Akehurst RL1, Moorhouse JA1, Jones E2, Mason N3, Schurer M4, Chapman AM1, Horscroft J1
1BresMed Health Solutions, Sheffield, UK, 2BresMed Health Solutions, Manchester , UK, 3BresMed, Sheffield, UK, 4BresMed Netherlands BV, Utrecht, UT, Netherlands

OBJECTIVES: Until 2017, the National Institute for Health and Care Excellence (NICE) in England was the only health technology assessment (HTA) agency that had a separate HTA process for products with very rare indications. Since then, the Australian Department of Health, the Scottish Medicines Consortium (SMC) and the Institute for Clinical and Economic Review (ICER) in the US have implemented similar processes. We compare the processes in these countries and consider the implications for access and reimbursement of technologies for very rare diseases in other countries.

METHODS: Targeted reviews of grey and published literature were conducted to capture relevant information on processes for drugs for very rare diseases in the four countries of interest.

RESULTS: While all countries note similar challenges (e.g. small population size and limited knowledge of natural history), they vary in their approaches to evaluation. In all countries, there is a wider consideration of the nature and impact of the condition than in the standard evaluation process; however, the countries differ in which aspects should be evidenced and how. In Australia and the US, evidence quality is assessed in the same way as for more common conditions, but in England and Scotland, less robust sources of evidence, such as observational studies, are acceptable. All countries recognise that there will be higher levels of uncertainty. Additional variations include: definition of ‘very rare’, analytical perspective, stakeholder consultation, and requirements for initial standard review.

CONCLUSIONS: All countries acknowledge the need for alternative decision-making criteria when evaluating drugs for very rare diseases, but differ in their processes and requirements. To ensure early access to new technologies for patients with very rare diseases and account for higher levels of uncertainty, these and other countries may need to routinely accept ongoing real-world evidence collection post-HTA approval and subsequent reassessment.

Conference/Value in Health Info

2019-11, ISPOR Europe 2019, Copenhagen, Denmark




Health Policy & Regulatory, Health Technology Assessment, Organizational Practices

Topic Subcategory

Decision & Deliberative Processes, Geographic & Regional, Reimbursement & Access Policy, Value Frameworks & Dossier Format


Multiple Diseases

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