturer gaining the possibility to change as a reaction to the draft reimbursed drugs list (the so-called pricing possibility). The remaining proposed changes are:

• Amendment of regulations on the total reimbursement budget. It is proposed to determine the actual budget for reimbursement at about 16.5%-17.0 and to introduce a full payback. Moreover, financial resources of the total budget for reimbursement, which were not used in a given year, should additionally increase the financial resources in the following year.
• Optimisation of the reimbursement system for medicinal products – other specific solutions (e.g. concerning patients’ co-payments):
  • moving drug groups which so far had not been covered by the total budget for reimbursement to the total budget for reimbursement, along with adequate financial resources,
  • patient co-payment – regular decreasing of patient’s engagement in the financing of reimbursed drugs.
• Introduction of the Reimbursement Mode for Development (RTR) which enables providing support to the Polish pharmaceutical sector through pro-development pricing and reimbursement policy. Furthermore, implementation of the Reimbursement Mode for Development will stimulate the increase in the activity of companies building the ecosystem of a pro-innovative pharmaceutical industry in Poland. Reimbursement preferences should be granted to companies which have the Partner of Polish Economy status. Expectations of local drug manufacturers concerning RTR:
  • Automatic renewal of reimbursement decisions.
  • Partners of Polish Economy exempt from payback.
  • Differentiation of applicants in terms of partnership with Polish and EU economy.

Trends in financing drug programmes on the eve of their 7th birthday

The National Health Fund’s (NFZ) perspective is an indispensable part of reimbursement changes, including also the financing of drug programmes. The presentation summarised the information concerning the financing of drug programmes and their evolution since 2012.

The number of drug programmes increased from 44 in 2012 to 91 in 2018 and the number of reimbursed active substances from 68 to 134. Over the years, the number of patients has also increased significantly, from over 47,000 to over 136,000. The aforementioned growth is linked with increased funds allocated to financing of drug programmes. The presented summaries clearly indicated an increase in NFZ’s expenditure on drug programmes from about 898 million to over 3.8 billion.

According to the drug reimbursement amount, most of the spending on reimbursement in the years 2012-2018 was incurred on drugs programmes concerning such indications as: chronic myelogenous leukemia, breast cancer, multiple sclerosis, hepatitis C virus (HCV), kidney cancer, rheumatoid arthritis (RA) and juvenile idiopathic arthritis (JIA), multiple myeloma and colorectal cancer. Drugs in drug programmes with the highest reimbursement amounts in the years 2012-2018:

1. TRASTUZUMAB
2. IMATINIB
3. OMBITASVIR + PARITAPREVIR + RITONAVIR
4. LENALIDOMID
5. LEDIPASVIR + SOFOSBUVIR
6. ADALIMUMAB
7. AND ETANERCEPT, INTERFERON BETA – 1B, PEGINTERFERON ALFA – 2A, SUNITINIB, BEVACIZUMAB, NIVOLUMAB

The number of oncological drug programmes increased from 12 in 2012 to 31 in 2018, while the reimbursement amount increased from 425 million to about 1.8 billion. Drugs in oncological programmes with the highest reimbursement amounts in the years 2012-2018:

1. TRASTUZUMAB
2. IMATINIB
3. LENALIDOMID
4. BEVACIZUMAB
5. SUNITYNIB
6. NIVOLUBAB

Future of reimbursement models in the digital health era

Digitalisation in healthcare is a topic which has been rapidly introduced in the discussion pertaining to the future of healthcare and reimbursement system. Digital solutions in healthcare support treatment effectiveness and searching for the efficiency in the system. In health economics, we primarily deal with real-world data (available primarily on the basis of historical data analyses and prospective studies). When it comes to medical devices, real-world evidence (RWE) is used more frequently than data from randomised control tests (which very often are not performed at all). FDA has established that real-world evidence will be used in the regulatory context, and furthermore payers also try to define the scope of reimbursement based on the analysis of these retrospective data resulting from the patient’s electronic records. Currently, the regulation on free movement of non-personal data is being developed in Europe.

