This NMA provides comparative efficacy for MM treatments not studied in head-to-head RCTs. The NMA suggests that, compared with other approved MM treatments in the United States, DRd and DVd have a higher probability of providing the longest PFS in patients who have received at least 1 prior therapy and in patients who have received only 1 prior therapy.

Our findings support the use of OC-Sensor for CRC detection. The diagnostic estimates obtained may be extended to derive model parameters for economic decision models and to offer insight for future clinical and public health decision making. Our findings could influence the future of FOBTs within the CRC screening arsenal.

Objectives: To report resource utilisation and annual cost for chronic immune thrombocytopenia (ITP) patients enrolled consecutively and followed for one year.Methods: A single-center, single-arm, retrospective, one-year observational cohort study of adult patients with chronic ITP from a French hospital. Healthcare resource utilisation and mean cost per patient (with 95% confidence intervals) were estimated for the whole group.Patients requiring at least one hospitalisation formed subgroup 1. Patients with the most severe category of disease formed subgroup 2 (defined as hospitalised patients with ≥1immunoglobulin (IVIg) infusion [usually reserved for those with bleeding score >8]).Results: Fifty-seven patients (42F/15M) with a mean age of 48 years (SD: 19) at ITP diagnosis were studied. Mean platelet count at diagnosis was 28±26x10 9 /L. Mean duration of ITP was 3.1 years (SD: 2); 8 patients had undergone splenectomy at baseline. Subgroup 1 included 27 patients who were hospitalised (full hospitalisation, n=23; and/or day hospitalisation, n=8). Of those, 12 patients received at least one IVIg infusion during hospitalisation (subgroup 2). Total mean cost per patient for the one-year study period was €7,293 (95% CI = 3,584) for the whole group, €15,334 (95% CI = 7,876-27,459) for subgroup 1, and €26,581 (95% CI = 12,578) for subgroup 2. IVIg accounted for 33% of costs for subgroup 1 and up to 43% of costs for patients with more severe disease (subgroup 2).Conclusions: Management of adults with chronic ITP is costly in France, especially for patients with severe disease. IVIg use was a major cost driver.

Background and objectivesThe objective of our paper is to offer a new, payer-friendly taxonomy of market entry agreements (MEAs) that aims to twin contracts with their methodological designs in an effort to clarify the distinction between contracts that are based on performance and those that are based on demonstrated effect.MethodsOur analysis proceeds in two stages: First, we delimit the scope and framework of pay for performance (P4P) and pay for demonstrated effect (P4E) agreements. Second, we distinguish the methodological designs supporting the implementation of each of these contracts.ResultsWe elucidate why P4P contracts prevent the payer from funding the true effectiveness of an innovation by expanding on their limitations. These include: 1) the normative nature of comparisons, 2) the impossibility of true effect imputability for each individual, and 3) the use of intermediary outcome measures. We then explore three main criticisms that payers must take into account when reasoning in terms of performance rather than in terms of the product effectiveness.ConclusionThe potential effect that performance-based reimbursements may have on dissociating the components of the cost-effectiveness ratio constitutes an obstacle to a true health economic reasoning.

8042 Background: Treatment for multiple myeloma (MM) in the US has undergone significant advances, with several new therapies recently FDA approved for relapse/refractory MM (RRMM), including carfilzomib+lenalidomide+dex (KRd), carfilzomib+dex (Kd), daratumumab+lenalidomide+dex (DRd), daratumumab+bortezomib+dex (DVd), ixaxomib+lenalidomide+dex (IRd), and elotuzumab+lenalidomide+dex (ERd). These new therapies have shown improvements in clinical outcomes in randomized controlled trials (RCTs). However, with few head-to-head RCTs, there is little comparative evidence to determine the most effective treatment for specific patients. A systematic literature review (SLR) and network meta-analysis (NMA) was conducted to determine the comparative efficacy (progression free survival (PFS)) of MM therapies for treating first relapse. Methods: The SLR searched MEDLINE, Embase, and the Cochrane Library for RCTs investigating the efficacy of treatments for RRMM (to August 2016). NMA was conducted on the PFS hazard ratios (HR), where available in RCTs for patients with one prior line of treatment, using Bayesian fixed effects mixed treatment comparisons. Results: Data formed two evidence networks. Network 1: RCTs with Rd; Network 2: RCTs with Vd. Analyses found DRd and DVd had the highest probability of being the best treatment (0.96 and 0.89, respectively). Compared to other MM therapies, DRd and DVd had the lowest risk of progression or death (PFS HR <1.0) (Table 1). For example, compared to KRd, DRd had a 41% (PFS HR 0.59) reduced risk of progression or death. Conclusions: This analysis provides comparative evidence among treatments where head-to-head RCTs have not been conducted. For treating first relapse, compared to other MM treatments, this analysis found that DRd and DVd had the highest probability of providing the longest progression free survival. [Table: see text]