Real-world data from expanded access programmes in health technology assessments: a review of NICE technology appraisals 119 6♦ Furthermore, the committee noted that ‘Some potential cost savings associated with caplacizumab may not be included in the company’s model’ as ‘The company stated that, based on its observations from the compassionate use scheme for caplacizumab, in NHS clinical practice, people would have it for a shorter duration than in the trials. The committee in general prefers not to disassociate estimates of cost and effectiveness from a trial. However, it appreciated that many assumptions about caplacizumab’s effectiveness in this model were not taken from the main trial. It also thought that some potential cost savings associated with caplacizumab may not have been included in the company’s model.’ Despite the remaining uncertainty, ‘(…) the assumptions in the economic modelling are plausible. Also, there are potential benefits with caplacizumab that are not included in the cost-effectiveness estimates. Overall, the estimates are within the range normally considered a costeffective use of NHS resources. So, caplacizumab is recommended for treating acute acquired TTP.’ TA588 Nusinersen for treating spinal muscular atrophy In 2018-2019, NICE assessed the cost-effectiveness of the antisense oligonucleotide nusinersen, used in the treatment of spinal muscular atrophy. Nusinersen promotes the formation of the functional SMN protein, through modulation of intron splicing, essential for normal function of motor neurons. Sanofi was the submitting Company and the Peninsula Technology Assessment Group (PenTAG) produced the evidence review group (ERG) report. NICE initially did not recommend the use of nusinersen for treating SMA as it was not deemed a cost-effective use of NHS resources. NICE consulted with the public and professionals and noted that ‘Following consultation, the committee heard that there was real-world evidence that would be relevant for the committee’s decision making that had not been considered by the company.’ Although the Company briefly touches upon data from the early access programme (EAP) in UK and Ireland (63 patients, of which 25 males and 38 females) and additionally points at the publication of a second European EAP conducted in other European countries (N=36, Gargaun et al.), the Spinal Muscular Atrophy Support UK and The SMA Trust points to several other studies in the consultation period: ‘We note that the real-world studies only review outcomes for children with SMA Type 1 for the first six months of treatment but consider ‘real world’ evidence critical to decision making. They all assist with confirming the certainty of evidence of effectiveness (see below). In particular we refer to: Reviews of the Expanded Access Programme: • Europe - 33 children aged from 8.3 to 113.1 months - December 2016 - May 2017. AragonGawinska, K et al. (2018) • Australia – 16 patients aged 2.5 months to 35.7 years November 2016 – September 2017 Farrar, M et al. (2018)
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