Public health aspects, which are closely related to digital health, need to be mentioned as well. Ageing of the society results in the need to look for new solutions and savings in healthcare expenditure. One of the possible solutions is digitalisation of healthcare aimed at supporting the effectiveness of treatment and preventing medical staff shortages. London may serve as an example. A smartphone application was developed there, which substitutes the access to the GP (there is a video conference with AI GP instead).

Digitalisation in healthcare is very diversified – it is not only telemedicine but also, among others, mobile applications. There are over 300,000 mobile applications concerning healthcare. NHS offers 70 applications supporting patients in their everyday fight against disease (research has proved effectiveness of these applications). More and more companies, such as Bayer or Novartis, establish separate organisations aimed at investing in digital solutions (including research grants and connecting with start-ups in the digital health area). Increasing availability of data will soon make us move only within the scope of digitalisation. Nevertheless, most of the data is not structured.

DAY I: SESSION II

HOSPITALS AND THEIR FINANCING

Speakers: Iga Lipska, Dominik Dziurda, Maja Więckiewicz, Michał Bedlicki, Daniel Rutkowski
Chairman: Jacek Walczak

Hospital-Based HTA

The goal of the project is Hospital-Based HTA methodology implementation through creating HB-HTA pilot units and HB-HTA nets supporting them. This methodology is aimed at increasing the capacity to manage the healthcare system by providing the stakeholders with systematic and clear innovative (non-drug) health technology assessment, both on local (hospitals’ managements) and national level (public payer). The goal of HTA is to systematically evaluate the attributes and effects of using health technology (of a drug, medical equipment, medical procedure), for therapeutic, diagnostic or preventive purposes, based on scientific evidence. HTA has a well-established advisory or binding position in the decision-making process concerning reimbursement of health technology in many countries across the world, including Poland. HB-HTA is a health technology assessment adopted to the specific hospital context and used in order to support the decision-making process pertaining to investments in evaluated technologies The three-year HB-HTA-PL project is financed by The National Centre for Research and Development and realised by the NFZ Consortium, The Institute of Cardiology and Lazarski University. The expected results of the project are as follows: know-how concerning the current status of health technologies implementation in Europe and Poland, organisational basis for HTA hospital units/HB-HTA nets in Poland, HB-HTA and HB-HTA nets in Poland hospital units model, HB-HTA system pilotage in Poland. Details linked with the project implementation were also presented.

Guaranteed health services package – AOTMiT perspective

One of the achievements in AOTMiT work connected with the guaranteed health services package is complete care in breast cancer treatment. There are also plans concerning complete care of the patients suffering from lung cancer, colorectal cancer and prostate cancer, as well as changes in the field of hematooncology. Evaluation of rehabilitation services, although necessary, appears to be a slightly different area. Financing here is provided from a wide range of overlapping sources regulated by different legal provisions. The total budget for rehabilitation (apart from spa healthcare) offered by NFZ amounts to PLN 2.35 billion. There are also a lot of other sources of its financing in the system. This area could definitely be optimised. Financing is distributed among inpatient centres (of specialist and systemic rehabilitation), day care centres, outpatient physiotherapy and outpatient medical rehabilitation care. Rehabilitation financed by the NFZ is subject to various organisational and procedural limitations (including waiting times and availability of rehabilitation services). The concept of changes consists of developing eligibility criteria for health services, defining places for the provision of services, determining intervention profiles, specifying conditions for the provision of services, as well as defining quality ratios. It is very important to define the patient’s health needs clearly and to determine what health service parameters are appropriate for them and under what conditions certain type of the service should be provided. Basic physiotherapeutic care should be available for each patient within a developed system.

