Compassionate use of cell products

Investigational medicinal products are available to patients through two pathways. The first is by participating in a clinical trial, the second as part of a compassionate use program, which is the use of an investigational medicinal product outside a clinical trial in patients with serious or life-threatening diseases for which no other treatment is available or expected to be effective. Usually, the medicinal product is an unauthorized drug, but it could also be a cell product. Some countries have specific regulations for compassionate use of medicinal products. In the USA, Food and Drug Administration (FDA) regulations allow access to investigational medicinal products for an individual patient, and for intermediate and large-size groups of patients who otherwise do not qualify to participate in a clinical trial. In Europe, each country has its own regulations. Despite this, the European Medicines Agency adopted a guideline on this issue to facilitate access by patients to experimental treatment. Regulation (EC) No 726/2004 of the European Parliament and of the Council states that " …a common approach should be followed, whenever possible, regarding the criteria and conditions for the compassionate use of new medicinal products under Member States (MS) legislation. " In Brazil there are no such regulations. An interesting discussion on this issue took place some years ago as a round table sponsored by the Canadian Parliament. After an introductory explanation, the rights of catastrophically-ill patients were discussed. The work of Dixon was cited. He stated that " a catastrophically-ill patient has the right to be free from any paternalistic interference in electing, in consultation with his physician, any therapy whatsoever that does not cause direct harm to others " (1,2) , which means that the seriously-ill individual has the right to take measures to save his own life. This should be understood as an aspect of individual right, so it cannot be forbidden. In the same document, the statement of JS Mill was cited " …the only purpose for which power can be rightfully exercised over any member of a civilized community, against his will, is to prevent harm to others " (3). However, things are more complicated as the right to be treated means that someone else has the corresponding duty to treat or to provide the medicinal product to the patient. The right to be treated is considered a " positive right " , which is the right to be subjected to …