Validation of the FROM-16 in family members of patients receiving advanced therapy medicinal product (ATMP).

Purpose: Outcome-based pricing models which consider domains of value not previously considered in healthcare, such as societal outcomes, are of increasing interest for healthcare systems. Societal outcomes can include family-reported outcome measures (FROMs), which measure the impact of disease upon the patient's family members. The FROM-16 is a generic and easy-to-use family quality of life tool, but it has never been used in the context of patients undergoing advanced therapy medicinal product (ATMP) treatment.

Association of child weight and adverse outcomes following antibiotic prescriptions in children: a national data study in Wales, UK.

Objective: To examine if the weight of a child determines adverse events following oral antibiotics prescription.

Design: Population respective cohort using linked general practice (GP), hospital data and linkage with the Welsh Demographic Service for demographic information. Data linkage was performed using Wales health data, extracted from the SAIL (Secure Anonymised Information Linkage) databank.

Value-Based Healthcare in Practice: IDEATE, a Collaboration to Design and Test an Outcomes-Based Agreement for a Medicine in Wales.

Objective: To develop a sustainable, scalable methodology for the design of outcome-based agreements (OBAs) that works on the ground and dynamically overcomes historical challenges.

Methods: Project IDEATE co-created solutions to known (and emergent) challenges via iterative workshops and real-world data analysis to develop and refine a hypothetical model for an OBA in a trusted research environment. A cross-disciplinary collaboration between National Health Service (NHS) Wales, industry and academia was developed.

Outcomes and characteristics of nonmelanoma skin cancers in patients with myeloproliferative neoplasms on ruxolitinib.

Nonmelanoma skin cancers (NMSCs) in ruxolitinib-treated patients with myeloproliferative neoplasms behave aggressively, with adverse features and high recurrence. In our cohort, mortality from metastatic NMSC exceeded that from myelofibrosis. Vigilant skin assessment, counseling on NMSC risks, and prospective ruxolitinib-NMSC studies are crucial.

Can we collect health-related quality of life information from anticoagulated atrial fibrillation participants who have recently experienced a bleed? An observational feasibility study in primary and secondary care in Wales and through a UK online forum.

Objective: To evaluate the feasibility of recruiting participants diagnosed with atrial fibrillation (AF) taking oral anticoagulation therapies (OATs) and recently experiencing a bleed to collect health-related quality of life (HRQoL) information.

Design: Observational feasibility study. The study aimed to determine the feasibility of recruiting participants with minor and major bleeds, the most appropriate route for recruitment and the appropriateness of the patient-reported outcome measures (PROMs) selected for collecting HRQoL information in AF patients, and the preferred format of the surveys.