Cost accounting standard – current state of play

Cost accounting is extremely important for AOTMiT due to tariff processes. It is crucial to unify the approach, registration and collection of costs. Cost accounting has different recipients/users in the healthcare system (division managers, directors, founding entities, AOTMIT, Ministry of Health/NFZ). Its purpose is to improve management of individual entities, as well as the entire healthcare system, by providing the Agency with highest-quality, detailed information, which should enhance tariff processes.

The goals of cost accounting are linked with the creation of decision bases, control of production processes and of the achieved effects, as well as examination of changes in the company’s structure and providing information to internal and external recipients. Currently, it can be noted that there is a significant lack of integration of medical and administrative systems in the case of healthcare providers, lack of collected data harmonisation and process automation, and moreover there are some important differences in the level of preparation of units for the planned changes (i.e. implementation of the cost accounting standard). The cost accounting standard does not only consist in the collection of cost/accounting data, but also a whole system, thanks to which directors can analyse and monitor the financial condition of their units on an ongoing basis. The main assumptions of the draft regulation on recommendations regarding cost accounting standards are: cost-effectiveness of cost information, reality (possibility of implementation of the proposed solutions) and the introduction of two-stage implementation of cost accounting standards (2 regulations). The first regulation presents a “simple” version of the list of cost accounts and calculations, and enters into force on 1 January 2020. The second one, presenting the target model of the detailed list of cost accounts and calculations, will be introduced later, probably in 2023. At the same time, a law amendment concerning the eligibility criteria for healthcare providers, as well as gratuities for the earlier introduction of detailed requirements, was introduced.

DAY I: SESSION III

NON-DRUG HEALTH TECHNOLOGIES

Speakers: Grzegorz Mączyński, Jan Szulc, Wojciech Szefke, Łukasz Szajda, Magdalena Piegat
Chariman: Izabela Pieniążek

Ecosystem for the development of non-drug technologies

Currently, the boundaries between drugs, medical devices or tissue transplants are becoming blurred. When developing new technologies, one should also think about how they will be classified in a formal and legal way. This classification is becoming really difficult. New 3D drugs and drugs whose active substance comprises of stem cells taken from the patient might serve as an example. Nowadays, computerisation and digitalisation of the healthcare system as well as the development of telemedicine solutions and health condition monitoring are becoming more and more common. These processes give rise to new opportunities for medical devices. Medical devices are generally faster and cheaper to introduce to the market. At the same time, there are also certain threats to innovative medical devices. Start-ups from the areas of life sciences, the health sector and biotechnology have the least regular revenues. Medical devices are supported by a smaller number of studies, and thus less evidence, which can be problematic when the manufacturer applies for reimbursement. Additionally, the dynamic growth of the IT market is connected with greater competition risk. The difficult regulatory environment (including different national circumstances) poses a challenge as well.

What will the new law on medical devices bring about?

The changing reality on the medical devices market requires adjusting of the legal environment. Next year, we are going to face a major reform of the legal system concerning medical devices, in connection with the entry into force of the EU regulation which pertains to a larger group of medical devices. The URPL (the Polish Office for Registration of Medicinal Products, Medical Devices and Biocidal Products (URPL) is preparing a law which will implement these provisions into the Polish legal system. The law will be of a strongly technical nature. A public register/list of devices entered into the market or imported into Poland is to be created. A crucial change will be also linked with the regulations concerning advertising of medical devices. The main goal of these provisions will be to identify harmful and negative phenomena, limit unfair advertising, specify what needs to be included in certain product advertisement (and what does not). In addition, it is planned to extend the legal requirements for the health services provided with the use of medical devices. Other changes will include: the possibility to use English in communication with the office and in documentation for professional users; the change of criminal-law provisions from penalties provided for in the Criminal Code to administrative penalties, as well as the possibility to allow reprocessing of single-use medical devices (destined for export).

