EXPANDED ACCESS OF AN
INVESTIGATIONAL PRODUCT

Expanded Across Program

The U.S. Food and Drug Administration (FDA) Expanded Access (EA) program (often referred to as compassionate use) was initiated to provide a regulatory framework for patients with serious or life-threatening diseases or conditions to safely access investigational medical products outside of a clinical trial when no comparable or satisfactory alternative treatment exists. The EA program was first established and codified into regulations for drugs and biologics in 1987 and for devices in 1996. Through the EA program, tens of thousands of patients with serious or life-threatening conditions, who have exhausted all or have no alternative treatment options, have been able to safely access potentially beneficial, investigational therapies. The EA program continues to evolve and refine itself based on stakeholder feedback. The EA program has simplified documentation requirements and streamlined Institutional Review Board (IRB) requirements, thus aligning itself to be more patient-centered, efficient, flexible, and responsive. (1,2)

21st Century Cures Act

The 21st Century Cures Act, signed into law in 2016, requires manufacturers of investigational drugs and biologics (and implicitly, devices) to make their EA policies readily available on a public website. Manufacturers fulfill this requirement by posting their EA policy directly on their website or through the Reagan-Udall Foundation’s Expanded Access Navigator (https://navigator.reaganudall.org/). Manufacturers are not required to offer or participate in an EA program, but publicly posting policies promote transparency, consistency, realistic expectations, and make it easier for patients and clinicians to explore emerging treatment options. (3,4)

Right-to-Try

In 2018, the federal Right To Try Act went into effect in the U.S. creating an additional, more permissive route for terminally ill patients to gain access to investigational drugs outside clinical trials. (5) Existing in parallel to the EA program, the Right To Try legislation allows individuals who meet the strict eligibility criteria to appeal directly to the manufacturer of an investigational drug or device, which must have completed phase 1 clinical trials. The right-to-try pathway is not overseen by the FDA nor does it require IRB approval, but some regulations must still be followed. The manufacturer must report the number of doses provided, the indication, and any serious adverse events observed to the FDA through an Investigational New Drug (IND) annual report. Similar approaches and methods are permissible for the use of medical devices, including those under Investigational Device Exemptions (IDEs). (6)

Not without controversy, the manufacturer may stand at odds with agreeing to an individual’s request due to concerns over the risk-benefit profile, limited efficacy and safety data, restricted product supply or high production costs, potential impacts to ongoing clinical trials and continued product development, and liability for harms. To reduce the manufacturer’s perceived risk, terminally ill patients often agree to waive harm, or even death, resulting from experimental treatment and acknowledge there are no guarantees that experimental treatment would be beneficial or extend their life. Under right-to-try, manufacturers are permitted to charge patients for “direct costs” of making the investigational product available to offset some of the financial burden. (7,8,9)

Yet, even when a manufacturer agrees to provide access, the right-to-try pathway remains highly debated. Because FDA and IRB oversight are intentionally removed under the Right to Try Act, no independent body is verifying that patients are appropriately selected, adequately informed, and monitored under the safeguards that ordinarily guide ethical and safe use of investigational products. This gap in oversight creates uncertainty about who, manufacturer or treating physician, assumes responsibility for these essential protections, leaving patients potentially exposed to additional and unforeseen risks. (7,8,9)

Right to try is widely regarded as “Right to Ask” rather than a guaranteed pathway. (7) The Right To Try Act is limited not by statute, but by manufacturer discretion, liability concerns, financial barriers, and debate over appropriate oversight. As such, the FDA EA program remains the primary mechanism for patients with serious or life-threatening conditions to access investigational medical products outside clinical trials. (2,10)

EA PROGRAM CRITERIA

FOR A PATIENT TO QUALIFY FOR EXPANDED ACCESS, A LICENSED PHYSICIAN MUST DETERMINE THAT:

ADDITIONALLY, FOR THE FDA TO CONSIDER APPROVAL:

*Note: The manufacturer may decide to charge for the development of its medical product per individual case which is likely to be passed on directly to the patient.

EXPANDED ACCESS FOR DRUGS, BIOLOGICS, AND DEVICES

If patient(s) meet the criteria, EA requests for drugs, biologics, and devices are submitted to the FDA either by a licensed treating physician or by the investigational medical product’s manufacturer (Sponsor). While the EA frameworks for drugs/biologics and devices are slightly different, they are structured similarly with three distinct pathways designed to match the level of need (Emergency use or Non-emergency Use) and the number of patients requiring access to the investigational medical product. (11,12)

SUMMARY

Many patients fall through cracks in our healthcare system and have nowhere to turn for help. The regulatory hurdles and nuances to supporting such cases may be overwhelming to both Sponsors and physicians.

CRS is a proven expert partner to ensure the regulations are being met, and the safety and confidentiality of the patient is maintained. Urgent support including weekends and holidays is available for Emergency Use cases. Contact us for more information about ways we can help your organization, medical practice, or an individual patient.

References

1. FDA. (2025, September 08). Expanded Access. U.S. Food & Drug Administration. https://www.fda.gov/news-events/public-health-focus/expanded-access
2. FDA. (2024, May 02). Expanded Access (Compassionate Use) Submission Data. U.S. Food & Drug Administration. https://www.fda.gov/news-events/expanded-access/expanded-access-compassionate-use-submission-data
3. United States Congress. (2015-2016). 21st Century Cures Act, H.R. 34, 114th Cong. https://www.congress.gov/bill/114th-congress/house-bill/34
4. FDA. (2020, January 31). 21st Century Cures Act. U.S. Food & Drug Administration.  https://www.fda.gov/regulatory-information/selected-amendments-fdc-act/21st-century-cures-act
5. United States Congress. (2018). Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017, S. 204, 115th Cong. https://www.congress.gov/bill/115th-congress/senate-bill/204
6. FDA. (2024, December 12). Right to Try. U.S. Food & Drug Administration. https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try
7. Hudis, C. (2018, July 09). Right-to-Try: A Conversation With Dr. Richard L. Schilsky. ASCO Connection. https://connection.asco.org/do/right-try-conversation-dr-richard-l-schilsky
8. Shoemaker, D., Barber, K., and Whitney, K. (2019, August 05) Right-to-try or right to ask? Rho. https://www.clinicalleader.com/doc/right-to-try-or-right-to-ask-understanding-right-to-try-and-fda-s-expanded-access-programs-0001
9. Miseta, E. (2018, November 29). Now That Right To Try Is Law, What Does It Mean To You? Clinical Leader. https://www.clinicalleader.com/doc/now-that-right-to-try-is-law-what-does-it-mean-to-you-0001
10. FDA. (2025, July 09). Right to Try Annual Reporting Summary. U.S. Food & Drug Administration. https://www.fda.gov/patients/learn-about-expanded-access-and-other-treatment-options/right-try-annual-reporting-summary
11. FDA. (2018, January 04). Expanded Access Categories for Drugs (Including Biologics). U.S. Food & Drug Administration. https://www.fda.gov/news-events/expanded-access/expanded-access-categories-drugs-including-biologics
12. FDA. (2019, June 21). Expanded Access for Medical Devices. U.S. Food & Drug Administration. https://www.fda.gov/medical-devices/investigational-device-exemption-ide/expanded-access-medical-devices#compassionate