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Role of Hospital Pharmacist in the compassionate use of Risdiplam since the submission to Ethics Committee to the therapy in patients affected by Spinal Muscular Atrophy: an example of close collaboration with the neuromuscular team.

European Statement

Introductory Statements and Governance

Author(s)

Anna Esposito, Claudia Panico , Loretta Cervi, Emilio Albamonte, Alice Zanolini, Valeria Sansone

Why was it done?

This allows a standardized and safe access to this therapeutic opportunity in close collaboration with the NT formed by child neurologist and neurologists.

What was done?

The hospital pharmacist (HP) supports the neuromuscular team (NT) in submitting the request to access compassionate use with Risdiplam in patients affected by spinal muscular atrophy (SMA). Risdiplam is an orally administered, survival motor neuron 2-directed RNA splicing modifer for the treatment of SMA.1 The HP is involved in Ethics Committee (EC) approval, is responsible for reconstituting and storage of oral solution and is also responsible of re-supply and pharmacovigilance surveillance. This allows a standardized and safe access to this therapeutic opportunity in close collaboration with the NT formed by child neurologist and neurologists.

How was it done?

HP is involved from the initial phases of the compassionate use of the drug. The NT that screens and enrolls the patients in the compassionate use program with Risdiplam by submitting a formal request to the EC. The teams of the EC, which includes the HP, evaluates the appropriateness and inclusion criteria for each patient. Once the patient is considered eligible, and treatment is approved, the HP receives the drug from Pharma, provides quality control and assigns the drug for each patient. HP prepares the oral solution and stores the products at 2-8°C until dispensing. After the initial dispensation it is the HP’s responsibility to work closely with the NT in order to maintain treatment over time, ensuring the right timing for re-supply of the drugs and medical devices.

What has been achieved?

There are currently 30 patients on therapy and they are monitored to ensure re-supply and reporting of any adverse events. Home delivery procedure was implemented in agreement with EC in cases where outpatient dispensing was difficult, especially due to logistical difficulties caused by the Covid-19 pandemic, if clinical conditions were stable. In addition, a strong collaboration between the HP and NT was implemented in order to provide access to an increasing number of patients.

What next?

Risdiplam has recently been approved and the HP is involved in the procurement and maintenance of ongoing therapies and ensures supply to new patients. The drug is under negotiation and HP will be involved in contracting procedures based on the expected number of patients and definitive inclusion criteria to access the drug

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