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 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) drugs, biologics, and medical devices 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, biologic, and medical device development process and the differences between the available options. Note: A “biologic” is defined as a biological product used to diagnose, prevent, treat, and cure diseases and medical conditions. Biological products are a diverse category of products and are generally large, complex molecules.
Basic Drug, Biologic, and Medical Device 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 both unwanted and 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 drug, biologic, or medical device outweigh potential harms that may result from its 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 drug, biologic, or medical device 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.
Drugs, biologics, and medical devices shown to be safe and efficacious (that is, they produce the intended health outcomes) are approved for marketing, making them available to the broadest number of patients who need them.
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 drug, biologic, or medical device 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 drug, biologic, or medical device 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. All require the sponsor—the product manufacturer, physician, or other entity—to obtain an IND or IDE.
- Individual (Single) Patient IND or IDE: Provides access for a single, named patient and can be requested by the treating physician, product manufacturer, or other entity that serves as the sponsor.
- Intermediate-size Patient Population IND or Small Group Access 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. This option is available to the treating physician, product manufacturer, or other entity that serves as the sponsor.
- 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. This option is available only to the product manufacturer.
What Is Right-to-Try?
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 Right-to-Try Pathway.
- 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.