A VALUE FOR MONEY THRESHOLD FOR NEW TECHNOLOGIES FOR VERY RARE DISEASES- DOES THE NICE HIGHLY SPECIALISED COMMITTEE EMPLOY ONE?
Author(s)
Kusel J1, Griffiths SA2, Spoors J3
1Costello Medical Consulting Ltd, Cambridge, UK, 2Costello Medical Consulting Ltd., Cambridge, UK, 3RJW & Partners, Royston, UK
OBJECTIVES: The Highly Specialised Technology (HST) programme at NICE appraise new technologies for orphan/ultra-orphan conditions and use a cost-consequence approach to assess whether the technologies provide sufficient value for money to the NHS. Whilst NICE’s Technology Appraisal programme employs a threshold of £20,000-£30,000/QALY, the HST Committee currently does not publish a threshold. METHODS: All materials published by 11/05/16 on the NICE website regarding completed or in-process appraisals by the HST Committee were reviewed. The quality-adjusted life year (QALY) gains most favoured by the Committee, the price per patient, the annual budget impact of introducing the new technology and the Committee decision were extracted. The data were analysed to assess whether a ‘value for money’ threshold could be deduced. RESULTS: Three completed (eculizumab, elosulfase-alfa, ataluren) and two in process (asfotase-alfa, sebelipase-alfa) appraisals had published documents that reported the desired outcomes. Only one completed appraisal (eculizumab) gained a recommendation at list price. All other technologies were rejected at list price; elosulfase-alfa and ataluren had to offer managed access agreements (MAA) incorporating confidential discounts to obtain approval. Incremental costs were confidential in manufacturer submissions except for ataluren; in this case, the incremental cost per QALY (prior to MAA) was calculated to be approximately £1.6 million, at which ataluren was rejected. Eculizumab had the highest budget impact (at list prices) in year 1 (£57.8 million), which was only overtaken by asfotase-alfa and sebelipase-alfa in year 5, indicating that budget impact alone is unlikely to be the deciding factor for the NICE HST Committee. CONCLUSIONS: There is clearly a threshold at which the cost becomes too high to offer sufficient value for money for the NHS, as technologies have been rejected by HST at draft stage. However, this analysis was limited by the small number of HST appraisals published thus far and by confidential prices.
Conference/Value in Health Info
2016-10, ISPOR Europe 2016, Vienna, Austria
Value in Health, Vol. 19, No. 7 (November 2016)
Code
PSY156
Topic
Health Technology Assessment
Topic Subcategory
Decision & Deliberative Processes
Disease
Multiple Diseases, Rare and Orphan Diseases