Neratinib

Extended indication	Abecma is indicated for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.
Therapeutic value	Possible added value
Total cost	16,250,000.00
Registration phase	Registered

Product

Active substance	Idecabtagene vicleucel
Domain	Hematology
Reason of inclusion	New medicine (specialité)
Main indication	Multiple Myeloma
Extended indication	Abecma is indicated for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.
Proprietary name	Abecma
Manufacturer	BMS
Mechanism of action	CAR-T therapy
Route of administration	Intravenous
Therapeutical formulation	Intravenous drip
Budgetting framework	Intermural (MSZ)
Centre of expertise	AMC locatie VU

Registration

Registration route	Centralised (EMA)
Type of trajectory	Accelerated assessment
Particularity	New medicine with Priority Medicines (PRIME)
ATMP	Yes
Submission date	April 2020
Expected Registration	August 2021
Orphan drug	Yes
Registration phase	Registered
Additional remarks	Positieve opinie van de CHMP in juni 2021. Dit middel is voor deze indicatie in de sluis geplaatst.

Therapeutic value

Current treatment options	Chemotherapie combinaties
Therapeutic value	Possible added value
Substantiation	Gebruik van ide-cel in de "real world" is vergeleken met standard of care in een populatie van patiënten met een vergelijkbaar profiel (Jagannath et al, JCO mei 2020). Hier wordt een significant betere overall response rate (76 versus 32%) en progressie vrije overleving (11.3 versus 3.5 maanden) gezien.
Duration of treatment	one-off
Dosage per administration	150 - 450 x 10^6 CAR+ T cells
References	NCT03361748

Expected patient volume per year

Patient volume	< 50 Market share is generally not included unless otherwise stated.
Additional remarks	Gezien de vele behandelopties komen naar schatting maximaal 50 patiënten per jaar hiervoor in aanmerking. Naar de toekomst toe kan CAR-T mogelijk naar eerdere behandelrichtlijnen verschuiven, in dat geval zouden meer patiënten in aanmerking komen.

Patient volume

< 50

Market share is generally not included unless otherwise stated.

Additional remarks

Gezien de vele behandelopties komen naar schatting maximaal 50 patiënten per jaar hiervoor in aanmerking. Naar de toekomst toe kan CAR-T mogelijk naar eerdere behandelrichtlijnen verschuiven, in dat geval zouden meer patiënten in aanmerking komen.

Expected cost per patient per year

Cost	320,000.00 - 330,000.00
Additional remarks	De kosten mogelijk vergelijkbaar met andere CAR-T therapieën tisagenlecleucel en axicabtagene ciloleucel (€320.000-€330.000). Dit middel is voor deze indicatie in de sluis geplaatst tot er een financieel arrangement overeengekomen is.

Potential total cost per year

Total cost	16,250,000.00 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.

Total cost

16,250,000.00

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.

Off label use

There is currently nothing known about off label use.

Indication extension

Indication extension	No
References	AdisInsight.

Other information

There is currently no futher information available.

Idecabtagene vicleucel