Food allergy (FA) poses a substantial burden on patients and their families, with reported physical and emotional impacts that can significantly influence daily life. Multiple disease-specific patient-reported outcome measures (PROMs) have been developed to measure the impact of FA on health-related quality of life (HRQoL). This review aimed to identify suitable PROMs to enable the assessment of interventions on the HRQoL impacts of patients with FAs. A literature search was performed in Medline and Embase from Jan 2010-April 2020. Key words like “food allergy” and “HRQoL” identified PROMs developed or used in patients with FA. Psychometric properties including content validity, reliability, validity, responsiveness and interpretability, were evaluated in accordance with the FDA PRO Guidance (2009). A total of 1028 abstracts were identified and 241 were selected for full-text review resulting in a list of 46 PROMs. They were classified as generic (n=22), allergy specific (n=2), FA-specific (n=19), and treatment satisfaction-specific (n=3). From the FA-specific PROMs, only 7 were conceptually relevant for further evaluation— Food Allergy Quality of Life Questionnaire (FAQLQ), Food Allergy Independent Measure (FAIM), Food-Pet-Allergy in Children Questionnaire (PFAC), Food Allergy Quality of Life Assessment Tool for Adolescents (FAQL-teen), You and Your Food Allergy, Food Intolerance Quality of Life Questionnaire (FIQLQ) and Pediatric Food Allergy Quality of Life Questionnaire (PFA-QL). You and Your Food Allergy, FAQLQ, PFAC, FAQL-teen involved patient’s qualitative inputs to establish content validity. All measures showed internal consistency (reliability) to be above 0.70 threshold; in terms of validity, PROMs moderately (0.30-0.70) correlated with existing measures. Only the FAQLQs showed both responsiveness to change and interpretability, key attributes for use in clinical trials. The FAQLQs validated for measuring HRQoL in FA have relevant conceptual coverage and adequate psychometric strength. Therefore, FAQLQs are suitable for further exploration of the impact of interventions within FA clinical trials.

The objective of this study was to establish current practice in TDM in K-Tx in a real-world clinical setting, and to develop a best-practice concept to improve patient care. TDM processes for transplanted patients were collected at 13 centers in 5 countries between January 2015 and June 2015. More than 35 Interviews with physicians, nurses and lab assistants were combined with observation of relevant steps during standard examination sessions. Duration of each main step to adjust the patient medication dose was recorded. Also dependencies from outside variables were recorded. Several process patterns were identified. Main observed differences among centers were process steps and inefficiencies in exam setup and time required getting test results. The time gap between taking blood samples and availability of results leads to long patient stay and/or multiple visits or contacts with the patient (blood test, examination, result communication). Average time for patients in a center was 2:12 hours with a large range from 0:49 hours to 6:22 hours. Process time for blood drawing, examinations and results communication (excluding lab processing) was on average 30 min ranging from 12 min to 52 min. Complexity and time to get the results could be improved if a near-patient-test (NPT) scenario would be available while others are subject to normal process improvements. Results suggest that improvements could be made in all types of scenarios. Especially fast availability of test results in a NPT scenario could reduce complexity and facilitate the development of an improved process setup.

In the US the monitoring of patients with Chronic Myeloid Leukaemia (CML) presents extensive intra- and inter-lab variability, thus a standardised, automated test should allow for improvement in patient management and health outcomes. The aim of the study was to estimate the budget impact and improved testing accuracy associated with a the use of a standardised, automated BCR-ABL monitoring test (SBAT) when compared to laboratory developed tests (LDTs) for newly diagnosed CML patients over a 5-year period in the US. Epidemiology data regarding the incidence of Philadelphia positive (Ph+) CML patients who would be treated with a tyrosine kinase inhibitor (TKI) were combined with workflow cost and accuracy (sensitivity and specificity) data associated with the sequential testing and monitoring of newly diagnosed CML patients. A survey of US laboratories was conducted to determine the labour and materials costs associated with the SBAT versus LDTs. A testing algorithm based on NCCN guidelines was used to capture a number of different tests including testing for major molecular response (SBAT versus LDTs), complete cytogenetic response (routine and FISH- fluorescence in situ hybridisation), and mutation analysis. Results indicate that the SBAT is both less resource- and labour- intensive, and can be carried out at a cost that is lower than when an LDT is used. In addition, overall test accuracy increases when the SBAT is used instead of an LDT. For example, for every 100 patients who follow BCR-ABL monitoring according to NCCN guidelines, savings of approximately $386,180 and approximately 327 more accurate test results could be achieved over 5 years. The benefits from a SBAT when compared to LDTs are not only from the reduction of intra- and inter-lab variability (increased accuracy) but also in economic terms due to lower overall costs. Therefore, a SBAT represents a cost-saving alternative versus LDTs.