The European Health Technology Assessment (EU-HTA) aims to harmonise HTA across Europe, enhancing efficiency and reducing duplication of work for national authorities. This paper explores key components of the EU-HTA framework, challenges, future perspectives, and implications for pharmaceutical companies, primarily concentrating on drug evaluation over medical devices

Scientific evidence needs might differ substantially between the different stakeholders, but they also vary between countries due to different philosophies in the assessment of healthcare interventions. This requires early Global Evidence Generation planning. This paper deals with the process of developing an Evidence Generation Plan (EGP).

Systematic literature review to identify real-world studies reporting NST or attrition rates in la/mUC from 2017–2022 (including data reported since 2015). Of 2439 publications screened, 29 reported NST rates, ranging from 40–74% in eight European-based studies, 14–60% in 12 US-based studies, and 9–63% in nine studies in other locations (meta-analysis estimate, 39%). Factors associated with NST or no second-line therapy included older age, female sex, poor performance status, poor renal function and distant metastases.

This paper explores the basic processes around an MCR as well as its advantages and disadvantages and provides a couple of recommendations on how an “ideal” MCR should be conducted. Trade-offs researchers should be aware of in designing an MCR will be explored.

We introduce a novel, growing database that links administrative claims and medical records from an MS patient management system, allowing for the complete capture of patient profiles. Using the AOK PLUS sickness fund and the Multiple Sclerosis Documentation System MSDS3D from the Center of Clinical Neuroscience (ZKN) in Germany, a linked MS-specific database was developed (MSDS-AOK PLUS).

This study aimed to evaluate differences in healthcare resource utilization and cost among patients with controlled and uncontrolled asthma. Claims data from a German sickness fund was linked to patient survey data. Outpatient physicians enrolled patients and assessed asthma control using the ACT questionnaire. All-cause and asthma-specific healthcare resource use (HCRU)/costs were compared descriptively and based on multivariable models using a continuous ACT score.

This German claims data study included continuously insured persons with at least two outpatient diagnoses and/or one inpatient diagnosis of T2DM if they started EMPA, DPP-4i, or GLP-1-RA in 2015–2018. Healthcare costs were assessed from therapy initiation until the end of data availability, death, or therapy discontinuation and compared among propensity score matched cohorts. Although the individual clinical patient profile and physician assessment are paramount in treatment decisions, substantial differences in the economic impact of different antidiabetic therapies should be considered.

Anonymized claims data of patients ≥18 years treated with drugs for cardiovascular (CV)-related diseases either as a single pill combination or multi-pill combination were evaluated. After propensity score matching, 59,336 out of 1,369,840 patients were analyzed. In all cohorts, patients receiving a single pill combination had a lower frequency of general practitioner and specialist visits. The patients also had a significantly lower ratio of all-cause hospitalization days and number of CV-related prescriptions as well as all-cause prescriptions (with one exception) compared with those receiving a multi-pill combination.

In an explorative study, we analyzed anonymized claims data sets of patients treated with CV drugs for hypertension and/or CV disorders who were insured by the German AOK PLUS statutory health fund covering 01/07/2012-30/06/2018. Patients aged ≥18 years who received either an SPC or MPC with identical drugs were followed for up to one year. A one-to-one propensity score matching (PSM) was applied within patient groups who started identical drug combinations, and results were reported as incidence rate ratios (IRRs) as well as hazard ratios (HRs). After PSM, data from 59,336 patients were analyzed. In 30 out of 56 IRR analyses, the superiority of SPC over MPC was shown. In 5 out of 7 comparisons, the HR for the composite outcome of all-cause death and all-cause hospitalizations was in favor of the SPC regimen.

The previously validated (indications: atrial fibrillation, human immunodeficiency virus, chronic inflammatory lung disease) Adherence Barriers Questionnaire (ABQ) was revised according to specifications of IVT, within the framework of an expert panel. A cross-sectional patient survey was conducted in combination with a retrospective medical chart review in order to validate the developed new instrument (ABQ-IVT).

This study aimed to investigate the prevalence, incidence, and real-world diagnostic and treatment patterns, healthcare resource utilization (HCRU), and associated costs of narcolepsy in Germany.

A multi-center observational study was conducted addressing adult patients with asthma who were prescribed regular maintenance medication between 2014 and 2016 to identify the potential barriers to medication adherence among adult patients with asthma in Germany. Data were derived from physicians’ documentation and claims data, which were linked to the primary observational data and patient survey data (Adherence Barriers Questionnaire - ABQ).

A discrete choice experiment was conducted among general practitioners, cardiologists, neurologists and ophthalmologists in Germany to examine physicians’ preferences regarding adherence-promoting programs (APPs). The design considered five attributes with two or three attribute levels each: validation status of the APP; possibility for physicians to receive a certificate; type of intervention; time commitment per patient and quarter of the year to carry out the APP; reimbursement for APP participation, per included patient and quarter of the year.

The patient’s perspective is becoming increasingly important in clinical and policy decisions. This study examined atrial fibrillation (AF) patient preferences for different characteristics of nonvitamin K antagonist oral anticoagulants (NOACs).

As there is a general lack of data around T2DM patients newly initiating an insulin therapy, it was the main objective of this study to collect these data. The main strength of this analysis is the use of a large claims dataset unaffected by any selection bias and an additional collection of MCR data for reasons of a validity check of claims data analysis results, as the claims data specifically addressed two German states.

The aim of this investigation was to develop and validate a respective questionnaire (Adherence Barriers Questionnaire for HIV: ABQ-HIV), based on an earlier version of the ABQ.

The study showed that the ABQ-HIV is a practical, reliable, and valid instrument for identifying patient-specific barriers to

adherence in the HIV treatment that is also useful in identifying HIV patient subgroups.

This study describes preferences of German relapsed refractory multiple myeloma (RRMM) patients with novel proteasome inhibitor-based combination treatments.

Real-world evidence (RWE) can inform patient management decisions, but RWE studies are associated with limitations. Linkage of different RWE data types could address such limitations by enriching data and improving scientific quality. Using the example of chronic obstructive pulmonary disease (COPD) in Germany, this study assessed the value of data linkage between primary and secondary data sources for RWE.

The aim of this study was to assess patients’ views and xpectations with regard to neovascular age-related macular degeneration (nAMD) and intravitreal anti-VEGF therapy (IVT).

This multicenter, non-interventional, prospective cohort study showed that only a minority of patients are aware of the chronic nature of nAMD. To motivate patients to accept a life-long IVT treatment, physicians and caregivers must know that there exist different patient types with significant differences in communication needs.

Since the introduction of non-vitamin K antagonist (VKA) oral anticoagulants (NOACs), an additional treatment option, apart from VKAs, has become available for stroke prevention in patients with atrial fibrillation (AF). For various reasons, it is important to consider patients’ preferences regarding the type of medication, particularly given the established relationship between preferences towards treatment, associated burden of therapy, and treatment adherence. This review aimed to systematically analyze the scientific literature assessing the preferences of AF patients with regard to long-term oral anticoagulant (OAC) treatment.