Establishing Expanded Use Programs

Laura Ng

Senior Editor

Loyola University Chicago School of Law, JD 2021

The ability to access experimental drug treatments has long been contentious in the United States. Prior to the 1938 Food, Drug & Cosmetic Act, pharmaceutical drugs were largely unregulated. This Act required, for the first time, that drugs sold to the public were safe. Increasing regulations regarding the marketing, testing, and distribution of pharmaceutical drugs were established throughout the next fifty years. In the 1980s, however, a puzzling illness became known to the public. This illness was called HIV/AIDS, a debilitating virus that caused the body’s own immune system to attack itself. This illness has killed over 32 million people in the United States and worldwide, and particularly impacted the population of gay men. Because homosexuality was still fairly taboo in the 1980s, many argue that the country dragged its foot in researching and approving treatment for HIV/AIDS. Due to significant advocacy, much progress has been made with this particular illness, and the country has slowly evolved from the slow and strict processes that once regulated potential pharmaceutical drug treatments.


Advocacy to gain access to new, unproven pharmaceutical drugs have continued in recent years, particularly with the development of social media in the 2000s. In turn, Right-to-Try legislation have been passed, allowing patients to receive experimental drug treatments before official FDA approval. It is important to note that “Right to Try” does not guarantee patients the right to access any particular drug; the discretion is that of the companies. Companies have also created Expanded Use programs allowing patients who cannot participate in clinical trials to access the drugs, and for whom no comparable drug is available on the current market.

Establishing Expanded Use Programs

In creating Expanded Use programs, companies ought to keep certain things in mind: first, information on participating in the Expanded Access policy should be posted on the company’s website in a clear and understandable manner for prospective patients and their families to access. Contact information with the program manager of the expanded access policy should also be provided, as well as full eligibility criteria (age, condition, etc.) in addition to exclusion criteria (conditions that may cause a poor outcome, etc.). The inclusion criteria and information regarding Expanded Use should clearly outline the purpose of the such a program: that it is for those who cannot participate in the clinical trials of the treatment in question, and that the program is only for those who cannot obtain a comparable drug on the market.

The company’s Expanded Access policy should comply with all regulatory requirements at the state and federal level. For international patients, such compliance should be in accordance with both the U.S. regulatory standards as well as the standards of the patient’s country. Additionally, the company should consider writing into the policy a statement that allows for case-by-case analysis in order to avoid false hope from patients, or perhaps the perception that the company is obligated to accept all who apply and meet the criteria.

Exclusion criteria should include any patient with a history of drug interactions with the treatment in question, of course. The company should also require that all participants/families of participants read and sign informed consent agreements prior to beginning the treatment. All patients should be required to provide a complete medical history, to be pre-approved by physicians at the company, before participating in the program. Patients should also grant access for the company to review their full medical history and to communicate with prior physicians who have treated the patients to verify their conditions.


While it is naturally the mission of pharmaceutical companies to aid those suffering from illness as quickly and effectively as possible, companies must still be prudent in their selection criteria and experimental research. Failure to do so could jeopardize the entire clinical trial program and risk setting the treatments back. Not screening out patients who could have poor reactions to the drugs could create a negative public perception of the drug, thus dooming its reputation before it is even given the chance to cure other patients who may not have such reactions. In terms of clinical setting, it is important to make sure that only licensed physicians are administering the treatments to the patients. All physicians and healthcare sites should be required to undergo training with the company to learn best uses of the drug and be pre-approved before being eligible to participate. Additionally, the pharmaceutical company ought to require that all physicians monitor the patients on a regular basis and report their findings back to the company. In addition to reports of adverse events, the company may find it prudent to obtain the right to collect additional data from the patients.