Maciej Niewada

Medical University of Warsaw · Department of Experimental and Clinical Pharmacology

Background: To analyze the time from drug registration to reimbursement recommendations, we examined medicinal products, including new clinical indications, registered by the EMA between 2014 and 2019 across various therapeutic areas. Materials and methods: The Polish Agency for Health Technology Assessment and Tariffication (AOTMiT) was compare...

Objectives: Human papillomavirus (HPV) is a prevalent sexually transmitted infection with significant implications for public health. In Poland, a nationwide vaccination program offers a choice between the 9-valent (9v) and 2-valent (2v) HPV vaccines. We aimed to assess the cost-effectiveness of the 9v vs 2v vaccine from the public payer perspecti...

The efficacy of pembrolizumab in the treatment-naïve non-small-cell lung cancer (NSCLC) patients was proved in the KEYNOTE-024 randomized trial. The aim of this systematic literature review was to identify and summarize the real world evidence (RWE) of overall survival (OS) in previously untreated patients with NSCLC receiving pembrolizumab monothe...

The purpose of this article is to investigate the common facilitators and barriers associated with the implementation of hospital-based health technology assessment (HB-HTA) across diverse hospital settings in seven countries. Through a two-round Delphi study, insights were gathered from a panel of 15 HTA specialists from France, Hungary, Italy, Ka...

This study examines the vaccine market access pathway in Poland to evaluate its efficiency and propose recommendations for its improvement. The research spans a comprehensive analysis of the vaccine assessment process, ranging from pre-registration to sustainability, encompassing critical components such as national immunization technical advisory...

Our objective was to compare AOTMiT (Polish: Agencja Oceny Technologii Medycznych i Taryfikacji) recommendations to other HTA (Health Technology Assessment) agencies for newly registered drugs and new registration indications issued by the European Medicines Agency between 2014 and 2019. The study aims to assess the consistency and justifications o...

Introduction: The Registry of Stroke Care Quality (RES-Q) is used in Poland for quality monitoring by numerous hospitals participating in the Angels Initiative. Our aim was to assess the degree of improvement in highly stroke-oriented centres that report cases to the RES-Q each year. Material and methods: This retrospective analysis included Pol...

Introduction: The uptake of complex technologies and platforms has resulted in several challenges in the pricing and reimbursement of innovative pharmaceuticals. To address these challenges, plenty of concepts have already been described in the scientific literature about innovative value judgment or payment models, which are either (1) remaining...

Introduction: Our objective was to analyze and compare systematically and structurally reimbursement systems in Poland and other countries. Methods: The systems were selected based on recommendations issued by the Polish Agency for Health Technology Assessment and Tariffication (AHTAPol), which explicitly referred to other countries and agencies)....

Introduction Meaningful patient involvement in health technology assessment (HTA) is essential in ensuring that the interests of the affected patient population, their families, and the general public are accurately reflected in coverage and reimbursement decisions. Central and Eastern European (CEE) countries are generally at less advanced stages...

Introduction: The combination of immunotherapy and targeted therapy is currently marking a new era in the treatment of renal cancer. The latest clinical guidelines recommend the use of drug combinations for the first-line treatment of advanced renal cancer. The aim of this review is to compare the efficacy of combined cabozantinib + nivolumab thera...

Background and purpose Wilson's disease (WD) is a rare autosomal recessive disorder causing excessive copper deposition and a spectrum of manifestations, particularly neurological and hepatic symptoms. We analysed the clinical characteristics of patients with WD admitted to the country's only reference centre, which provided long‐term care to most...

Background Parenteral nutrition-associated liver disease (PNALD) is diagnosed after at least 2 weeks of total parenteral nutrition (PN). However, its symptoms may occur early during PN. The aim of this study is to determine the early biochemical predictors of PNALD. Methods This cross-sectional retrospective study included 160 patients on total pa...