Modern health technologies in the health benefits package

Legal changes concerning the medical device market appear to be inevitable. There is no single market of technologies and medical devices. There are 6-7 product markets (hospital market, procedures contracted separately, treatment equipment, market of the medical devices included in the reimbursement list, medical devices procured based on an order, open market, telemedicine). A legal course of action is possible only for devices from the reimbursement list, but there are no legal regulations for the hospital market/medical devices procured based on an order. Treatment units manage to gain knowledge about what is available on the market, partially in the form of a technical dialogue. 3 MD financing models (French, Italian and German) have been analysed in the presentation, The German model appears to be the most beneficial one for our market, as it gives industry the opportunity of introducing pilot projects for hospital technologies in the chosen centres. The best stimuli for creating/generating/supplementing the guaranteed health benefits package is the introduction of co-payments to primary healthcare services, to AOS (Outpatient Specialist Care) and to the hospital market, or supplementing NFZ with funds in a different manner. The problem consists in a very low healthcare contribution (as compared to the neighbouring countries). Therefore, the preferred action is linked with defining what model of co-payment we would like to adopt. Poland is the only country in Europe without additional health insurance system. The state ought to create horizontal solutions in this area.  

Creating modern health technologies with government funds

The institution that deals only with new technologies and their financing is The National Centre for Research and Development (NCBiR). The majority of funds for the development of new technologies (i.e. R&D actions) comes from the European Union. For the years 2014-2020, our government has negotiated EUR 82.5 billion. It should be noted that the eligible costs of the project may include gross remuneration, subcontracting, test equipment costs, intangible and legal assets. Financing is easily accessible, we only need to reach for it. The Kozminski Business Hub initiative, which supports companies development, may turn out to be really helpful at that point.

Modern technologies from the patients’ perspective

In the discussion concerning modern solutions entering the market of medical devices, we also consider preferences of our Patients. According to the surveys, many patients are ready to “replace” doctors with advanced technologies equipped with artificial intelligence tools. As the PwC report “Patient in the digital world” indicates, almost 60% of patients from Poland and the region of Central and Eastern Europe are interested in telemedicine solutions. The medical system in Poland is not yet well developed and appears to be rather chaotic. Stoma patients, whose number in Poland is estimated to be about 50,000, may serve as an example. This group is constantly growing and has a constant need for contact with specialists in various aspects. The best solution in such cases would be remote contact with a practitioner. The reimbursement amount for the equipment for stoma patients has not changed for 14 years and equals PLN 300 monthly. Therefore, the limits do not enable access to modern equipment. It would be thus incredibly significant to introduce e-orders and advice that could be provided on-line. We need a patient-oriented system which focuses on the well-being of the patient and aims to cure or improve his/her life quality.

DAY II: SESSION I

MULTIPLE-CRITERIA DECISION ANALYSIS IN THE REIMBURSEMENT OF ORPHAN DRUGS – WHOSE PREFERENCES?

Speakers: Gabriela Sujkowska, Stanisław Maćkowiak, Jolanta Sykut-Cegielska, Maciej Niewada, Piotr Szymański
Chairman: Marcin Czech

DISCUSSION (opening the session):

How to behave in the case a child is diagnosed with a rare disease (e.g. cystic fibrosis) who has a chance to be treated with a drug which is unavailable or not reimbursed in Poland. What do we do in such a situation? Such questions arise more and more frequently. Knowing a specific drug, it is important to provide information about its current status (whether it has already obtained marketing authorisation) and whether any clinical trials are being conducted, and, if so, is there any chance of participating in such a trial? Sometimes people move from one country to another to be covered by insurance which provides access to a specific drug. There are also patient organisations which help patients buy drugs that are unavailable in Poland or are not reimbursed. Organisations try to talk to companies that own these drugs and run so-called charitable therapies. They also try to discuss systemic solutions with the Ministry of Health (MZ). Unfortunately, legislative problems often arise in this area. From the systemic point of view, by trying to find a treatment for a disease, we can check whether the application for public funding has been made, what stage in the refund process it is at, whether we can move towards emergency access to medicines (RDTL), or targeted import. However, the first key step is to contact a clinical expert who understands and knows the disease.

