Expanded access, also known as compassionate use, is a program that allows patients with serious or life-threatening conditions to gain access to investigational drugs, biologics, and medical devices outside of clinical trials. For patients facing serious or life-threatening conditions with no viable treatment options, gaining access to investigational medical products before full FDA approval can be a critical opportunity.

This pathway, known as Expanded Access (EA)—sometimes referred to as Compassionate Use—allows eligible patients to receive investigational treatments when clinical trials are not an option. For companies developing these products, understanding EA’s regulatory requirements, benefits, and risks is essential. Here’s what you need to know.

What Is Expanded Access (EA)?

FDA defines EA as a potential pathway for a patient with a serious or immediately life-threatening 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.

This can be thought of as providing a patient with emergency or last-resort access to a treatment that has not yet been fully approved for marketing in the U.S. by FDA. In this unique situation, FDA works with healthcare providers and drug manufacturers to determine whether expanded access is appropriate. It’s not a substitute for participation in clinical trials, but rather an option when clinical trials are not available or suitable. Ensuring drug safety and effectiveness through clinical trials is crucial for ongoing drug and product development efforts.

Expanded Access Programs enable the distribution of investigational drugs or products to patients without available treatment options outside of clinical trials. These programs provide access to potentially life-saving treatments for individuals facing serious or life-threatening conditions.

How Does Expanded Access Differ from Compassionate Use?

Although the terms Expanded Access and Compassionate Use have been used interchangeably, FDA now prefers the term Expanded Access (EA). The concept remains the same: providing investigational treatments to patients who lack other options.

Does My Product Qualify for Expanded Access?

From FDA’s standpoint, a product can be considered for expanded access (EA), if it meets all of the following conditions:

  • The disease or condition is serious or immediately life-threatening.
  • No comparable or satisfactory alternative FDA-approved therapy exists.
  • The patient is unable to enroll in a clinical trial.
  • The potential benefits outweigh the potential risks of treatment.
  • Providing the product for EA use will not interfere with the medical product’s development, clinical trials supporting regulatory approval, or marketing approval for the treatment indication.

Understanding FDA’s Expanded Access Process in the U.S.

If a company wishes to provide investigational products under EA, they must submit an EA application to FDA for which must be approved before treatment can begin. 

Expanded access is usually granted on an individual, case-by-case basis.  However, there are 3 different types of EA applications:

1. Individual Patient Expanded Access

  • For a single patient in need, often under emergency conditions.
  • The physician submits an Individual Patient IND (Investigational New Drug application) or Emergency IND request to FDA.

2. Intermediate-Size Population Expanded Access

  • For a group of patients with a similar condition who do not meet clinical trial criteria.
  • Requires submission of an Intermediate Access IND.

3. Treatment IND or Protocol

  • For larger patient populations, often in the final stages of clinical development.
  • Typically used when a product is in Phase 3 trials or under NDA (New Drug Application) or BLA (Biologics License Application) review.

What are the Pros and Cons of Providing Investigational Product Access to Patients Under EA?

Potential Benefits:

  • Allows potentially life-saving treatments to reach patients with limited treatment options.
  • Collecting real-world safety and efficacy data from patients who are treated under an approved EA IND that may support future regulatory submissions.
  • Helps physicians gain experience with the investigational product.
  • In some cases, companies may supplement some costs by charging for access to an investigational product.

Potential Challenges:

  • Safety and efficacy of the product are not fully established, increasing potential risks.
  • Companies must prepare and submit IND safety reports and annual reports to FDA. This data may be reviewed by FDA during NDA or BLA review and may impact regulatory decision-making.
  • The costs of manufacturing and providing the drug under EA may be prohibitive.
  • A company’s legal and regulatory liabilities should be carefully evaluated and understood prior to treating patients under EA.

As a Small Company, What Are a Company’s Obligations Regarding Expanded Access?

While companies are not required to provide investigational products under EA, they must comply with certain legal requirements. Under the 21st Century Cures Act, companies developing investigational drugs must:

  • Publicly post their Expanded Access policy on their website.
  • Include details on how they evaluate and respond to EA requests.
  • Provide this information on the Reagan-Udall Foundation’s Expanded Access Navigator or their own webpage.
  • Establish different EA policies for different investigational drugs, if necessary.
  • Follow FDA guidance on fulfilling these requirements.

Expert Guidance on Expanded Access and Regulatory Compliance

For small biopharmaceutical companies, EA can be both an opportunity and a challenge. Understanding its impact on product development, regulatory approvals, and company resources is critical. If your organization is considering an EA program, consulting regulatory experts can help navigate the complexities and ensure compliance.

For expert guidance on Expanded Access and regulatory compliance, contact Facet Life Sciences today.