Key Takeaways
- The U.S. Food and Drug Administration (FDA) has issued updated guidance on Expanded Access to Investigational Drugs for Treatment Use, clarifying how drug developers, physicians, and patients can obtain investigational drugs outside traditional clinical trials. The guidance underscores the FDA’s intent to balance compassionate use with the integrity of ongoing drug development, while outlining key procedural safeguards and compliance obligations.
- For drug developers, the updated guidance has significant implications for intellectual property (IP) management and related data protection strategies.
In October 2025, the FDA released a revised guidance titled, “Expanded Access to Investigational Drugs for Treatment Use—Questions and Answers.”1The document updates prior interpretations of 21 C.F.R. Part 312, Subpart I, providing detailed instructions on how and when patients may access investigational products to treat serious or life-threatening conditions when no satisfactory alternatives exist. The FDA reiterates that expanded access (also known as “compassionate use” and/or “preapproval access”) is intended primarily for treatment rather than data collection and remains subject to the same safety and oversight requirements as applicable to investigational new drugs.
The guidance confirms that three expanded access pathways are available:
- Individual patient use (including emergency use)
- Intermediate-size population programs
- Treatment Investigational New Drug applications (INDs)2or protocols for larger groups
Each request must demonstrate that 1) there are no comparable or satisfactory alternative therapies; 2) the potential benefit justifies the potential risk; 3) the use will not interfere with ongoing clinical development; and 4) the patient does not qualify for any ongoing clinical trials. In addition, drug developers must obtain institutional review board approval, secure informed consent, and comply with IND reporting requirements. The guidance further clarifies that expanded access submissions may be filed either by the drug developer (as the IND sponsor) or by a treating physician acting as a sponsor-investigator, and that the sponsor of a treatment IND does not need to be the sponsor of the well-controlled clinical trials for the same drug.
When a company has an active IND but declines to submit its own expanded access protocol, it may support a physician-initiated request by providing a Letter of Authorization (LOA). An LOA permits the FDA to rely on the company’s underlying IND (particularly CMC,3nonclinical, and clinical information) without disclosing proprietary data to the treating physician. Many companies rely on LOAs to enable access while maintaining control of their development IND.4
While the FDA cannot require drug developers to provide expanded access, drug developers must publicly post their expanded access policy in accordance with the requirements under the 21st Century Cures Act and the FDA Reauthorization Act for an investigational drug, including one’s decision to offer or to not offer any expanded access and contact information, among other required contents, by the earlier of 1) the first initiation of a phase 2 or phase 3 study with respect to such investigational drug or 2) within 15 days after the drug receives a fast track, breakthrough, or regenerative advanced therapy designation.
The FDA’s clarification aims to facilitate patient access while ensuring that expanded access does not undermine clinical trials, particularly in rare disease settings where patient populations are small. The guidance also provides drug developers with a clearer framework for structuring requests, managing adverse event reporting, coordinating with treating physicians, and planning for appropriate oversight.
For biotech and biopharma companies, the document underscores the need to align regulatory actions with patent and exclusivity strategies, especially when investigational data or observations from Expanded Access programs could predict future filings.
Integrating IP and Regulatory Timelines
Expanded access programs can expose investigational data, dosing regimens, or patient outcomes before a product’s formal approval. Drug developers should ensure all core patent applications, such as those covering composition, methods of use, and formulations, are filed before initiating any expanded access use or supplying the investigational product outside controlled development settings. Coordination among regulatory, clinical, and IP teams helps avoid inadvertent public disclosures that could compromise patentability or shorten exclusivity periods.
Companies should also work with legal counsel to review or prepare the supply agreements under which investigational product is provided to treating physicians or institutions. These agreements typically include robust IP and confidentiality terms, restrictions on data use, operational and safety compliance obligations, and liability and indemnification provisions to ensure appropriate handling of the investigational drug and compliance with FDA requirements while protecting the drug developer’s interests.
Capturing and Protecting Emerging Insights
Although data from expanded access use are limited, subject to bias and small sample size, typically uncontrolled and do not establish safety or efficacy for broader patient populations, they may still reveal valuable clinical insights. Drug developers should evaluate whether such findings warrant additional IP filings, including continuation or continuation-in-part applications to capture new uses, dosing strategies, or patient subgroups. Maintaining confidentiality through Non-Disclosure Agreements (and data-use agreements with treating physicians and institutions is also essential to preserving proprietary rights, including access and rights to permitted use of the expanded access data).
For additional information about how to navigate the FDA’s expanded access framework while safeguarding IP value as well as questions related to requirements for an expanded access policy, LOA, and potential impact of expanded access on ongoing clinical trials, please contact Wilson Sonsini attorneys Eva F. Yin, William Barrett, Daniel E. Orr, or Roshni Ghosh.
[1] U.S. Food and Drug Administration, “Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers” (October 2025), available at https://www.fda.gov/media/162793/download.
[2] Investigational New Drug application is a request to the FDA for permission to test an unapproved drug or biologic in humans.
[3] Chemistry, Manufacturing, and Controls (CMC) information refers to the data describing core elements such as composition, manufacturing process, quality controls, and stability of an FDA review for any investigational drug to ensure product quality and safety.
[4] See U.S. Food and Drug Administration, Expanded Access—How to Submit a Request (Forms), https://www.fda.gov/news-events/expanded-access/expanded-access-how-submit-request-forms (last visited: Dec. 4, 2025).
