PROTALIX BIOTHERAPEUTICS, INC. (PLX)

[Kronberg]

FDA
Expanded access (sometimes called “compassionate use”) is the use of investigational drugs, biologics or medical devices outside the clinical trial setting for treatment purposes. Expanded access may be appropriate when all the following apply:

Patient has a serious disease or condition, or whose life is immediately threatened by their disease or condition.

There is no comparable or satisfactory alternative therapy to diagnose, monitor, or treat the disease or condition.

Patient enrollment in a clinical trial is not possible.

Potential patient benefit justifies the potential risks of treatment.

Providing the investigational medical product will not interfere with investigational trials that could support a medical product’s development or marketing approval for the treatment indication

https://www.fda.gov/NewsEvents/PublicHealthFocus/ExpandedAccessCompassionateUse/default.htm

EMA

Questions and answers on the compassionate use of
medicines in the European Union
This document describes how ‘compassionate use’ programmes may be set
up in the European Union, and the role of the European Medicines Agency in
these activities, in accordance with Article 83 of Regulation (EC) No
726/2004

What is compassionate use?

Compassionate use is a way of making available to patients with an unmet medical need a promising
medicine which has not yet been authorised (licensed) for their condition.
A medicine can be marketed in the European Union (EU) only after it has been authorised. However, it
is sometimes in the interest of patients to have access to medicines before authorisation. Special
programmes can be set up to make these medicines available to them under defined conditions. This is
known as ‘compassionate use’.

Which medicines can be made available in this way?

Compassionate use programmes can only be put in place for medicines that are expected to help
patients with life-threatening, long-lasting or seriously disabling illnesses. These programmes are
expected to benefit seriously ill patients who currently cannot be treated satisfactorily with authorised
medicines, or who have a disease for which no medicine has yet been authorised. The compassionate
use route may be a way for patients who cannot enrol in an ongoing clinical trial to obtain treatment
with a potentially life-saving medicine.
At this stage in the development of the medicine, what is known of the medicine’s safety may be
limited. Generally, toxicology studies will have been completed and analysed, and early studies looking
at how the medicine is handled by the body will have been completed. However, there may still be
some uncertainties about the best way to give the medicine to patients, such as the exact dose to use,
and the dose frequency, and the medicine’s safety profile (which side effects it can cause) is not yet
fully established.

http://www.ema.europa.eu/docs/en_GB/document_library/Other/2010/01/WC500069898.pdf