Resources for Expanded Access
Clinical Research Pathways is committed to helping desperately ill patients who want the chance to try investigational drugs, biologics and medical devices. These patients have exhausted all treatment options and are not able to participate in a clinical trial or wait for existing trials to be complete. As a last resort, patients can seek access to investigational (unapproved) products through two pathways: Food and Drug Administration’s (FDA’s) expanded access process and, under new federal legislation, right-to-try. Before considering either of these pathways, it’s helpful to understand the drug development process and the differences between the available options.
Basic Medical Product Development
Medical products (drugs, biologics, or medical devices) in the United States are developed through a process called a “clinical trial,” which is the study of how a proposed diagnostic, therapeutic, or vaccine affects the human body and a disease process. Clinical trials are carefully controlled to help protect study participants from potential harms caused by the medical product, and to demonstrate what effect can be expected in preventing or treating a medical problem. And, since virtually all medical products can cause unwanted, as well as desired, therapeutic effects, clinical trials are used to identify side effects. This information is used to determine whether the potential benefits offered by the investigational agent outweigh potential harms that may result from their use.
Usually, clinical trials are conducted in three distinct phases. Each progresses based on information gained from the phase before, to gather better information about the safety of a proposed product and its ability to produce the intended results. Ideally, clinical trials provide data from increasing the number of people enrolled in the research through the three phases, potentially identifying less common side effects and giving insight into longer-term safety.
What Is FDA Expanded Access?
Sometimes called “compassionate use” or “treatment access,” expanded access is the use of an investigational product outside of a clinical trial for treatment. An investigational product is called an investigational new drug (IND) if it is an unapproved drug or biologic and an investigational device exemption (IDE) if it is an unapproved medical device.
Clinical trials involving the use of an investigational medical product by patients generate data that lead to the approval of products and, therefore, provide the widest availability to patients who might benefit from the product. However, not all patients are eligible to participate in clinical trials. And not all patients have time to wait for an investigational product to be approved for marketing.
Normally, unapproved medical products cannot be legally provided to patients. Expanded access is an exception. This regulatory pathway, created by FDA, allows medical product developers to provide access to their products in development, outside of the clinical trial.
Expanded access is possible at all stages of medical product development. Decisions about whether to grant expanded access for a treatment use are made on a case-by-case basis—first by companies and then by FDA—based on the relative benefit/risk determinations specific to individual patients and products.
Only a licensed physician can request an IND or IDE for an expanded access use; patients cannot submit a request directly.
There are three distinct tiers of expanded access. In each case, the physician or the product manufacturer or other entity that serves as the sponsor must obtain an IND or IDE.
- Individual (Single) Patient IND or IDE: Access for a single, named patient.
- Intermediate-size Patient Population IND or IDE: Intended to allow treatment of a limited number of patients (usually up to about 100). Based upon information about the safety of the product from clinical trial testing that demonstrates adequate confidence that the product will not cause undue harm to patients.
- Treatment IND or IDE: Used when there is significant safety information about a proposed product from clinical trials and demonstrated efficacy. This usually means that the product is in a phase 3 clinical trial, or the clinical trials have been completed, and data are being analyzed. The treatment IND or IDE is used for treating large numbers of patients, such as from hundreds to tens of thousands, outside of a clinical trial, but before the product is granted final marketing approval.
Right-to-try is legislation passed by Congress in 2018 to allow treatment access to investigational drugs and biologics. An alternative pathway to FDA expanded access, right-to-try differs from FDA expanded access in significant ways. A partial list is featured below. For more detailed information, go to Making Sense of the New Right-to-Try law.
- Only applies to certain investigational drugs and biologics. It does not include medical devices.
- Can only be used for life-threatening diseases and conditions. It does not include “serious diseases and conditions” and therefore is narrower in application than FDA’s expanded access program.
- Only applies after phase 1 clinical trials are completed.
Only applies to drugs and biologics that are actively in development for marketing.
- Requires informed consent by the patient but does not provide criteria for that consent.
- Does not require institutional review board (IRB) review or oversight. IRBs provide independent review to help ensure that risks are reasonable based on the information available about the product, and that the informed consent is adequate to allow patients to make informed judgment about using an investigational product.
- Releases manufacturers, distributors and physicians from liability related to providing access to unapproved products, except for reckless or willful misconduct, gross negligence, or an intentional tort under any applicable state law.
Forty-one states have also adopted right-to-try laws. Local laws differ from the federal law and from each other.
Patients and physicians who wish to obtain an investigational product through the right-to-try pathway should contact the product manufacturer to determine whether the product is available under right-to-try. Physicians affiliated with institutions, such as a hospital or academic medical center, should contact the relevant office within their institution to determine whether the right-to-try pathway is an option.
The Role of Clinical Research Pathways
To help patients and to support the critical roles of physicians and IRBs, Clinical Research Pathways seeks to reduce the procedural obstacles that desperately ill patients can face. We offer assistance to physicians who are applying for expanded access for their patients. We also offer assistance to IRBs, providing information on FDA requirements, how to streamline the IRB review process for individual (single) patient expanded access, and what to consider when reviewing protocols for intermediate-size patient population expanded access.
Perhaps most important, Clinical Research Pathways is a resource to those involved in any way in requesting expanded access to investigational products. If you have questions about pathways to investigational products or are looking for the latest information on FDA expanded access or right-to-try, we are here to help.