Clinical ResearchRegulatory

Navigating the Regulatory Landscape for Expanded Access Programs

Navigating the Regulatory Landscape for Expanded Access Programs


Expanded access programs (EAPs) provide pathways to treatment options prior to local marketing authorization in cases where traditional clinical trial participation is not feasible. Usually, these treatments involve investigational therapies (i.e., drugs, biologics, and medical devices) that have not yet been approved by the relevant regulatory bodies, such as the Drug Administration of Vietnam (DAV); the Ministry of Food and Drug Safety (MFDS) of South Korea; The National Agency for the Safety of Medicines and Health Products (ANSM) of France; or the MoH (MPPS) division of drugs, medicines, and cosmetics from Venezuela. 

With the pandemic over and a drive for international collaboration and equity on the rise, the expansion of these access programs has been a focus for many in the scientific research community. And with this, the regulatory landscape has been adapting accordingly. On a global scale, regulations and ethical considerations vary, with different bodies mandating specific conditions under which EAPs can be permitted. 

For example, the approval of an EAP may be dependent on the patient fulfilling one or more of the following criteria:

  • The patient is suffering from a serious or life-threatening condition;
  • There is a lack of alternative, satisfactory treatment;
  • The patient is unable to participate in a clinical trial;
  • The benefits of the therapeutic regimen outweigh the risks; and/or
  • The treatment does not interfere with the ongoing drug development process.

Regulatory Considerations

The global regulatory landscape for EAPs is continuing to evolve. In recent years, several countries, including France, Lithuania, Kenya, and Ukraine, have introduced/updated/clarified regulations to facilitate access to unapproved treatments for patients with serious illnesses. Additionally, some countries, such as the United Kingdom, have introduced legislation to protect patients who participate in EAPs from legal action if the product causes harm.

Overall, the regulatory landscape for EAPs is changing rapidly. Drug manufacturers are now able to provide access to their products to patients with serious illnesses while still meeting regulatory requirements. 

In 2020, France implemented a new regulation that allows EAPs to provide access to unapproved drugs to patients with life-threatening or seriously debilitating conditions. This regulation was driven by real-world data showing that EAPs could provide access to potentially life-saving treatments for patients who do not qualify for clinical trials or have exhausted all other treatment options. The regulation requires that EAPs be approved by the French National Agency for the Safety of Medicines and Health Products (ANSM) and that they be conducted in accordance with the principles of Good Clinical Practice. In addition, the regulation requires EAPs to be conducted following the principles of transparency, patient safety, and ethical considerations.

Country-specific regulatory frameworks for EAPs are unified to ensure patients have access to critical medications when alternatives are not an option. Under this common goal, healthcare providers and regulators are implored to collaborate and communicate effectively to ensure mutual understanding of the requirements, risks, and operational considerations before providing an exception for critical patient use. However, they may differ on the regulatory submission process to the MoH, monitoring or reporting requirements, labeling, the number of patients allowed before different requirements are instilled, or the distribution and duration of providing the treatment commitments.

The pathway for obtaining authorization for this type of program can be complex with several variables to consider. Complexities can be further compounded when clinicians or providers are not familiar with the process or in some countries where the patient must apply directly for access.

These examples are non-exhaustive, as all parties must continue to comply with diverse country requirements for developing EAPs, which must, in turn, comply with new batch imports and renewed regulatory submissions. It is up to the drug manufacturer/future marketing authorization holder to approve or decline such requests when initiated by prescribers, as they deem appropriate.

Driving Compliance in Early Access to Medicines for Critically Ill Patients

In the US, the 21st Century Cures Act requires that a company developing investigational drugs (including biologics) shall make its policy regarding evaluating and responding to requests for expanded access public and readily available. This should be considered as the first step for any drug developer to define their approach to such requests. 

Ensuring compliance with all local regulatory requirements is an overarching, critical aspect of the expanded access pathways. Providers and drug manufacturers require up-to-date knowledge and understanding of the current local regulatory requirements wherever their patients are located around the globe. They should also ensure the provision of the necessary paperwork and forms, such as Form FDA 3926 in the US or any other local forms, informed consent, treatment protocols to the Institutional Review Board (IRB), safety reporting for adverse event monitoring, and logistics for global distribution and provision of the drug to the patient, factoring in specific importation and dispensation constraints that may exist.

Furthermore, ensuring compliance with complex and evolving regulations within the EAP pathway can be complemented by automating and validating the regulations. This simplifies the process for healthcare providers, pharmaceutical companies, and regulators when managing necessary documentation and reporting. 


A successful EAP application hinges on a collaborative effort among healthcare providers, pharmaceutical companies, patients, and regulators. Despite variations in regulatory frameworks globally, the goal across the board is consistent—ensuring timely access to essential treatment options for patients with severe illness, following the exhaustion of all other options. Despite the challenges associated with early patient access to medicines, TransPerfect can mitigate bottlenecks in country regulatory intelligence monitoring to confirm EAP regulations. 

Demystifying regulatory complexities and unburdening your teams can be seamless. Connect with us today to learn how TransPerfect Life Sciences' comprehensive suite of regulatory solutions provides expert insights and powerful automation tools designed to streamline approval processes for regulatory pathways.