OBJECTIVES: The aim was to explore the potential cost-effectiveness of cell-therapy adjuvant to surgery, and to assess the potential gains that could be achieved by cell-therapy to suggest its maximum acceptable price (MAP).

METHODS: The analysis took a National Health-Service perspective, used lifetime horizon and had a cycle length of one-year. The model was developed through semi-structured interviews of paediatric surgeons and gastroenterologists and an examination of treatment guidelines. The model has six health states and four transition-matrices (covering different time periods post-surgery). Scenarios examined the impact of therapy being 100%, 75%, 50%, and 25% curative. MAP was identified by estimating incremental net monetary benefit (INMB) of treatment at zero price.

RESULTS: When cell-therapy has a price of zero, the 100% curative scenario produced 23.03 quality adjusted life-years (QALYs) and a cost of £8,242 per patient. Surgery (Standard of care) showed the lowest QALYs and the highest cost with 21.99 and £21,929.27, respectively. INMB for the 100% curative scenario was £34,343 at a threshold of £20,000, which implies a MAP of that value. MAPs at 75%, 50% and 25% effectiveness were £33,365,£31,733, and £26,601, respectively.

CONCLUSIONS: The modelling illustrates that cell-therapy has the potential to be cost-effective at a price of over £25,000 even if only 25% effective. In addition to producing effectiveness data, the modelling highlighted that important quality of life and natural history data were lacking and need to be the focus of future research.