Description: Objective In 2008, the Thai government introduced the ‘high-cost medicines E2 access program’ as a part of the National List of Essential Medicines to increase patient access to medicines, improve clinical outcomes and make medicines more affordable. Our objective was to examine whether the ‘high-cost medicines E2 access program’ achieved its goals. Design Interrupted time-series design study. Setting 3 tertiary hospitals in different regions of Thailand, January 2006 to December 2012. Participants Patients with target acute and chronic disease diagnoses who newly met E2 program criteria for selected study medicines. Intervention High-cost medicines E2 access program. Main outcomes measures Level and trend changes over time in the proportions of eligible patients who received the indicated E2 medicines and who improved clinically, as well as in costs of treatment. Results A total of 2024 patients were included in utilisation analyses and 1375 patients with selected acute diseases contributed to analyses of clinical outcome. After 1 year of the E2 program implementation, the percentage of eligible patients receiving the indicated E2 program medicines increased significantly (relative change 12.7% (95% CI 4.4% to 21.0%), especially among those insured by the government's universal coverage scheme (relative change 19.9% (95% CI 9.5% to 30.5%)). The increase in the proportion of clinically improved patients with acute conditions was not significant (relative change 6.2% (95% CI –1.9% to 15.1%)). Quarterly healthcare costs per patient dropped significantly (relative change –13.5% (95% CI –26.9% to –1.7%)). Conclusions In the study hospitals, the E2 access program seems to have facilitated patient access to specialty medicines, may have contributed to improved health outcomes, and decreased treatment costs. Routine monitoring is needed to assess effects of expanding the programme, including effects on quality of care and financial sustainability.

Description: Introduction Recognising a tumour predisposition syndrome (TPS) in patients with childhood cancer is of significant clinical relevance, as it affects treatment, prognosis and facilitates genetic counselling. Previous studies revealed that only half of the known TPSs are recognised during standard paediatric cancer care. In current medical practice it is impossible to refer every patient with childhood cancer to a clinical geneticist, due to limited capacity for routine genetic consultation. Therefore, we have developed a screening instrument to identify patients with childhood cancer with a high probability of having a TPS. The aim of this study is to validate the clinical screening instrument for TPS in patients with childhood cancer. Methods and analysis This study is a prospective nationwide cohort study including all newly diagnosed patients with childhood cancer in the Netherlands. The screening instrument consists of a checklist, two- and three-dimensional photographic series of the patient. 2 independent clinical geneticists will assess the content of the screening instrument. If a TPS is suspected based on the instrument data and thus further evaluation is indicated, the patient will be invited for full genetic consultation. A negative control group consists of 20% of the patients in whom a TPS is not suspected based on the instrument; they will be randomly invited for full genetic consultation. Primary outcome measurement will be sensitivity of the instrument. Ethics and dissemination The Medical Ethical Committee of the Academic Medical Centre stated that the Medical Research Involving Human Subjects Act does not apply to this study and that official approval of this study by the Committee was not required. The results will be offered for publication in peer-reviewed journals and presented at International Conferences on Oncology and Clinical Genetics. The clinical data gathered in this study will be available for all participating centres. Trial registration number NTR5605.