Objective: Health technology assessment (HTA) is a process of assessing evidence to inform policy decisions about public subsidy of new drugs and medical procedures. Where evidence is uncertain but the technology itself is promising, funders may recommend funding on an interim basis. It is unknown whether evidence from clinical registries is used to resolve uncertainties identified in interim-funded decisions made by Australian HTA bodies. Therefore, the present study evaluated the role of evidence from clinical registries in resolving evidence uncertainties identified by the Medical Services Advisory Committee (MSAC).
Methods: All HTAs considered by MSAC between 1998 and 2015 were reviewed and assessments that recommended interim funding were identified. The MSAC website was searched to identify reassessments of these recommendations and sources of evidence used to resolve the uncertainties were identified.
Results: Of 173 HTA reports considered by MSAC, 17 (10%) contained an interim funding recommendation. Eight recommendations cited uncertainty around safety, 15 cited uncertainty around clinical effectiveness and 13 cited uncertainty around economics (cost-effectiveness and/or budget impact). Of the 17 interim funding recommendations, 11 (65%) have been reassessed. Only two reassessments relied on clinical registry evidence to resolve evidence gaps identified at the time of the interim funding recommendation.
Conclusions: Clinical registries are underused as a source of evidence for resolving uncertainties around promising new health technologies in Australia. An open dialogue between stakeholders on the role of registries in this context is needed.
What is known about the topic? HTA is a process of assessing the evidence to inform policy decisions about public subsidy of new health technologies (e.g. pharmaceuticals, diagnostic tests, medical procedures). Where evidence is uncertain but the technology under evaluation is promising, funders may recommend the funding of the technology on a temporary basis while additional evidence is collected. Clinical registries have been suggested as a means of collecting additional evidence in these situations.
What is does this paper add? It is currently unknown whether evidence from clinical registries is used to resolve uncertainties identified at the time that temporary (interim) funding decisions are made by Australia's HTA bodies, in particular MSAC. The present study found that MSAC rarely relies on the interim funding mechanism (17/173 assessments). Of the 11 subsequent reassessments of interim recommendations, two relied on registry evidence to provide Australian-specific data for addressing uncertainties around long-term safety, effectiveness and cost-effectiveness. These findings suggest that clinical registries, although a feasible source of evidence for HTAs, are rarely used for this purpose.
What are the implications for practitioners? Given the registries' ability to resolve both a wider range of questions than those typically addressed by randomised control trials and applicability to a wider group of patients (and, hence, providing estimates of outcomes that are more generalisable), the potential of clinical registries to resolve HTA issues needs more attention from both researchers and decision makers. Stakeholder collaboration to define the evidence requirements for new technologies early in their development phase would be valuable to determine the potential role for clinical registries.