Purpose This study aimed to elucidate the patient experience of hepatocellular carcinoma (HCC) to guide patient-centered outcome measurement in drug development. Methods Patients with HCC participated in qualitative interviews to elicit disease-related signs/symptoms and impacts, using discussion guides developed from literature searches and discussions with oncologists. Interview participants rated the disturbance of their experiences (0–10 scale). A conceptual model was developed and mapped against patient-reported outcome (PRO) instruments identified from database reviews. Results Interviews were conducted with 25 individuals with HCC (68% were men; median age: 63 years; 12% Barcelona clinic liver cancer (BCLC) stage A; 32% stage B; and 56% stage C) in the USA. Fifty-one HCC-related concepts were identified from the interviews and were grouped into eight sign/symptom categories (eating behavior/weight changes; extremities [arms, legs]; fatigue and strength; gastrointestinal; pain; sensory; skin; other) and four impact categories (emotional; physical; cognitive function; other) for the conceptual model. The most prevalent and disturbing experiences across the disease stages were fatigue/lack of energy and emotional impacts such as frustration, fear, and depression. Abdominal pain and skin-related issues were particularly common and disturbing in individuals with HCC stage C. The EORTC QLQ-C30 and HCC18 were identified as commonly used PRO instruments in HCC studies and captured the relevant signs/symptoms associated with the patient experience. Conclusion Patients with HCC reported a range of signs/symptoms and impacts that negatively affect daily functioning and quality of life. Including PRO measures in HCC clinical trials can provide meaningful patient perspectives during drug development.

Objectives: Untreated hepatitis C virus (HCV) infection results in chronic liver disease. The prevalence in The Netherlands is estimated at 0.1-0.4% with 50% of patients having HCV genotype 1 (GT1). Sofosbuvir (SOF), a novel Direct Antiviral Agent (DAA), reached high rates of sustained virological response (SVR) when given with pegylated interferon-α and ribavirin (PegIFN-α /RBV) in chronic HCV (all genotypes). This study compares the costs per successfully treated patient with sofosbuvir compared to current standard of care (SoC) in the Netherlands in treatment-naïve GT1 patients. MethOds: A Markov transition cost-utility model was used, reflecting efficacy and safety data from published RCTs with SOF+PegIFNα /RBV, PegIFN-α /RBV, telaprevir (TVR) +PegIFN-α /RBV and boceprevir (BOC) +PegIFN-α /RBV. Medical resource use is based on clinical guidelines and expert opinion. Costs include treatment costs, monitoring costs, costs for treatment of complications and adverse events. The model has a lifetime horizon and costs are discounted with 4% and outcomes with 1.5%. Successfully treated patients are defined as having an SVR. Results are presented for a treatment-naïve GT1 population. Results: The SVR rate for SOF ranged from 91.7% in non-cirrhotic patients to 80.1% in cirrhotic patients. This was 43.6% and 23.6% for PegIFN-α , 75.4% and 61.9% in TVR and 64.1% and 55% for BOC. Total treatment costs ranged from € 55,376 to € 70,336 for SOF (non-cirrhotic and cirrhotic), € 22,240 and € 44,751 for PegIFN-α , € 42,593 to € 60,071 for TVR and € 39,634 to € 57,647 for BOC. The costs per SVR varied from € 60.388 to € 87,050 for SOF (non-cirrhotic and cirrhotic), € 51,009 to € 189,623 for PegIFN-α , € 56,489 to € 97,045 for TVR and € 61,832 to € 104,813 for BOC. cOnclusiOns: The costs per successfully treated patient with sofosbuvir are comparable to current standard of care in GT1 treatment naive patients without cirrhosis and are lower than SoC in cirrhotic patients in The Netherlands.

Introduction: Patients living with biliary tract cancer (BTC) experience a decline in healthrelated quality of life (HRQoL). This study aimed to obtain a comprehensive understanding of the patient experience of BTCrelated signs/symptoms and the impacts of these on daily functioning and HRQoL. Methods: Patients with BTC participated in qualitative semi-structured concept elicitation interviews. Signs/symptoms and impacts of BTC were initially explored by targeted literature searches and interviews with five clinicians. Patient interviews were transcribed and coded using qualitative research software. Concept saturation was assessed over five interview waves. A sign/symptom or impact was defined

Objectives: NICE of England and SMC of Scotland are responsible for issuing appraisal guidance for reimbursement of new health technologies. While both agencies consider clinical and cost-effectiveness in their decisions, they have unique policies and procedures and may yield different outcomes. We conducted an analysis of past appraisal decisions by NICE and SMC for oncology treatments to compare results and identify trends. MethOds: Oncology appraisal decisions from January 2012 to May 2017 were identified on the SMC website and Xcenda's Health Technology Assessment (HTA) Decision map. Appraisal decisions for both countries were evaluated and categorized as favorable, mixed, or unfavorable. Additionally, reported incremental cost-effectiveness ratio (ICER) and tumor type were compared for each decision. SMC decisions that were classified as withdrawn or superseded were not included. Results: A total of 91 decisions were identified for NICE, of which 64.8% (59) were favorable, 3.3% (3) were mixed, and 31.9% (29) were unfavorable. 110 decisions were identified for SMC, of which 40.9% (45) were favorable, 26.4% (29) were mixed, and 32.7% (36) were unfavorable. Between agencies, there were 53 pairs of decisions that were matched across 15 tumor types; of which, 62.3% (33) matched exactly, 24.5% (13) were similar (one agency's decision was favorable and the other agency's decision was mixed), and 13.2% (7) were in disagreement. All 18 NICE decisions with reported ICERs less than £30,000 received favorable guidance; but, the guidance for SMC was more varied. For the 25 SMC decisions with a reported ICER of less than £30,000, 12 were favorable, 10 were mixed, and 3 were unfavorable. cOnclusiOns: The majority (86.8%) of oncology HTA decisions by NICE and SMC were either the same or similar across tumor types. However, compared to NICE, there appears to be less association between the reported ICERs and SMC appraisal decision results.

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