Multi-criteria decision analysis (MCDA) in the evaluation of orphan drugs. Whose preferences?

Rarity of occurrence (of the diseases) generates difficulties in obtaining high-quality data and we often deal with limited knowledge. Hence the idea to use the multi-criteria decision analysis, which is increasingly used in pharmacoeconomics in response to the lack of an appropriate tool that would measure the additional benefits of using a given health technology and would take socio-economic aspects into account. We are looking for new solutions for orphan drugs, e.g. as part of joint negotiations within the Visegrad Group (V4+), Horizon Scanning, RSS, cost-effectiveness threshold and ICER value, prices of drugs related to purchasing power dependent on GDP, registers / RWD / RWE.

Methods of measuring preferences and the choice of respondents – challenges and pitfalls

When we speak of MCDA, it is important to establish which preferences we want to follow. The preferences are very specific. Involving the payer / decision-maker in the process is the prerequisite for the successful use of MCDA on a daily basis in reimbursement decisions. It is very important to determine who should be the source of the preferences (decision-makers or their representatives, experts, patients or their representatives, the society). The choice of the MCDA model must be practical and must depend on the list of respondents. Respondents, on the other hand, must be competent from the perspective of the entity using MCDA to support decision making.

Rare diseases in the agency’s perspective

Rare diseases have a prevalence of less than 40 cases per 100,000. A drug’s centralised (EU-wide) marketing authorisation is not equivalent to it being reimbursement. The drugs obtaining marketing authorisation most commonly are those affecting the smallest number of patients (often cancer diseases). Of the 149 orphan medicinal products with MA granted by the European Medicines Agency (EMA), approximately 30% are reimbursed in Poland. A known and already widely used substance may apply for the OD status provided that the manufacturer indicates a new, rare and not yet approved therapeutic indication. The most important aspects are whether the product offers an additional health benefit, what the cost of the additional benefit it (and whether that cost is acceptable) and what financial consequences the reimbursement will have. In the USA, drugs may also obtain orphan subset status (subpopulation). Clinical trials relating to rare diseases are subject to many limitations which later result in estimation uncertainty. Many drugs are authorised on the basis of limited clinical trials. It is important to remember that the fact that a drug is reimbursed in another country does not mean that it will be reimbursed in our country. There is a tendency to support the differentiation of therapies and to seek a different approach to financing of orphan drugs. A very large number of factors impact drug availability and possible delays in their introduction. Certainly, clinical registers are needed. It is important to move towards finding solutions that offer greater opportunities for those therapies which are indeed very effective, very safe and are characterised by great potential.

Availability of orphan drugs from a clinician’s perspective

Congenital metabolic defects are (ultra) rare diseases. The treatment in this case is very difficult. In protracted illnesses, proper homeostasis of the system is preserved. It is enough to support the defence mechanisms of the human body in order for the treatment to be effective. In a congenital metabolic defect, the homeostasis is disordered. The treatment methods are therefore focused on attempting to artificially modify the environment. Access to the FSMPSs and OMPs is based mainly on the requests for individual target import and emergency access to OMPs (RDTL). On the other hand, there is no temporary admission procedure. Often, treatment is not available and applications are blocked. The decisions made regarding the reimbursement of therapies that have been implemented in the framework of the requests for target import are negative. So the question arises as to the point of diagnosing diseases when treatment is not available. The increasing number of rare diseases, the development of new health technologies and the high cost of orphan drugs constitute a burden to the health budget. Clear criteria for pricing and reimbursement decisions are therefore needed. Access to orphan drugs in Poland compared to other European countries is definitely limited. The situation of total expenditure per capita is similar (we are classified at the very end of the ranking). Rare disease therapies are not cost effective. From the clinician’s perspective, the following conditions should be applied to the financing of treatment for rare diseases: severe course of disease, inhibiting the progression of the disease through treatment, lack of the alternative treatment. At present, in the case of many rare diseases, the treatment exists, but is not available. There is therefore an urgent need for systemic solutions which will improve the availability of orphan drugs (for example using MCDA).  

