HTA and Reimbursement Considerations for Rare Diseases in European Markets: What Are the Implications for Manufacturers?
Heyes A, McBride D, Pearson I, Copley-Merriman C
More companies are developing orphan drugs for rare diseases. But the available evidence to support orphan drug reimbursement is often of lower quality due to smaller patient populations available for clinical studies. Several European countries have introduced special considerations for rare diseases, but evidence guidelines for orphan and nonorphan drugs remain similar. This makes economic evaluations and subsequent application of general HTA rules challenging for orphan drug manufacturers.
We have reviewed special HTA and reimbursement considerations introduced for assessment of orphan drugs in Europe and present implications for manufacturers in this research. In Scotland and Germany, special HTA consideration provides flexibility around evidence levels. In France and Germany, ‘proven additional benefit’ is accepted if the budget impact falls below a maximum threshold. In Sweden, the TLV takes a flexible approach to reimbursement, based on the level of unmet need and economic uncertainty. And in England, NICE's Highly Specialised Technology Programme takes a similar approach.
To ensure equitable decision-making, manufacturers should use a range of evidence sources and techniques, including comparative real-world information, to bridge data gaps and address economic uncertainty.
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