Human normal immunoglobulin Medicine 08 December 2020 Extended indication Replacement therapy in adults, children and adolescents (0‑18 years) in: Secondary immunodeficiencies (SID) in patients who suffer from severe or recurrent infections, ineffective antimicrobial treatment and either proven specific antibody failure (PSAF) or serum IgG level of <4 g/l. Therapeutic value No estimate possible yet Total cost € 2,250,000.00 Registration phase Registered and reimbursed Product Active substance Human normal immunoglobulin Domain Infectious diseases Reason of inclusion Indication extension IND Main indication Infectious diseases other Extended indication Replacement therapy in adults, children and adolescents (0‑18 years) in: Secondary immunodeficiencies (SID) in patients who suffer from severe or recurrent infections, ineffective antimicrobial treatment and either proven specific antibody failure (PSAF) or serum IgG level of <4 g/l. Current proprietary name Already available biosimilars / generics Proprietary name HyQvia Manufacturer Takeda Portfolio holder Mechanism of action Immunostimulation Route of administration Subcutaneous Therapeutical formulation Injection Budgetting framework Extramural (GVS) Centre of expertise Additional remarks Registration Registration route Centralised (EMA) Type of trajectory Normal trajectory Particularity Unknown ATMP Unknown Submission date March 2020 Expected Registration September 2020 Orphan drug No Registration phase Registered and reimbursed Reimbursement Medicine sluice Additional remarks Positieve CHMP-opinie in juli 2020. Therapeutic value Current treatment options Therapeutic value No estimate possible yet This assessment does not indicate any potential inclusion in the package. Substantiation De verwachting is dat dit geneesmiddel een meerwaarde zal hebben voor een kleine groep patiënten. Duration of treatment Frequency of administration Dosage per administration References NCT03054181 Additional remarks Zie de huidige behandeling volledig uitgeschreven in het Farmacotherapeutisch Kompas. https://www.farmacotherapeutischkompas.nl/bladeren/preparaatteksten/n/normaal_immunoglobuline__im_en_sc_ Expected patient volume per year Patient volume < 100 Market share is generally not included unless otherwise stated. Maximum patient volume for sluice References GIP databank Additional remarks Meer dan 1.000 gebruikers per jaar van normaal immunoglobuline subcutaan en meer dan 1.600 gebruikers normaal immunoglobuline IV. Met de aanpassing van de indicatie zal mogelijk een deel van de patiënten die nu intraveneus behandeld wordt subcutaan worden behandeld. De inschatting is dat er maximaal 100 patiënten in aanmerking zullen komen voor deze nieuwe toedieningsvorm. Expected cost per patient per year Cost € 20,000.00 - 25,000.00 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 GIP databank Additional remarks Kosten in 2019 zo'n €20.000-€25.000 per patiënt per jaar. Geen Budget impact, daar deze patiënten nu al met intraveneuze immuunglobulinen worden behandeld. Potential total cost per year Total cost € 2,250,000.00 Total cost for sluice Additional remarks Off label use Off label use Unknown Indications off label use References Additional remarks Indication extension Indication extensions Yes Indication extensions Chronic inflammatory demyelinating polyradiculoneuropathy References fase 3 studie Adis Insight Additional remarks Other information Additional remarks