Issues, Challenges and Opportunities for Economic Evaluations of Orphan Drugs in Rare Diseases: An Umbrella Review.

Background And Objectives: There are significant challenges when obtaining clinical and economic evidence for health technology assessments of rare diseases. Many of them have been highlighted in previous systematic reviews but they have not been summarised in a comprehensive manner. For all stakeholders working with rare diseases, it is important to be aware and understand these issues.

A Cost-Utility Analysis of SQ Tree SLIT-Tablet versus Placebo in the Treatment of Birch Pollen Allergic Rhinitis from a Swedish Societal Perspective.

Background And Aims: Allergic rhinitis (AR) is an immunoglobulin E antibody-mediated inflammatory condition that arises in response to inhaled allergens such as pollen. Pollens from trees in the birch homologous group are the most common allergenic tree pollens in Northern and Central Europe and North America. SQ® Tree SLIT-Tablet (ITULAZAX®) is a sublingual immunotherapy tablet indicated for moderate-to-severe AR and/or conjunctivitis induced by pollen from the birch homologous group.

ITULAZAX versus Alutard SQ in the treatment of allergic rhinitis induced by pollen from the birch homologous group: A cost-minimization modeling analysis from the Danish societal perspective.

Background And Aims: Allergic rhinitis (AR) is an inflammatory disorder triggered by an allergic immune response to inhaled allergens. Birch pollen is the major allergenic tree pollen in parts of Europe. ITULAZAX® is a sublingual immunotherapy tablet for the treatment of adults with moderate-to-severe AR and/or conjunctivitis induced by pollen from the birch homologous group.

The long-term cost-effectiveness of oral semaglutide in the Netherlands based on the PIONEER 2, 3 and 4 randomized controlled trials.

Aims: To assess the long-term cost-effectiveness of novel glucagon-like peptide-1 (GLP-1) analog oral semaglutide versus sodium-glucose cotransporter-2 inhibitor empagliflozin, dipeptidyl peptidase-4 inhibitor sitagliptin and injectable GLP-1 analog liraglutide in the Netherlands, based on the results of the PIONEER clinical trials.

Methods: Outcomes were projected over patient lifetimes using the IQVIA CORE Diabetes Model. Clinical data were derived from PIONEER 2, 3 and 4.

Cost-Effectiveness Of The SQ Grass SLIT-Tablet In Children With Allergic Rhinitis: A German Payer Perspective.

Background: The Grazax Asthma Prevention (GAP) trial has recently demonstrated significant reductions in the odds of asthma symptoms or medication use in patients treated with SQ grass SLIT-tablet relative to placebo, both in combination with allergy and asthma pharmacotherapy. The objective of the present analysis was to evaluate the cost-effectiveness of SQ grass SLIT-tablet relative to placebo in children with AR from the perspective of a German healthcare payer.

Methods: A cost-utility model was developed in Microsoft Excel (Microsoft Corporation, Redmond, WA, USA) to evaluate the cost-utility of SQ grass SLIT-tablet in combination with pharmacotherapy versus pharmacotherapy alone in patients with AR.

The cost-effectiveness of oral contraceptives compared to 'no hormonal treatment' for endometriosis-related pain: An economic evaluation.

Objective: To develop a preliminary cost-effectiveness model that compares oral contraceptives and 'no hormonal treatment' for the treatment of endometriosis-related pain.

Methods: A de novo preliminary state transition (Markov) model was developed. The model was informed by systematic literature review and expert opinion.

Utility elicitation in adults and children for allergic rhinoconjunctivitis and associated health states.

Purpose: Allergic rhinitis and asthma symptoms are detrimental to health-related quality of life (HRQoL). Health technology appraisal agencies often require cost-utility analysis when assessing new interventions. Appropriate utility estimates, which quantify the value of different conditions in cost-utility analyses, are scarce for allergic rhinitis and asthma health states.