Rare Diseases and Orphan Medicinal Products – Challenges for Poland

There are several formulas for the availability of drugs used in rare diseases: pharmacy reimbursement, targeted import, drug programmes, emergency access to medicines RDTL. None of these forms are fully effective. In drug programmes, the time between the reimbursement decision and the availability of the medicine is often extended. Targeted imports were practically cancelled (except for continuation of treatment). Applications for targeted import concerns health and life-saving medicines and cannot wait. Nevertheless, such decisions are currently postponed until the President of AOTMIT has given his opinion. This can take even one year. During this time, the patients do not have access to medicines or foodstuffs for particular nutritional uses that are necessary for their health and life.

DISCUSSION (only opinion/speech of Piotr Szymański)

We are now discussing something which effectively is a system for limiting access to medicines. The structure of the system is arranged in such a way that first we have limited access to the market at the level of MA, and then at the level of accessing reimbursement. It is hard to be a guard. Looking globally at the access to health technologies: 40% of the global health budget is spent on 5% of the American population. The US is also where new technologies are being developed. It is also worth remembering that access to medicines is largely regulated by the EMA and the FDA. Apart from the enthusiasm for new health technologies and the understanding of patients’ needs, it is also important to understand the market forces that are behind the transition of new technologies. Drugs are very expensive. The so-called orphan subsets, i.e. subgroups of frequent diseases, are now sometimes qualified as orphan drugs. It is discussed that large populations of patients should be divided into orphan subsets, which will result in very high treatment costs in these small subgroups.
When talking about responsibility and rare diseases, it is important to mention surrogate endpoints and the minimum clinically significant difference at the endpoints. The last but very important thing is to agree on decisions relating to the exclusion of patients and the discontinuation of treatment if it is ineffective or does not bring any noticeable benefits.

DAY II: SESSION II

MODERN DATA MANAGEMENT IN HEALTHCARE

Speakers: Piotr Szymański, Dariusz Dudek, Maciej Banach, Artur Fałek, Wojciech Szefke, Iga Lipska
Chairman: Mateusz Nikodem

Managing uncertainty of evidence in the post-marketing phase

After the pre-marketing trial phase, a given health technology is placed on the market, then it is subject to health technology assessment and may be reimbursed. When talking about pre-marketing research, the uncertainty of evidence must be taken into account. The benchmark we refer to when we introduce a technology to the market are randomised clinical trials. Very often, they are the basis for obtaining marketing authorisation and marketing (but not necessarily for oncological medicines or medical devices). Many examinations are also based on the endpoints. The results of randomised examinations are often not correct (even if only due to their insufficient power or small bias), and the replication of the results of the trials is necessary for obtaining marketing authorisation for drugs is very unlikely. All this affects the approval of drugs. In the case of faster approval (as in the case of orphan drugs), the risk of occurrence of undesirable events in the post-marketing phase and various safety alerts increases. All of this is subject to uncertainty. In addition, in the case of approval of trials on the basis of limited evidence, very often there is no further follow up (subsequent, complementary trials). So, how can we manage uncertainty? It can be done through the registers and a post-marketing supervision. On the other hand, the inevitable uncertainty about evidence must be accepted and data collection must continue. At the same time the technology should not be accepted and should not be subjected to HTA. It is important to leave space for observation in real time before it is widely implemented.

Modern technologies in the hospital – how should they be financed?

