Using Real-World Data in Health Technology Assessment (HTA) Practice:A Comparative Study of Five HTA Agencies

BACKGROUND: Reimbursement decisions are conventionally based on evidence from randomised controlled trials (RCTs), which often have high internal validity but low external validity. Real-world data (RWD) may provide complimentary evidence for relative effectiveness assessments (REAs) and cost-effectiveness assessments (CEAs). This study examines whether RWD is incorporated in health technology assessment (HTA) of melanoma drugs by European HTA agencies, as well as differences in RWD use between agencies and across time. METHODS: HTA reports published between 1 January 2011 and 31 December 2016 were retrieved from websites of agencies representing five jurisdictions: England [National Institute for Health and Care Excellence (NICE)], Scotland [Scottish Medicines Consortium (SMC)], France [Haute Autorité de santé (HAS)], Germany [Institute for Quality and Efficacy in Healthcare (IQWiG)] and The Netherlands [Zorginstituut Nederland (ZIN)]. A standardized data extraction form was used to extract information on RWD inclusion for both REAs and CEAs. RESULTS: Overall, 52 reports were retrieved, all of which contained REAs; CEAs were present in 25 of the reports. RWD was included in 28 of the 52 REAs (54%), mainly to estimate melanoma prevalence, and in 22 of the 25 (88%) CEAs, mainly to extrapolate long-term effectiveness and/or identify drug-related costs. Differences emerged between agencies regarding RWD use in REAs; the ZIN and IQWiG cited RWD for evidence on prevalence, whereas the NICE, SMC and HAS additionally cited RWD use for drug effectiveness. No visible trend for RWD use in REAs and CEAs over time was observed. CONCLUSION: In general, RWD inclusion was higher in CEAs than REAs, and was mostly used to estimate melanoma prevalence in REAs or to predict long-term effectiveness in CEAs. Differences emerged between agencies' use of RWD; however, no visible trends for RWD use over time were observed

Statelessness Determination in the Netherlands

The Netherlands lacks an adequate mechanism for the identification of stateless persons, and is therefore not complying with its international obligations in the field of statelessness. Contrary to the position of the Dutch government, the existing procedures that stateless persons can appeal to are not in conformity with the relevant international standards. This paper contains a detailed analysis of the two procedures which have been invoked by the Dutch government in defence of the current statelessness protection and identification regime in the Netherlands, namely the registration of individuals in population registers, and the procedure for obtaining the so-called ‘no-fault’ residence permit. It illustrates that neither of the two are effective alternatives for statelessness determination. In addition, recent legislative changes that might affect the registration of statelessness in the Netherlands are discussed, and a proposal put forward by the UNHCR for a determination procedure in courts is evaluated