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Special Supplies

What is 'Expanded Access Program' ?

Some manufacturers may run “expanded access programs”. A company may choose to run an expanded access program to allow early access to their medicine, for example, for patients who have been treated with the medication during a clinical trial and wish to continue treatment. In an expanded access program, patients are usually followed up in the same way as patients in a clinical trial.

"Sometimes called “compassionate use”, expanded access is a potential pathway for a patient with an immediately life-threatening condition or serious disease or condition to gain access to an investigational medical product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.

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.

Investigational drugs, biologics or medical devices have not yet been approved or cleared by FDA and FDA has not found these products to be safe and effective for their specific use. Furthermore, the investigational medical product may, or may not, be effective in the treatment of the condition, and use of the product may cause unexpected serious side effects."

What is Emergency Use Authorization (EUA)

General recommendations and procedures applicable to the authorization of the emergency use of certain medical products, to sustain and strengthen national preparedness for public health, military, and domestic emergencies involving chemical, biological, radiological, and nuclear (CBRN) agents, including emerging infectious disease threats such as pandemic influenza, or other pandemics.

What is 'Named Patient Basis' Supply?

In some cases medical fraternity or even the regulatory agency may approach a manufacturer directly to request the supply of a new medicine that does not have a product license, to be used for a patient under their direct responsibility.

Named Patient Basis is different from Compassionate use.

What is Compassionate Use of medicinal product?

Compassionate use is a treatment option that allows the use of unauthorised medicine. Under strict conditions, products in development can be made available to groups of patients who have a disease with no satisfactory authorised therapies and who cannot enter clinical trials. In the European Union, these programmes are only put in place if the medicine is expected to help patients with life-threatening, long-lasting or seriously debilitating illnesses, which cannot be treated satisfactorily with any currently authorised medicine.