Table 1.

A comparison of Early Access Provision (EAP) programs for Europe and the USA.

	European Medicines Agency	US Food and Drug Administration
Definition Compassionate use	A treatment option for patients in the European Union suffering from a disease for which no satisfactory authorized alternative therapy exists or who cannot enter a clinical trial. These programs 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 authorized medicine.	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 medicinal product (drug, biologic, or medical device) for treatment outside of clinical trials when no comparable or satisfactory alternative therapy options are available.
Criteria to apply	• The medicinal product is to be made available to “patients with a chronically or seriously debilitating disease, or a life threatening disease, and who cannot be treated satisfactorily by an authorized medicinal product” in the European Union, • The compassionate use program is intended for a “group of patients,” • The medicinal product is either “the subject of an application for a centralized marketing authorization in accordance with Article 6 of Regulation (EC)# 726/2004 or is undergoing clinical trials” in the European Union or elsewhere.	• 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.
Vs clinical trials	• Clinical trials are practically the only means of obtaining reliable and interpretable efficacy and safety data for a medicinal product. • Although safety data may be collected during compassionate use programs, such programs cannot replace clinical trials for investigational purposes. • Compassionate use is not a substitute for properly conducted trials. • Compassionate use should therefore not slow down the implementation or continuation of clinical trials intended to provide essential information relative to the benefit/risk balance of a medicinal product. • Patients should always be considered for inclusion in clinical trials before being offered compassionate use programs.	Whenever possible, an investigational medical product should be used as part of a clinical trial
Vs off-label use	Product is authorized/available and it is the physician's decision to use it outside of official indications/label.
Vs patient support program (PSP)	PSP or disease management program is used to collect safety data of authorized products in post-approval/post-launch phase of product life cycle management.
Different role and responsibility	Compassionate use programs are intended to facilitate the availability to patients of new treatment options under development. National compassionate use programs, making medicinal products available either on a named patient basis or to cohorts of patients, are governed by individual Member States (MS) legislation.	Sponsor to report (individual and cohort) IRB review is mandatory
Different types	• Patient cohort (compassionate use). • Individual/single patient use is different and regulated by country	• (1) Individual patient use, the most common category, in which access is granted for a single person with a serious disease and no viable alternative option and may be for emergency or non-emergency use • (2) Limited use for an intermediate size patient population not exceeding 1,000 • (3) Widespread use for a larger treatment population on the basis of a successful clinical trial result, as the drug has not yet been approved for public access
Competent authorities involved in Compassionate use/ Expanded Access Programs (4–6)	European Medicines Agency (EMA) MHRA (UK) ANSM (France) PEI and BfArM (Germany) HPRA (Ireland) Austrian Medicines and Medical Devices Agency FAMHP (Belgium) Danish Medicines Agency (Denmark) AIFA (Italy) AEMPS (Spain) MPA (Sweden) MEB (Netherlands) JAZMP (Slovenia) NOMA (Norway) Ravimiamet (Estonia) Amt für Gesundheit (Liechtenstein)	US Food and Drug Administration (US FDA)