Background and aims: WD is a rare genetic disorder causing copper accumulation and subsequent liver and brain damage. Since the end of the 1950s, life-long anti-copper therapies (mainly d-penicillamine - DP, trientine, zinc salts) have been available. We present seven decades of experience of a single reference centre covering most adult Wilson dis...

I won the 1st prize at the Internal Medicine Session held during the 30th International Medical Students Conference Cracow in the beginning of June 2022. My work was titled: “Long Term Follow-Up of Wilson Disease Patients in Poland - Report from the National Reference Center”, mentored by Dr hab. n. med. Maciej Niewada and Prof. dr hab. n. med. An...

Background and aim: Since the first desription of hepatolenticular degeneration by SK Wilson the expertise about Wilson disease (WD) clinic, pathogenesis, diagnosis and therapy has increased our daily practice. The aim of our study is to present seven decades of experience of a single reference centre covering most adult WD patients in Poland Metho...

Background : Epidemiologic data on pediatric-onset multiple sclerosis (POMS) in Central and Eastern Europe are limited. The aim of this study was to determine the incidence, prevalence and the clinical features of POMS in Poland. Methods : Registry-based retrospective study was conducted among Polish children population (age ≤ 18 years), between 1...

Raport powstał z inicjatywy Polskiego Towarzystwa Neurologicznego we współpracy z Instytutem Zarządzania w Ochronie Zdrowia Uczelni Łazarskiego. Opracowanie to jest efektem wielomiesięcznej pracy szerokiego grona ekspertów klinicznych, reprezentujących ośrodki z całego kraju, przedstawicieli organizacji pacjenckich, a także ekspertów systemowych. W...

Objective: We present seven decades of experience of a single refer-ence centre covering most adult Wilson disease (WD) patients in Poland.Background: WD is a rare genetic disorder causing copper accumula-tion and subsequent liver and brain damage. Consequently, two symptom-atic forms, neurological and hepatic, are recognized; additionally,presympt...

Value in drugs do not always lies simply in them curing diseases: they may also be used to prolong life, improve its quality, and relieve patients' families. How much these elements matter should weigh on reimbursement decisions. Rare diseases often lack strong clinical evidence; hence, overlooking other elements that society values may lead to sub...

Background: TELEREH-HF study showed a statistically significant improvement in the tertiary outcomes i.e. New York Heart Association (NYHA) class after a 9 week follow-up, consistent with telerehabilitation-related benefit in quality of life (QoL) measured by the 36-Item Short Form questionnaire (SF-36). Aim: We analyzed the cost-effectiveness o...

Objectives: Methodological challenges in the evaluation of medical devices (MDs) may be different for early and late technology adopter countries, as well as the potential health technology assessment (HTA) solutions to tackle them. This study aims to provide guidance to Central and Eastern European (CEE) countries on how to address key challenges...

Aim of study: The Fluoxetine Or Control Under Supervision (FOCUS)-Poland trial tested in a Polish cohort the hypothesis that fluoxetine improves recovery after stroke. Clinical rationale for study: Some studies have suggested that fluoxetine may improve functional outcomes after stroke, but these results needed confirmation. Between 2012 and 201...

Background Cardiovascular diseases are associated with growing public and private expenditure on healthcare regardless geographic region. Therefore, it is necessary to accurately estimate the overall societal costs—both direct and indirect expenses from the perspective of patients, caregivers and employers. Research Design The aim of this paper is...

Selected and basic indicators of acute ischaemic stroke care in Poland are reported cross-regionally based on the analysis of claims data of the National Health Fund (NFZ) in 2017, the most reliable source of healthcare funding in the country, being a single public payer. For research purposes, a selection algorithm based on the diagnosis coded as...

Objectives To perform cost-consequence and cost-effectiveness analyses of 2 methods of donor human milk (DHM) preservation—Holder pasteurization (HoP) and high-pressure processing (HPP)—in human milk banks in Poland. Methods We used the results of the LACTOTECHnology as an answer to special nutritional requirements of preterm infants (LACTOTECH) p...

