Only in Research (OIR) versus Approval with Research (AWR): An Assessment
Whether widespread approval of a technology should be withheld until research findings are available (an only in research policy), or granted while the research is being conducted, (an approval with research policy), requires a comparison of the value of early access to a technology and the value of additional evidence which will accrue to future patients. Establishing the key principles of what assessments might lead to approval ‘only in research’ (OIR) or ‘approval with research’ (AWR) will enable them to be addressed in an explicit and transparent manner, with wider relevance beyond the National Institute for Health and Clinical Excellence (NICE) and the UK health service. The key findings of research funded by the Medical Research Council and NIHR in the UK to inform when NICE should recommend technologies only in the context of research, has recently been published, with the full report due to be published in early 2012 in the NIHR Health Technology Assessment Journal. Please find the summary of the key research findings here: Uncertainty, evidence and irrecoverable costs: Informing approval, pricing and research decisions for health technologies
This research aimed to establish the key principles of what assessments are needed to inform OIR recommendations and evaluate how these assessments might be informed by additional information and analysis through a series of case studies.
The key principles and assessments needed fall into four broad areas: i) expected cost-effectiveness and population net health effects; ii) the need for evidence and whether the type of research required can be conducted once a technology is approved for widespread use; iii) whether there are sources of uncertainty which will resolve over time; and iv) whether there are significant (opportunity) costs which will be committed and cannot be recovered once the technology is approved.
The sequence of assessment and judgment required is represented as an algorithm, which can also be summarised as a simple set of explicit criteria or a seven point checklist of assessments. This sequence of assessment identifies how different policies might be arrived at; the order in which they might be made; and how similar policies might be arrived at through different combinations of considerations. These principles demonstrate that cost-effectiveness is only a necessary but not sufficient condition for approval since OIR may be appropriate when a technology is expected to be cost-effective. Indeed, if there are significant irrecoverable costs OIR may be appropriate even when research is possible with approval. A more explicit assessment of OIR and AWR, founded on this sequence of assessment and judgement has implications for drug pricing and incentives for evaluative research, as well as the process and the methods of appraisal of health technologies.
The application of the check list of assessment to the series of case studies also show how analysis can be used to consider the value of: i) being able to conduct research while a technology is approved; ii) making evidence that is needed available at launch; and iii) acquiring evidence more quickly. This can inform investments in better data collection, early advice to manufacturers from payers, public investment in transitional and evaluative research earlier in the development process or other incentives for research and development.
The relevance of this work to NICE was evaluated through a series of two workshops involving key stakeholders. The briefing documents, which formed the basis of the workshop presentations and related group discussions, as well as a summary of feedback from participants which informed the recommendations is available here: Informing a decision framework for when NICE should recommend the use of health technologies only in the context of an appropriately designed programme of evidence development