Access and pricing of medicines for patients with rare diseases in the European Union: an industry perspective.

Introduction: The EU Orphan Regulation has successfully stimulated R&D of medicines for rare diseases, resulting in a substantial increase of orphan designations and authorized orphan medicinal products in the EU during last decade. Despite such advances, access to treatment across the 27 EU Member States is still highly variable.

Areas Covered: We provide an overview of the current situation of patient access to orphan drugs in the EU.

Economic evaluations in European reimbursement submission guidelines: current status and comparisons.

This study aimed to review European national health-economic (HE) guidelines and to identify recent developments in guideline recommendations by comparing the findings with those of a review published in 2001. Guidelines were identified by searching websites of the Internal Society for Pharmacoeconomics and Outcomes Research (ISPOR) and government health insurance agencies, and by a literature review. National guidelines showed broad consistency in ranking clinical data sources and choice of comparators for HE analysis, but varied in recommended costs to be included, methods related to cost calculation and discounting.

Cost per patient and potential budget implications of denosumab compared with zoledronic acid in adults with bone metastases from solid tumours who are at risk of skeletal-related events: an analysis for Austria, Sweden and Switzerland.

Objectives: To assess cost implications per patient, per year, and to predict the potential annual budget impact when patients with bone metastases secondary to solid tumours at risk of skeletal-related events (SREs) transition from zoledronic acid (ZA; 4 mg every 3-4 weeks) to denosumab (120 mg every 4 weeks) in Austria, Sweden and Switzerland.

Methods: Country specific costs for medication and administration, patient management and SREs (defined as pathologic fracture, radiation to bone, surgery to bone and spinal cord compression) were assessed over a 1-year time horizon. Drug administration and patient management costs were taken from available public sources.

Assessment of the reliability and repeatability of landmarks using 3-D cephalometric software.

The aim of this study was to analyze the reliability and repeatability of identification landmarks using 3-D cephalometric software. Ten orthognathic patients were selected for this study and underwent the following protocol: 1. radiographic evaluation (CBCT technique); 2.

Sensitivity of magnetic resonance imaging and computed axiography in the diagnosis of temporomandibular joint disorders in a selected patient population.

Purpose: The aim of this study was to compare sensitivity differences and interpretative agreement for magnetic resonance imaging (MRI) and computed axiography (CA) tracings in a patient population group with temporomandibular disorder (TMD).

Materials And Methods: A convenience sample of 173 patients (53 men, 120 women; mean age: 33.2 ± 2.

How does comorbidity affect cost of health care in patients with irritable bowel syndrome? A cohort study in general practice.

Background: Irritable bowel syndrome (IBS) is associated with other disorders (comorbidity), reduced quality of life and increased use of health resources. We aimed to explore the impact of comorbidity on cost of health care in patients with IBS in general practice.

Methods: In this cohort study 208 consecutive patients with IBS (Rome II) were recruited.

Economic evaluation of tegaserod vs. placebo in the treatment of patients with irritable bowel syndrome: an analysis of the TENOR study.

Objectives: Tegaserod is effective, safe, and well-tolerated in the treatment of patients with irritable bowel syndrome (IBS) with constipation. The aim of this study was to assess, from a payer perspective, the cost-effectiveness of tegaserod in the treatment of IBS patients, based on the TEgaserod in NORdic region (TENOR) trial data.

Methods: Female and male patients (Rome II criteria) were randomized to receive tegaserod 6 mg b.

Performance of the EQ-5D in patients with irritable bowel syndrome.

Objective: The EQ-5D is a standardized, nondisease-specific instrument for evaluating patients' preference-based valuations of health-related quality of life (HRQoL). This study's purpose was to determine the psychometric properties of EQ-5D in patients with irritable bowel syndrome (IBS).

Methods: Data from four European IBS studies were assessed: UK (n = 161 and n = 297), Spain (n = 503), and Germany (n = 100).