Objective The analysis aims to assess the cost-effectiveness of cefuroxime (Aprokam®) in the prophylaxis of postoperative endophthalmitis (POE) after cataract surgery compared with the absence of antibiotic prophylaxis from the National Health Fund perspective in Poland. Methods We performed a cost-effectiveness and cost-utility analysis using the...

Background In Poland drug programmes developed by the Minister of Health and financed by the National Health Fund are special reimbursement frameworks of innovative, expensive, and mostly hospital based medical products used for a small number of patients. Research Design The research presented in this paper is based on data analysis published by...

Objectives We performed a systematic review of health state utility values (HSUVs) obtained using the EQ-5D questionnaire for patients with hematologic malignancies. Methods The following databases were searched up to September 2018: MEDLINE, EMBASE, The Cochrane Library, and the EQ-5D publications database on the EuroQol website. Additional refer...

Background: Dysphagia is a well-known stroke complication characterised by difficulty in swallowing. It may affect the majority of stroke patients and increases mortality and morbidity, due to aspiration pneumonia and malnutrition. Food thickening may help patients to feed themselves, and its effectiveness was demonstrated. However, the cost-effec...

Background: Gastrointestinal symptoms are common in patients with Wilson disease (WD) and may be related to the disease itself or to adverse drug reactions (ADRs). Aim: To investigate gastroscopy findings in patients with WD and to analyze the risk of gastropathy in the context of different manifestations and treatments of WD as well as Helicobacte...

Background: Wilson disease (WD) is genetically induced failure of copper metabolism which can be successfully treated with pharmacological agents. The prognosis for survival in most WD patients is favorable if diagnosis and anti-copper treatment are provided early. Many observations imply that persistence with drug treatment is generally low in pa...

Objective Cost-utility analyses are becoming increasingly important in Central and Eastern Europe. We aimed to develop a Polish utility tariff for EQ-5D-5L health states. Methods Face-to-face, computer-assisted interviews were collected in a representative sample. Each respondent followed a standardised protocol to collect ten composite time trade...

Background To date, a multi-country review evaluating the cost-of-illness (COI) studies from the Central and Eastern European (CEE) region has not yet been published. Our main objective was to provide a general description about published COI studies from CEE. Methods A systematic search was performed between 1 January 2006 and 1 June 2017 in Medl...

Background: According to the current data around 1% of the Poland population have epilepsy, which consists about 400,000 people. This group of patients require life-long therapy including both, drug therapy and hospitalization. The character of the epilepsy has a significant impact on expenses borne by individual patients, the prevalence of the dis...

Background and aims: Familial hypercholesterolaemia (FH) elevates the cholesterol level and increases the risk of coronary events and death. Early detection and treatment reduce this risk. We aimed to determine the cost-effectiveness of FH screening in Poland in children, first job, and after an acute coronary syndrome (ACS) event, each followed by...

This paper aims to provide an overview of the rationalization strategies for the introduction and use of pharmaceuticals, focusing on the role of managed entry agreements (MEA) in Central and Eastern European (CEE) countries, namely Bulgaria, the Czech Republic, Croatia, Hungary, Poland and Romania. We developed a conceptual framework on MEAs that...

The impact of thrombolysis with recombinant tissue plasminogen activator (rtPA) on blood coagulation in acute ischemic stroke (AIS) patients is not completely understood. We studied the effect of thrombolysis on the thrombin generation (TG) profile as well as coagulant activity of activated factors IX (FIXa), XI (FXIa) and tissue factor (TF) in AIS...

Background: We aimed to investigate whether increased waist-to-hip ratio (WHR), waist circumference (WC), or improper body mass index (BMI) may differently predict short-term outcomes in females and males with first-ever acute ischemic stroke. Methods: This was a retrospective study of consecutive patients (1109 females and 939 males) admitted f...

Fibrin clot properties in acute ischaemic stroke (AIS) are unfavourably altered, including faster formation of denser and poorly lysable fibre networks. We investigated clot properties in AIS patients treated with recombinant tissue plasminogen activator (rtPA) and their impact on clinical outcome. In 74 consecutive AIS patients eligible for rtPA t...

