The public funds allotted to drug reimbursement have long been the subject of a heated public debate. How to assess if a decision to spend a certain sum of public money for a given medication or treatment was right? The problems of health care funding mechanisms are discussed by Dr Tomasz Bochenek from the Institute of Public Health of the JU Medical College.

What is the price of one pack of the most expensive drug currently reimbursed by the National Health Fund in Poland? The easiest way to answer this question is to check the  reimbursed drugs list published by the Ministry of Health [1]. A pack of the medication named Spinraza, containing the active ingredient nusinersenum (nusinersen) has a wholesale price of 341,334 zlotys gross (about 80 thousand euros). To be more specific, it should be added that the pack contains a single five-millilitre vial of an injectable solution, which includes 12 milligrams of the active ingredient. The drug is used to treat spinal muscular atrophy, which is considered a rare disease. The treatment requires prolonged use of the medication [2], which can be obtained for free, as its cost is fully covered by the National Health Fund, which is financed by compulsory health insurance contributions.

Is 341,334 zlotys a high price for a five-millilitre vial of a drug, which has to be administered once every 4 months? Intuition suggests that the cost is astronomical, since it equals the price of a brand new luxurious car or a two-roomed flat in a city. What makes a difference is that this is a medicine that can help people suffering from a very serious illness.

No illness, no problem?

To assess whether the cost of a certain treatment is worth incurring from the perspective of the entire healthcare system, it’s necessary to analyse the problem from many different perspectives. What kind of illness is to be treated? Are any other treatment options available? What is the expected outcome? What is the relation between the costs and outcomes? Are the expenses acceptable in the context of a given (in this case – Polish) healthcare system? What additional funds are required to reimburse the drug for the patients who will need it in the next year and the years to follow? What changes in the entire healthcare system are expected to occur when the new medication will be included in the reimbursement system? What are the ethical grounds for the decision to reimburse the treatment or the refusal to do it?

The cost of healthcare usually becomes apparent only when we fall ill or when it happens to someone close to us

The answers to the above questions can be provided by the health technology assessment (HTA), which allows decisionmakers to evaluate if the funds allocated to the healthcare service are wisely spent. Those in good health rarely bother themselves with such matters. The cost of healthcare usually becomes apparent only when we fall ill or when it happens to someone close to us. In such circumstances, we often pay large sums of money for medications, medical tests, or surgeries, but often remain unaware of the costs of medical services covered by the public health care, which can be (sometimes extremely) high.

Public funding? Only after proper research

According to all forecasts, healthcare will become more and more expensive. Meeting this challenge requires not only greater budgets, but also a more rational spending of the available money, especially bearing in mind that it all comes from taxpayers. The health technology assessment is aimed at improving the allocation of health care resources. Without the HTA, this process would be fully dependent on individuals’ decisions, not necessarily based on any research.

The Polish law requires a thorough assessment of each medication before making the reimbursement decision. To achieve this goal, the Agency for Health Technology Assessment and Tariff System (AHTATS) was established 15 years ago [3].

On the crossroads of disciplines

According to the AHTATS, health technologies are defined as “medications, equipment, diagnostic and therapeutic procedures, as well as administrative support systems for providing health services”, whereas health technology assessment is understood as “an interdisciplinary field of study, aimed at making scientifically-based decisions in the field of public health policy and clinical practice”. The HTA adopts an interdisciplinary approach, based on expertise from many different fields, including medicine, epidemiology, biostatistics, as well as economy, law, and ethics. Combining medical knowledge with social sciences, it plays an increasingly important role in modern health service systems. The health technology assessment process consists of four subsequent stages: decision problem analysis, clinical analysis, economic analysis, and healthcare system impact analysis [4] [5].

The HTA analysis of nusinersen  reimbursement in spinal muscular atrophy treatment has resulted in the positive recommendation from the AHTATS and then from the Ministry of Health, and, consequently, the decision to reimburse the drug [6]. The text of the recommendation is available to the public, along with the relevant source documents. The material has been partially censored, due to business confidentiality. In 2014 the confidentiality restrictions were partially relaxed, which improved the transparency of the HTA in Poland [7].

The Institute of Public Health of the Jagiellonian University Medical College offers courses that prepare students for a career in the field of health technology assessment. The international students can study HTA-related subjects within the framework of the EuroPubHealth+ programme.

The original text by Dr Tomasz Bochenek is available on the JU Institute of Public Heath blog.

Sources:

1. Obwieszczenie Ministra Zdrowia z dnia 20 grudnia 2019 r. w sprawie wykazu refundowanych leków, środków spożywczych specjalnego przeznaczenia żywieniowego oraz wyrobów medycznych na 1 stycznia 2020 r. 2019.
2. Charakterystyka produktu leczniczego – Spinraza (nusinersen). Europejska Agencja Leków.
3. Nizankowski R, Wilk N. From idealistic rookies to a regional leader: the history of health technology assessment in Poland. Int J Technol Assess Health Care. 2009 Jul;25 Suppl 1:156–62.
4. Agencja Oceny Technologii Medycznych i Taryfikacji. Wytyczne oceny technologii medycznych. Wersja 3.0. III. Warszawa: Wydawnictwo Lekarskie PZWL; 2016. 79 p.
5. Bochenek T. Technologie medyczne i ich ocena. Czyli co to właściwie jest HTA? Blog Zdrowia Publicznego. Instytut Zdrowia Publicznego WNZ UJ CM w Krakowie. 2018.
6. Rekomendacja nr 21/2018 z dnia 16 marca 2018 r. Prezesa Agencji Oceny Technologii Medycznych i Taryfikacji w sprawie objęcia refundacją produktu lecznicego Spinraza (nusinersenum), roztwór do wstrzykiwań, 12 mg, 1 fiol. 5 ml, w ramach programu lekowego… . 2018.
7. Bochenek T., Kocot E., Rodzinka M., Godman B., Maciejewska K., Kamal S., Pilc A. The transparency of published health technology assessment-based recommendations on pharmaceutical reimbursement in Poland. Expert Rev Pharmacoecon Outcomes Res. 2017;17(4):1–16.