A Multinational European Study of Patient Preferences for Novel Diagnostics to Manage Antimicrobial Resistance.

Background: Novel diagnostics are needed to manage antimicrobial resistance (AMR). Patient preferences are important in determining whether diagnostic tests are successful in practice, but there are few data describing the test attributes which matter most to patients. We elicited patients' preferences for attributes of diagnostic tests that could be used to reduce unnecessary antibiotic use in primary care across seven European countries.

An economic analysis of human milk supplementation for very low birth weight babies in the USA.

Background: An exclusive human milk diet (EHMD) using human milk based products (pre-term formula and fortifiers) has been shown to lead to significant clinical benefits for very low birth weight (VLBW) babies (below 1250 g). This is expensive relative to diets that include cow's milk based products, but preliminary economic analyses have shown that the costs are more than offset by a reduction in the cost of neonatal care. However, these economic analyses have not completely assessed the economic implications of EHMD feeding, as they have not considered the range of outcomes affected by it.

A framework to guide the optimal development and use of real-world evidence for drug coverage and formulary decisions.

Aim: To provide a framework for optimizing the development and use of real-world evidence (RWE) in drug coverage decisions. Materials & methods: The Institute for Clinical and Economic Review convened a Policy Summit with representatives from 23 payer and life science companies that compose the Institute for Clinical and Economic Review membership.

Results: Summit participants helped refine a new conceptual framework that emphasizes the central role of contextual considerations and the evidentiary argument that the RWE is intended to support in designing the process for the development and interpretation of RWE.

Real-world evidence for coverage decisions: opportunities and challenges.

Aim: To explore current uses of real-world evidence (RWE) in the US healthcare system, summarize key concerns and highlight various opportunities that could be realized through best use of RWE. Materials & methods: Information was gathered via a literature review and interviews to generate a background paper for the 2017 Institute for Clinical and Economic Review Policy Summit meeting.

Results: RWE is currently being utilized in drug development decisions, regulatory approval decisions, post-approval monitoring, payer coverage decisions (initial decisions and reassessments) and for outcomes-based contracting.

Advanced therapy medicinal products and health technology assessment principles and practices for value-based and sustainable healthcare.

Background: Advanced therapy medicinal products (ATMPs) are beginning to reach European markets, and questions are being asked about their value for patients and how healthcare systems should pay for them.

Objectives: To identify and discuss potential challenges of ATMPs in view of current health technology assessment (HTA) methodology-specifically economic evaluation methods-in Europe as it relates to ATMPs, and to suggest potential solutions to these challenges.

Methods: An Expert Panel reviewed current HTA principles and practices in relation to the specific characteristics of ATMPs.

Applying a Multicriteria Decision Analysis (MCDA) Approach to Elicit Stakeholders' Preferences in Italy: The Case of Obinutuzumab for Rituximab-Refractory Indolent Non-Hodgkin Lymphoma (iNHL).

Background: Healthcare decision makers need to make trade-offs between different elements of value of new treatments. Multicriteria decision analysis (MCDA) provides a framework that can help decision makers to understand stakeholders' preferences and be explicit about the trade-offs that are being made.

Objective: The objective of this study was to use MCDA to obtain preferences and views on decision criteria across three stakeholder groups (patients, clinicians and payers) in Italy and to use these to assess the performance of obinutuzumab for rituximab-refractory indolent non-Hodgkin lymphoma (iNHL).

Gene therapy: evidence, value and affordability in the US health care system.

Aims: To explore the challenges presented by gene therapies, discuss potential solutions, and present policy recommendations.

Methods: A review of the literature and series of expert interviews were conducted and discussed at a Policy Forum convened by The Institute for Clinical and Economic Review (ICER). The Policy Forum was attended by independent experts and senior representatives from 20 payer organizations and life sciences companies.

ASSESSING VALUE, BUDGET IMPACT, AND AFFORDABILITY IN ASIA.

Objectives: The aim of this study was to explore definitions of value and the use of budget impact and affordability considerations in health technology assessment (HTA) in the Asia region, particularly in relation to high cost technologies.

Methods: Issues were debated by senior representatives from HTA and payer systems in Asian countries, delegates from industry, and invited experts at the 2016 meeting of the HTAi Asia Policy Forum (HAPF). A premeeting survey was used to gather data on how value is assessed and budget impact calculations are used within current processes, as well as current approaches to managing affordability.

Short duration immunochemotherapy followed by radioimmunotherapy consolidation is effective and well tolerated in relapsed follicular lymphoma: 5-year results from a UK National Cancer Research Institute Lymphoma Group study.

Unlabelled: We report a phase II study to evaluate the efficacy and toxicity of abbreviated immunochemotherapy followed by (90) Y Ibritumomab tiuxetan ((90) Y-IT) in patients with recurrent follicular lymphoma. Of the 52 patients enrolled, 50 were treated with three cycles of R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, prednisolone) or R-CVP (rituximab, cyclophosphamide, vincristine, prednisolone), followed by (90) Y-IT regimen (15 MBq/kg, maximum 1200 MBq) preceded by two infusions of 250 mg/m(2) rituximab. The overall response rate was 98% with complete response (CR) 30% and partial response (PR) 68%.