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. METHODS: We conducted an economic analysis of EHMD compared to usual practice of care amongst VLBW babies in the US, which is to include cow's milk based products when required. Costs were evaluated from the perspective of the health care payer, with societal costs considered in sensitivity analyses. RESULTS: An EHMD substantially reduces mortality and improves other health outcomes, as well as generating substantial cost savings of $16,309 per infant by reducing adverse clinical events. Cost savings increase to $117,239 per infant when wider societal costs are included. CONCLUSIONS: An EHMD is dominant in cost-effectiveness terms, that is it is both cost-saving and clinically beneficial, for VLBW babies in a US-based setting.

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. RESULTS: All systems consider health benefit to be the key component of value. There is little consensus around "wider" elements of value that should be included. All systems use budget impact in decision making, although meeting attendees noted the challenges in making accurate estimates. The most common strategies used to address affordability concerns to date have been: restricting coverage, for example, to patients who are likely to get the highest value; discounts; and revenue caps. It was noted that these "solutions" may have unintended consequences of creating inequitable access to therapies and failing to provide adequate rewards for innovation. CONCLUSIONS: Decision makers, HTA agencies, and industry need to continue to work together to find mutually agreeable solutions to ensure that patients continue to get equitable access to effective therapies at costs that can be afforded throughout the Asia region.

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%. 18 patients with a PR following chemotherapy improved to a CR following (90) Y-IT: a conversion rate of 40%. Seven patients with PR following (90) Y-IT subsequently improved to a CR 12-18 months later, leading to an overall CR rate of 44%. With a median follow-up of 5 years, median progression-free survival was 23·1 months and overall survival was 77·5% at 5 years. High trough serum rituximab levels (median 112 µg/ml; range 52-241) were attained after four doses of rituximab, prior to (90) Y-IT; this was not found to influence response rates. The treatment was well tolerated with few (13·5%) grade 3 or 4 infective episodes and manageable haematological toxicity. Abbreviated immunochemotherapy followed by (90) Y-IT is an effective and well-tolerated treatment in recurrent follicular lymphoma patients previously exposed to rituximab. TRIAL REGISTRATION: clinicaltrials.gov identifier: NCT00637832.

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. METHODS: We used an online stated preference survey, including a discrete choice experiment (DCE). The DCE explored how patients make trade-offs between three key attributes of diagnostic tests: the speed that results were available, confidence in the test results, and how convenient it is to take the test. Individuals were eligible to complete the survey if they had taken antibiotics within the last 2 years and were resident in Germany, Italy, Spain, France, Greece, the Netherlands or the United Kingdom (UK). RESULTS: In total, 988 respondents completed the survey. The DCE responses illustrated that speed was the least important attribute in most countries. Responses from Germany and the Netherlands indicated that confidence was most important in these countries. Responses from the UK, France, Spain and Italy showed convenience as the most important attribute in these countries. Two attributes, confidence and convenience, were jointly favoured by respondents in Greece. CONCLUSION: Patients in different European countries do not have the same preferences for the attributes of diagnostic tests to manage AMR in primary care. Failure to account for such differences during test development could reduce test uptake, result in continued overuse of antibiotics, and hamper marketisation.

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. RESULTS: Three key topics were identified and prioritised for discussion-uncertainty, discounting, and health outcomes and value. The panel discussed that evidence challenges linked to increased uncertainty may be mitigated by collection of follow-on data, use of value of information analysis, and/or outcomes-based contracts. For discount rates, an international, multi-disciplinary forum should be established to consider the economic, social and ethical implications of the choice of rate. Finally, consideration of the feasibility of assessing the value of ATMPs beyond health gain may also be key for decision-making. CONCLUSIONS: ATMPs face a challenge in demonstrating their value within current HTA frameworks. Consideration of current HTA principles and practices with regards to the specific characteristics of ATMPs and continued dialogue will be key to ensuring appropriate market access. CLASSIFICATION CODE: I.

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. Solutions to key challenges and opportunities for future development are presented. CONCLUSION: Exciting opportunities for the use of RWE exist, yet important reservations remain. Solutions are within reach if effective partnerships between stakeholders can be nurtured.

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. CONCLUSION: This framework may provide a structured way for pharmaceutical manufacturers and payers to develop a shared understanding of the best way to develop RWE that will ultimately be useful in informing coverage and formulary decisions.

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). METHODS: We used EVIDEM V3.0, an MCDA framework, and collected participants' preferences via an online survey and structured meetings. RESULTS: Patients and clinicians expressed a preference for interventions targeting severe conditions. Payers expressed preference for treatments targeting areas with an unmet need, which are cheaper than the comparator, and with high-quality evidence. Obinutuzumab in combination with bendamustine, compared with bendamustine alone, received high positive scores for the criteria 'disease severity' and 'type of therapeutic benefit' by all three groups, and negative scores on the economic-related criteria, according to all stakeholder groups. CONCLUSIONS: MCDA can be used to elicit the views of different stakeholder groups and has the potential to structure and inform reimbursement decisions.

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. RESULTS: Three categories of challenges are identified: evidence generation, assessing value and affordability. Possible solutions and policy recommendations are presented for each of the three main categories of challenges. CONCLUSIONS: Gene therapies present exciting opportunities, but also pose major challenges. Dialogue between manufacturers and payers around the issues and possible solutions is crucial.