Expanded human allogenic mesenchymal adult stem cells extracted from adipose tissue Medicine 09 January 2018 Extended indication Treatment of complex perianal fistula(s) in adult patients with non-active / mildly active luminal Crohn’s disease, when fistula(s) have shown an inadequate response to at least one conventional or biologic therapy. Therapeutic value Possible added value Total cost Registration phase Unknown Product Active substance Expanded human allogenic mesenchymal adult stem cells extracted from adipose tissue Domain Chronic immune diseases Reason of inclusion Main indication Bowel diseases Extended indication Treatment of complex perianal fistula(s) in adult patients with non-active / mildly active luminal Crohn’s disease, when fistula(s) have shown an inadequate response to at least one conventional or biologic therapy. Current proprietary name Already available biosimilars / generics Proprietary name Alofisel / Cx601 Manufacturer Takeda Portfolio holder Mechanism of action Unknown Route of administration Intralesional Therapeutical formulation Injection Budgetting framework Intermural (MSZ) Centre of expertise Additional remarks Betreft stamcelpreparaat. Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date April 2016 Expected Registration February 2018 Orphan drug Yes Registration phase Unknown Reimbursement Medicine sluice Additional remarks Positieve CHMP opinie december 2017. Advanced therapy medicinal product. Therapeutic value Current treatment options Fabrikant: Chirurgie, medische ingrepen en/of anti-TNF Therapeutic value Possible added value This assessment does not indicate any potential inclusion in the package. Substantiation Als anti-TNF niet helpt dan kan dit een belangrijke bijdrage leveren en voor meer QOL zorgen. Duration of treatment Frequency of administration 1 times a day Dosage per administration Fabrikant: 1 toediening van 120 miljoen cellen References Fabrikant; Panés J, et al. Lancet. 2016;388:1281-90 Additional remarks Dosering zoals in de ADMIRE-CD studie. Expected patient volume per year Patient volume Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References Additional remarks Inschatting varieert: Beperkt aantal patienten, alleen als het invaliderend is? Of juist veel (39% van alle ZvC patienten heeft last van fistels). Fabrikant: Onbekend, reeds communicatie met VWS hierover. Expected cost per patient per year Cost € 20,000.00 - 40,000.00 This amount gives an indication of the total cost. It is the result of the average expected patient volume times the average cost per patient. both per year. References Additional remarks De verwachting is dat het duur zal zijn, eenmalige toediening. Potential total cost per year Total cost Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Unknown Indication extensions References Additional remarks Other information Additional remarks