Many people believe that as soon as a new drug is introduced, it will be immediately available to the patient. In fact, this process is much more complicated and requires a lot of verification. In order to do so, Poland needs proper communication between the Ministry of Health (MZ), AOTMiT and the National Health Fund (NFZ). The financing process begins with clinical trials, through grants (also independent) and targeted projects, to financing by the National Health Fund. The time between the examination and the financing is usually long. There are devices in use that should be present in everyday clinical practice, but there are problems with their availability. The ultimate problem is usually the public payer and the public money used to make it widely available and in sufficient quantity. Modern procedures and technologies (like the tricuspid valve) are implemented, but their availability remains insufficient against the background of other countries. The bicuspid valve is available in 35 countries, but the method of their financing and regulation (and thus the accessibility for patients) is slightly different in each country. Our financing system does not provide for different fees depending on the type of device used. The implementation of new technologies should be considered in the long term. We cannot measure only the direct costs, but also the indirect costs associated with the implementation of technology. The key result is the overall impact on the population in a long-time horizon (in accordance with value-based healthcare principles). All of this is difficult to achieve when based on the budgeting of individual treatment units.

Modern (non-)drug health technologies

Why do we have a problem with rare diseases in Poland? In fact, we do not even know how many patients with these diseases we have. The relevant data are not being collected and appropriately digitised. Data determines everything. They should be used and analysed in detail to determine the annual incidence/mortality trends, and based on which, in turn, we could be able to say what the needs (in the context of modern technologies) are. Only “big data” gives us the power to make the right decisions.

The Polish Mother’s Memorial Hospital Research Institute focuses on artificial intelligence, which can be used in primary prevention projects, where artificial intelligence helps to educate doctors. The second important project of the AI at the Institute helps women to live healthy lives in the period around and after the menopause. The introduction of teleCTG (i.e. the possibility of evaluating the foetal ECG), as well as additional screening tests, has enabled us to diagnose various rare diseases and implement appropriate treatment.

It is very difficult to adapt the facility to full digitisation. Often there is a shortage of computers and appropriate programs and the servers are too slow. However, without the right data, we will never be able to say how many patients should receive innovative therapies.
It is also extremely important to use data in clinical decision-making and individual financing. Real-world data are very important for balancing and controlling finances, but also predict the directions of development (on the basis of observed changes in morbidity). The aim should be to develop a single standard for the collection of medical data and their processing into scientific data.

The total reimbursement budget and the National Health Fund’s financial plan

The President of the Fund prepares a financial plan every year. The planned costs of financing healthcare services by a given regional branch of the Fund may not be lower than the costs of financing healthcare services for the given branch in the previous year, as set out in the financial plan. The financial plan includes a general provision in the amount of 1% of the planned revenues from health insurance premiums. When we compare the financial plans for the years 2012-2019, a steady upward trend is noticeable. Recently, this expenditure has been growing at an increasing pace. This is largely the result of our economic development. In reimbursement and in drug programmes, execution at the end of the year exceeds the financial plan drawn up at the beginning of the year. As a result, there is not enough money in the system to continue the therapy, and even less to enter into contracts on new drug programmes. In such situations, healthcare providers need to “extend a credit” to the National Health Fund. As a result, the financial standing of healthcare providers is deteriorating. The specificity of resource planning is therefore unfavourable. It would be worth considering whether such conservative planning is causing additional disruption to the system. It is thus suggested to increase spending on reimbursement and to introduce more rational distribution and allocation of funds.

The perspective of telemedicine development in Poland

In Poland, certain timid attempts have been made to finance telemedicine, but in practice they have not yet been introduced. The plan includes, among other things, telemedicine services financed under pilot programmes by the Kingdom of Norway. There are also attempts to implement and stimulate the development of telemedicine by companies (by the end of year 2, medical procedures should be submitted to the Ministry of Health as a proposal), as well as attempts to enter into public funds from the perspective of local governments. The government will introduce two remote possibilities for the use of telemedicine by mid-year. The greatest revolution we will be facing in this area (provided that the legislators opt for it) will be the possibility to open the teleconsultation market in the area of primary healthcare and secondary outpatient care.