Elisabeth George

Relative Effectiveness Assessment of Pharmaceuticals: Similarities and Differences in 29 Jurisdictions

OBJECTIVE Assessment of the effectiveness compared with alternative treatment(s) plays an important role in many jurisdictions in determining the reimbursement status of pharmaceuticals. This type of assessment is often referred to as a relative effectiveness assessment (REA) and is carried out by many jurisdictions. Increased sharing of information across jurisdictions may save costs and reduce duplication. The objective of this study was to explore the main similarities and differences in the major methodological aspects of REA in multiple jurisdictions. METHODS Data were gathered with a standardized data extraction form by searching publicly available information and by eliciting information from representatives at relevant organizations. RESULTS Of the initially included 35 jurisdictions, data were gathered for 29 jurisdictions. There seem to be substantial similarities on the choice of the comparator, the role of indirect comparisons, and preferred end points in REAs (except for the use of health state utilities). Jurisdictions, however, differ in whether effectiveness (usual circumstances of health care practice) is estimated in case no (comparative) effectiveness data are available and how this is done. CONCLUSION Some important methodological aspects for REA are approached in a similar way in many jurisdictions, indicating that collaboration on assessments may be feasible. Enhanced collaboration in the development of methods and best practices for REA between jurisdictions will be a necessary first step. Important topics for developing best practice are indirect comparisons and how to handle the gap between efficacy and effectiveness data in case good quality comparative effectiveness data are not yet available at the time of reimbursement decisions.

Utility values in National Institute for Health and Clinical Excellence (NICE) Technology Appraisals.

OBJECTIVE To review the selection and use of health-related utility values for economic models included in National Institute for Health and Clinical Excellence (NICE) Technology Appraisals. METHOD A cross-sectional review of reports of economic models submitted to the Technology Appraisals program was undertaken to review the health-related utility data included. Data reviewed included identification and selection of data and the methods used for utility elicitation. The methods used were compared with those from the 2004 Methods Guide issued by NICE. RESULTS Appraisals conducted after the implementation of the NICE 2004 Methods Guide were reviewed. After exclusion of documents that did not include a de novo cost-utility analysis, 71 submissions (53 manufacturer submissions, 18 assessment group reports) from 39 appraisals were identified, containing 284 unique utility values. Variation was found in the selection, elicitation, valuation, and use of the utility values. Thirty-nine submissions (55%) took utility values from published studies, of which only 31% were identified through a systematic review. Forty-seven (66%) submissions contained health state descriptions reported by patients, and 55 (77%) submissions applied a valuation set derived from the general population. The EQ-5D was used in 35 (49%) submissions, and mapping to a generic health-related quality of life measure was performed in 19 (27%) submissions. CONCLUSIONS Only 56% of submissions to NICE and assessment reports included utility values that met the NICE 2004 reference case. This highlights variation in the methods used to select and incorporate utility values in economic models for NICE Technology Appraisals.

European collaboration on relative effectiveness assessments: What is needed to be successful?

OBJECTIVE The objective of this study is to identify the possible barriers and critical success factors for the implementation of European collaboration in the field of relative effectiveness assessment (REA) of drugs. METHODS Data were gathered through semi-structured interviews with representatives from eight European health technology assessment (HTA) organisations involved in assessment of drugs for coverage decision-making (AAZ, AIFA, AHTAPol, HAS, HVB, IQWIG, NICE and ZiN). RESULTS Potential barriers identified mainly relate to methodology, resources and challenges with implementation in the respective national processes (e.g. legal restrictions). The most critical success factors for production of cross-border assessments were the continuous cooperation of competent partners, and the quality and timely availability of the assessment. CONCLUSION Further adaptation of the process and methods is required for optimal collaboration. In the near future it can be expected that cross-border assessments will meet in particular the needs of smaller/middle-sized European countries and also European countries with less developed HTA systems as the potential efficiency/quality gains are the highest for these countries. Therefore, national implementation of cross-border assessments is especially likely in these countries in the coming years. Once more experience is gained with cross-border assessments, and successes become more evident, efficiency/quality gains may also be likely for some larger countries with well established processes.

How real-world data compensate for scarce evidence in HTA

Most guidance developed by NICE is based on a value assessment using clearly articulated and published clinical and cost effectiveness criteria. In order to enable consistency and fairness across all decisions, NICE uses as a unit of health benefit the quality-adjusted life year (QALY). Both QALYs and costs for a technology are estimated by long-term disease modelling. This requires a variety of clinical input parameters, and often extrapolation beyond the trial period, and of intermediate or surrogate to final outcomes. RCT data will remain the main data source for the majority of appraisals, but because the data necessary for disease modelling is often not available from RCTs, particularly for the UK context, the use of non-RCT data is the norm in NICE technology appraisals. This does not only apply to data on resource use, service provision and HRQL data, but also to efficacy data. In some situations non-RCT data are more relevant to a decision context than the RCT data, and in some situations, as illustrated by 3 examples, it would be unreasonable, not to take account of existing non-RCT data. The use of non-RCT clinical evidence is most common for devices, interventions where RCTs are difficult, and in conditions with poor prognosis where single arm studies are often carried out. Therefore, a pragmatic approach to the available evidence is needed for many decision made by the NICE Appraisal Committees to come to a reasonable and defendable decision.

Cancer Drugs Fund: the bigger picture

We welcome the authors’ creative ideas on the appraisal of cancer drugs.1 But they are wrong to imply that the Cancer Drugs Fund exists only to support observational data collection.2 With the drug industry playing its part through fair pricing, patients can access NICE recommended treatments up to 8 …