Pralsetinib

Extended indication	Gavreto is indicated for the treatment of adults and pediatric patients 12 years of age and older with locally advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy following prior treatment with Tyrosine kinase inhibitors and not previously treated with a RET inhibitor.
Therapeutic value	No estimate possible yet
Total cost	1,950,000.00
Registration phase	No registration expected

Product

Active substance	Pralsetinib
Domain	Oncology
Reason of inclusion	Indication extension
Main indication	Thyroid cancer
Extended indication	Gavreto is indicated for the treatment of adults and pediatric patients 12 years of age and older with locally advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy following prior treatment with Tyrosine kinase inhibitors and not previously treated with a RET inhibitor.
Proprietary name	Gavreto
Manufacturer	Roche
Portfolio holder	Roche
Mechanism of action	Tyrosine kinase inhibitor
Route of administration	Oral
Therapeutical formulation	Capsule
Budgetting framework	Intermural (MSZ)
Additional remarks	Selectieve remmer van RET-kinase.

Registration

Registration route	Centralised (EMA)
Type of trajectory	Normal trajectory
Submission date	December 2021
Orphan drug	No
Registration phase	No registration expected
Additional remarks	Roche heeft de registratie van deze indicatie van pralsetinib teruggetrokken.

Therapeutic value

Current treatment options	Cabozantinib/vandetanib in de toekomst mogelijk ook selpercatinib
Therapeutic value	No estimate possible yet
Substantiation	ARROW, een fase 1-2 studie, toonde een overall response rate van 71% (95% CI 48–89) aan in patiënten met treatment-naive RET-mutant medullary thyroid carcinoom en 60% (95% CI 46–73) in patiënten die eerder cabozantinib or vandetanib, of beide.
Frequency of administration	1 times a day
Dosage per administration	400mg
References	https://www.sciencedirect.com/science/article/pii/S2213858721001200?via%3Dihub

Expected patient volume per year

Patient volume	10 - 20 Market share is generally not included unless otherwise stated.
References	IKNL 2018(1); Expert opinie (2);
Additional remarks	Er zijn maximaal 100 volwassen patiënten met schildkliercarcinoom (exclusief medullair). Mogelijk zo'n 10%-20% van deze groep zal "RET fusion positive" schildkliercarcinoom hebben (1). Bij de niet-medullaire schildkliercarcinoom wordt niet getest en gezien er daarvoor standaardbehandelingen is de inschatting dat er toch minder patiënten in aanmerking komen (2).

Patient volume

10 - 20

Market share is generally not included unless otherwise stated.

References

IKNL 2018(1); Expert opinie (2);

Additional remarks

Er zijn maximaal 100 volwassen patiënten met schildkliercarcinoom (exclusief medullair). Mogelijk zo'n 10%-20% van deze groep zal "RET fusion positive" schildkliercarcinoom hebben (1). Bij de niet-medullaire schildkliercarcinoom wordt niet getest en gezien er daarvoor standaardbehandelingen is de inschatting dat er toch minder patiënten in aanmerking komen (2).

Expected cost per patient per year

Cost	< 130,000.00
References	G-standaard
Additional remarks	Prijs voor Nederland nog niet bekend maar gelijke kosten verwacht met selpercatinib. Dit middel is voor deze indicatie in de sluis geplaatst tot er een financieel arrangement is overeengekomen.

Potential total cost per year

Total cost	1,950,000.00 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

1,950,000.00

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

There is currently nothing known about indication extensions.

Other information

There is currently no futher information available.