“Compassionate use”- COVID-19

Compassionate care or compassionate use of a drug represents the possibility of using drugs or therapies for therapeutic purposes, every time that the clinical trial phase has not yet been completed and which therefore do not have a specific indication in the pathology in which they would be employed.

The use of “compassionate use” for a medicinal product undergoing clinical trial, outside the trial itself, is foreseen in patients suffering from serious or rare diseases or from disease conditions endangering the patient’s life and when, according to medical opinion, there are no other valid therapeutic alternatives, or in the event that the patient doesn’t meet the necessary requirements to be included in a clinical trial.

According to the current legislation for tumors and rare diseases, it is possible to use medicines whose effectiveness has been evaluated throughout the experimental trials pertaining to Phase I, wherever safety records may be provided.

The claim must be based on the expected benefit based on the mechanism of action and the pharmacodynamics of the drug.

The draft protocol requires the approval of the Ethics Committee related to the clinical center submitting the request, subject to confirmation of the availability of free supply by the pharmaceutical manufacturing company

On the occasion of the circular on simplified procedures for studies and compassionate uses for the Covid-19 emergency, AIFA (The Italian Drugs Agency, translator’s note) reported the following definitions with reference to compassionate use:

• Program for therapeutic use: presented by the pharmaceutical industry for the use of medicines in the context of compassionate use to multiple patients, on the basis of a predefined and identical clinical protocol for all patients.
• Nominal therapeutic use: use of medicines in the context of compassionate use on a nominal basis for individual patients, based on scientific evidence and not in the context of a defined clinical protocol.

Currently, the Italian Drugs Agency has authorized five compassionate use programs for COVID-19 including that with the therapeutic molecule Solnatide developed by APEPTICO for the treatment of various life-threatening forms of acute pulmonary dysfunction and pulmonary edema in patients with ARDS.

Previously, Solnatide had been evaluated for safety and tolerability in a Phase I clinical study in healthy subjects and for preliminary efficacy studies in two Phase II studies in mechanically ventilated patients with acute respiratory distress syndrome (ARDS) and in patients with primary graft dysfunction (PGD) after lung transplantation.