Uncertainty, evidence and irrecoverable costs: informing approval, pricing and research decisions for health technologies

The general issue of balancing the value of evidence about the performance of a technology and the value of access to a technology can be seen as central to a number of policy questions. Establishing the key principles of what assessments are needed, as well as how they should be made, will enable them to be addressed in an explicit and transparent manner. This report presents the key finding from MRC and NHIR funded research which aimed to: i) Establish the key principles of what assessments are needed to inform an only in research (OIR) or Approval with Research (AWR) recommendation. ii) Evaluate previous NICE guidance where OIR or AWR recommendations were made or considered. iii) Evaluate a range of alternative options to establish the criteria, additional information and/or analysis which could be made available to help the assessment needed to inform an OIR or AWR recommendation. iv) Provide a series of final recommendations, with the involvement of key stakeholders, establishing both the key principles and associated criteria that might guide OIR and AWR recommendations, identifying what, if any, additional information or analysis might be included in the Technology Appraisal process and how such recommendations might be more likely to be implemented through publicly funded and sponsored research. The key principles and the assessments and judgments required are discussed in Section 2. The sequence of assessment and judgment is represented as an algorithm, which can also be summarised as a simple set of explicit criteria or a seven point checklist of assessments. The application of the check list of assessment to a series of four case studies in Section 3 can inform considerations of whether such assessments can be made based on existing information and analysis in current NICE appraisal and in what circumstances could additional information and/or analysis be useful. In Section 4, some of the implications that this more explicit assessment of OIR and AWR might have for policy (e.g., NICE guidance and drug pricing), the process of appraisal (e.g., greater involvement of research commissioners) and methods of appraisal (e.g., should additional information, evidence and analysis be required) are drawn together