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European countries. Methods: This was a cross-sectional study carried out in the United Kingdom, France and Germany. Data was collected from July 2015 to November 2015. Healthcare costs (hospitalizations, emergencies, medical tests, drugs consumption, visits to GPs and specialists, health material and healthcare transport) and non-healthcare costs (social services and informal care) were identified and valued (reference year: 2014). EQ-5D instrument, Barthel index and Zarit score were also used to reflect the burden and the social impact of the disease beyond the healthcare cost. Results: 86 children with SMA were included, 26.7% of them were Type I and 73.3% were Type II or III. The annual average costs associated with SMA reached 54,295V in UK, 32,042V in France and 52,985V in Germany. Direct non-healthcare costs ranged between 65%-80% of the total cost associated with SMA disease in the three countries analysed. More precisely, the cost associated with informal care was 40,526V (74% of the total cost) for UK, 26,619V (80% of the total cost) for France and 39,926V (77% of the total cost) for Germany. Regarding HRQOL, it was observed that in France and United Kingdom children have a very poor quality of life (0.11 and 0.16, respectively, using time trade-off (TTO) utility score). On contrary, German children had a significantly better quality of life with a TTO of 0.53. Conclusions: SMA represents a considerably high socioeconomic impact both in terms of healthcare and social costs and in reduced HRQOL of children affected. Our analysis may help to design more efficient and equitable policies, with special emphasis on the need to increase the resources and the support provided to the families.

clinical trials. Increased AXL expression is associated with innate immune suppression and the appearance of resistance to targeted therapies in models of NSCLC and pt samples. AXL inhibition via bemcentinib prevents the appearance of such resistance in vivo and has shown immunomodulatory effect in AML patients. Method: This study was designed to confirm the safety and tolerability of bemcentinib as a monotherapy and when administered in combination with erlotinib (arm A). Pts with activating EGFR mutation driven NSCLC who had progressed on an approved EGFR inhibitor (arm B) or were receiving erlotinib in the first line setting (arm C) were treated with bemcentinib at RP2D in combination with full dose erlotinib to evaluate the potential of bemcentinib to reverse or prevent resistance to EGFR targeted therapy, respectively. Plasma protein biomarker levels were measured using the DiscoveryMap v3.3 panel (Myriad RBM) at C1D1 and C2D1. Result: 2 out of 8 pts (25%) with stage IV disease who received bemcentinib monotherapy achieved SD for close to 1 year including evidence of tumour shrinkage of 19% in 1 pt. 1 pt who had progressed on previous erlotinib monotherapy (12.5%) achieved a PR receiving bemcentinib in combination with erlotinib and remains on treatment well beyond 2 years later (arm A). A further 3 pts had SD at 6 wks. 11 patients (4 female, median age 58; 38-67) were enrolled in arm B and had received a median of 2 (1 -4) previous lines of cytotoxic chemotherapy and a median of 2 previous EGFR inhibitors. 2 of these 11 pts (18%) including 1 pt who was refractory to erlotinib therapy at the onset of combination therapy remain on treatment more than 6 months into therapy at the time of writing with best responses of PR and SD, respectively. 1 further pt had SD at 6 weeks. The most common treatment-related AEs have been gastrointestinal and rash.There was no evidence of any impact of bemcentinib on erlotinib pharmacokinetics. Protein biomarkers predictive of pt benefit following bemcentinib treatment were identified. Conclusion: Bemcentinib can be safely administered in combination with erlotinib to pts with NSCLC and achieves additional benefit in a proportion of patients who do not have T790M and have progressed on EGFR inhibition or are maintained on erlotinib alone. Clinical trial information: NCT02424617.Background: Tumor heterogeneity causes different EGFR mutation abundances, and is believed to be responsible for varied progressionfree survival (PFS) in lung adenocarcinoma (ADC) patients receiving EGFR-TKI treatment. EGFR amplification and its common presence in EGFR mutant allele might be determined by the EGFR copy number variation. Examination of EGFR amplification status in EGFR mutant patients could predict the efficacy of EGFR-TKI treatment Method: 72 lung ADC patients, who harbored EGFR activating mutations and received erlotinib as first line treatment, were examined for EGFR amplification by FISH. We analyzed the relationship between the EGFR mutational status and copy number profile with clin...

Objectives: HTA is a rapidly growing area interest for healthcare systems in APAC. The APAC HTA landscape comprises a patchwork of emerging and developed economies, levels of experience, capabilities, requirements and recommendation frameworks. Cancer drugs represent expensive treatments that benefit from HTA to optimize resource use. APAC has one of the fasting growing cancer populations and an increasing demand for quality care. The current research considers HTA reviews from APAC countries for the last 5 years, assessing number of evaluations, trends in drugs evaluated, probability of success, drivers of success and evidence considerations. Methods: Secondary research of HTA reports in Japan, South Korea, Taiwan, Singapore and Indonesia, narrative synthesis of Information meeting study objectives. Results: 89 reports were identified, overwhelmingly from Korea and Taiwan (79/89). All countries primarily evaluated targeted therapies/immunotherapy drugs (82%-100%) apart from Singapore where chemotherapies were common (40%). CEAs were submitted for 16% of Taiwanese and 60% of Korean submissions. Korea offers CEA waivers for select drugs, instead developing RSAs with manufacturers. CEAs are not mandated for Taiwanese submissions. For all other countries CEAs are universally considered. Taiwan and Singapore considered external HTA evaluations where there was limited data for an economic model. The proportion of successful submissions varied from 0% (Japan) to 86% (Korea, 98% including resubmissions). All except Taiwan have rejected submissions on solely economic grounds. Budget impact was most explicitly considered in Taiwan, where uncertain/high budget impact were used to drive MEAs or price reductions. Korea and Taiwan made extensive use of recommendations with MEAs, RSAs and price reductions. Decisions informed public reimbursement in Korea, Singapore and Taiwan while informing pricing adjustments in Japan. Indonesian decisions informed formulary optimization, uniquely including recommendations for disinvestment. Conclusions: HTA oncology review processes in APAC differ considerably across countries in all categories evaluated. Pan-APAC market access strategies will benefit from country-specific tailoring.

OBJECTIVES: Tumor Necrosis Factor Antagonists (anti-TNF) are effective drugs used in the treatment of several inflammatory disorders. The objective of this study was to evaluate the utilization and pharmacy expenditures of (anti-TNF) drugs in Medicare Part D program in the period of 2014-2015. METHODS: This was a retrospective study using Medicare provider utilization and payment data, which are supplied for prescription drugs that physicians and other healthcare providers prescribed under Medicare Part D from 2014-2015. Descriptive statistics were performed. RESULTS: Five anti-TNF drugs were marketed in the US by 2015 (Adalimumab, Certolizumab, Etanercept, Golimumab and Infliximab). In 2014, there were 697, 428 claims for anti-TNF drugs. However, in 2015 the total claims were reduced to 449,531 claims. The total cost of anti-TNF were significantly higher in 2014 as compared to 2015 ($2.27, 1.70 Billions) respectively (P <0.001). Adalimumab and Etanercept were the most commonly prescribed anti-TNF drugs in 2014 (47%, and 46%) respectively. However, in 2015, a substantial increase in the utilization of Etanercept, (71% of the total claims) accompanied by substantial reduction in the utilization of Adalimumab (17%). During study period, states in the Southern region had the highest number of claims (39% and 40%) respectively. Rheumatologists prescribed the majority of claims in 2014 (64.15%) with substantial increase to (71%) in 2015. The average monthly cost anti-TNF drugs were significantly increased during study period