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EXPERIENCE OF PATISIRAN OPTIMIZATION BUILDING HEALTHCARE TEAM

European Statement

Clinical Pharmacy Services

Author(s)

CRISTINA GONZALEZ PEREZ, LUCIA GALÁN DAVILA, MARTA ORTIZ PICA, ELENA GARCIA SUAREZ, MARIA MOLINERO MUÑOZ, LIDIA YBAÑEZ GARCIA, NATALIA SANCHE-OCAÑA MARTIN, MARIA DE LA TORRE ORTIZ, JAVIER CORAZON VILLANUEVA, JOSE MANUEL MARTINEZ SESMERO

Why was it done?

Our hospital is a reference in our community for this rare disease. Since patisiran was approved, we have treated 19 patients. Due to the high cost of patisiran and the few patients treated, it is necessary to optimize patient treatments in the most efficient way.

What was done?

Transthyretin (TTR) amyloidosis is a rare disease caused by mutations in the TTR gene. These mutations alter the normal function of TTR protein, creating slowly progressive condition characterized by the buildup of abnormal deposits of amyloid in body’s organs and tissues.
Patisiran is a TTR specific small interfering RNA (siRNA) formulation in lipid nanoparticles, which has been shown to substantially reduce the production of abnormal TTR in patients with hereditary amyloidosis TTR.
With this project we look for achieve the greatest possible savings from patisiran drug without affecting the patient’s efficacy.

How was it done?

We created a working group with neurology department, to define patient’s groups according to:
– Prescribed doses (doses per patient weight; 0.3 mg/kg).
– Preparation’s losses because of the filters
– Losses of the extraction process.
– Other individual conditions as posology or personal preferences.
Patients belonging to the same group have to administrate patisiran the same days.
As the patient’s weight can change along the time, we agree with doctors and nurses to weight the patients every six months to perform doses adjustments. Therefore, every six months we should regroup the patients to optimize the most.

What has been achieved?

These clusters have made possible to reduce wasting vials and therefore to permit significant savings. We estimate around 208.115 € savings/year.

What next?

With this practice we look for a more efficient and sustainable rational health system. The savings achieved can be used to treat other patients or promote new investigations. Also, it makes possible a better healthcare team performance, working together for a better attention, health quality, security, and treatments efficiency. Monitoring weights to adjust doses and closer follow-up of patients by the different members of the multidisciplinary team are examples of the achievements.

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