Despite progress of pharmacological research in the paediatric field, the problem of the availability of medicines for children has still not been solved and it is more evident in newborns, because some issues related to clinical trials are even more highlighted.
The consequence is the use of extemporaneous galenic preparations and off-label medicines, i.e. drugs used for an indication different from the authorized one.
However, the use for a different therapeutic indication, age, formulation, route administration, posology (dosage and frequency), exposes children to major risks, because it is easier to fall into therapeutic mistakes or in unexpected adverse events.
Moreover, if it is not possible to intervene with targeted medicines, it seems crucial to use alternative solutions, as long as they are scientifically proven and patients’ parents are well informed on the therapy.
In some cases, in Italy the free access to pharmacological therapy before AIFA authorizes it or the off-label use of already authorized medicines are granted.
The ways for early access to a medicine are:
- Law 648/1996
- Compassionate use
- AIFA National Fund (Law 326/2003 – “fondo 5%”)
- non-repetitive use of advanced therapies
The choice of the most suitable way depends on the specific indication.
Furthermore, it is possible to access treatment with a medicine available on the market, but for a different indication from the one it was authorized for (Law 94/98 art.3, subsection 2 – ex Di Bella law), even when there are alternative treatments regularly authorized. Considering all this, this off label use should be known and compliant with works appeared on internationally accredited scientific publications.
In this case, the patient or the hospital, in case of hospitalization, are in charge of the therapy. All these early access ways are under the responsibility of a doctor.
Ensuring the access to medicines to children is important, but ensuring access to efficient and safe medicines is even more important!
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