Purpose: In most religions, the preservation of one's own, God-given, life is considered obligatory, while the time trade-off method (TTO) forces one to voluntarily forego life years. We sought to verify how this conflict impacts TTO-results among the religious. Methods: We used the data from the only EQ-5D valuation in Poland (2008, three-level...

Objective: Complications contribute largely to the economic gravity of diabetes mellitus (DM). How they arise and are treated differ substantially between countries. In this paper we assess the total annual, direct and indirect, cost of severe hypoglycaemia events (SHEs) in nine European countries: Bulgaria, Croatia, the Czech Republic, Greece, Hu...

Background: Arterial hypertension (AH) represents a public health problem in Poland, firstly due to the huge, still growing population of patients (10.45 million patients based on NATPOL 2011 and PolSenior Surveys) and secondly because of the substantial cost of reimbursement from the National Health Fund (NHF). The most commonly used drugs in the...

Objectives: We aimed to investigate whether prior use of antiplatelet agents (AP) may be associated with lower severity and improved short-term outcome of the first-ever acute ischaemic stroke. Methods: This was a retrospective, case-control study based on a prospective hospital stroke registry covering consecutive acute stroke patients admitted...

Introduction: The new, five-level version of the EQ-5D (EQ-5D-5L) questionnaire has better psychometric properties than the standard three-level version (EQ-5D-3L), including a reduced ceiling effect. Currently, there are few existing population norms for the EQ-5D-5L. The aims of this study were to provide population norms for the EQ-5D-5L in Pola...

Purpose of review: The purpose is to summarize recent evidence on lifestyle modifications and first or recurrent stroke risk. Recent findings: Weight reduction, low-risk diet, regular physical activity, smoking cessation, and low-to-moderate alcohol consumption may reduce stroke risk up to 50% or more, but level one evidence is still lacking for...

Data on potential variations in delivery of appropriate stroke care over time are scarce. We investigated temporal changes in the quality of acute hospital stroke care across five national audits in Europe over a period of six years. Methods. Data were derived from national stroke audits in Germany, Poland, Scotland, Sweden, and England/Wales/North...

Background Diabetes mellitus (DM) leads to multiple complications, including severe hypoglycaemia events (SHEs). SHEs can impact a patient’s quality of life and compliance and may directly result in additional costs to the health care system. The aim of this review was to evaluate the risk of severe hypoglycaemia in patients with type 1 (T1) and 2...

Objectives To evaluate the use of biological agents for the treatment of psoriasis and to explore country-specific differences within six Central and Eastern European (CEE) countries, namely Bulgaria, Croatia, the Czech Republic, Hungary, Poland and Romania.MethodsA literature overview on the epidemiology and disease burden of psoriasis in CEE was...

Diabetes mellitus (DM) is associated with severe hypoglycemia events (SHEs) that vary in severity and resource consumption. Here we perform a systematic review in Medline of studies evaluating SHE-related health resource use. Eligible studies investigated patients with DM and included ≥10 SHEs. We also assessed studies identified in another systema...

Despite estimates about general trends in stroke epidemiology worldwide, there are only a few reports of detailed longitudinal data and none of them reflects the economic transition that occurred in Central and Eastern Europe over the last two decades. The aim of this study was to investigate long term trends in risk factors and their pre-stroke co...

The role of antiplatelet agents in stroke prevention is well documented, especially for secondary prevention, but many patients with no previous stroke but with other risk factors (high cardiovascular risk, women with diabetes mellitus, chronic kidney disease, peripheral artery disease, or asymptomatic carotid stenosis) can also benefit. The findin...

Introduction: A Polish EQ-5D normative study published in 2010 was conducted in 2008 as a pilot study. The group of respondents was relatively small and had limited representativeness. Objectives: The aim of the study was to derive population norms for the 3-level EQ-5D (EQ-5D-3L) questionnaire in Poland using a large, representative sample. Re...