UK health technology assessment of orphan drugs for rare oncology versus rare disease: Does orphan designation make any difference in HTA process?

November 19, 2018

The orphan drug (OD) technology appraisal (TA) rate is low with an absence of TAs in half of indications by NICE and a third by SMC. Published SMC TAs steadily increased to plateau in 2016-17, versus an exponential increase with 40% published in 2017 by NICE. NICE is less likely to appraise rare disease (RD) than SMC. As NICE is more selective, the likelihood of recommendation is higher (a quarter of SMC TAs are not recommended). Variation exists on undertaking HTAs, leading to absence of guidance in England and a higher proportion not recommended in Scotland despite PACE being introduced in May 2014. For TAs common to both NICE and SMC the level of consensus is high, but with some disparity for RD. Increased consistency on topic selection, a quicker approval process and more TAs undertaken (particularly for RD) are needed to assess the success of HTA processes for ODs.

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