Early access programs (EAP) are adopted by Pharmaceutical Companies because they offer ethical and controlled mechanisms of access to new drugs for patients without available treatment options, outside clinical trials and before being launched on the market.

These programs, also known as compassionate use, expanded access to the market, etc. are regulated by EMA with Article 83 of Regulation (EC) No 726/2004.

Although each state has its own National regulations, EMA recommendations play a guideline role, along with the program PRIME (PRIority MEdicines) that EMA launched in March 2017 to accelerate access to life-saving experimental drugs.

The analysis of the use of EAP in the Policlinico “Umberto I” of Rome shows how in recent years this instrument is becoming increasingly important.

Because the drugs introduced with the EAP are characterized by high cost and have a significant economic impact, they should be evaluated more in the definition of the annual budget.

For this purpose, the List of Compassionate Use Programs made available by AIFA is easy to consult, it is a useful tool for an overview of existing agreements and can offer valuable help in the management of innovative drugs.

 

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Key words: drug, clinical trial, HTA study, Early Access Program (EAP), budget impact