If you are critically ill and have exhausted available treatments, depending on your situation, you may want to consider two remaining options: clinical trials or expanded access to investigational drugs, biologics, and medical devices. (For medical devices, expanded access is called “compassionate use.”) Both clinical trials and expanded access/compassionate use may involve higher-known risks as well as unknown risks associated with products in development.
To help you understand your options—and decide whether to pursue them—Clinical Research Pathways has developed “Points for Patients to Consider” Feel free to download the document and discuss it with your healthcare provider. Patient advocacy groups are welcome to share this resource in either PDF or brochure form.
One of the best places to find out about investigational drugs, biologics, and medical devices is ClinicalTrials.gov. You can search the site by disease and geographic region. An Advanced Search page provides more options, such as phase of study, certain eligibility criteria, and whether expanded access is available.
Talk to your provider
Discuss your options with your physician or other healthcare provider, who can help you understand terminology, judge risk associated with the investigational drugs, biologics, and medical devices, and help you decide which, if any, might be an option for you. You might be eligible to participate in a clinical trial. If not, the Food and Drug Administration’s (FDA’s) expanded access program could be another option.
Be sure to consider how much is known about the investigational medical product, the severity of your condition, the availability of other therapies, and the likelihood that the investigational product will produce the outcome that you are seeking. Talk with your provider, also, about other conditions you may be experiencing, and medications you may be taking.
If your healthcare is managed by anyone other than a physician, you will need to find a licensed physician who is willing to do all of the following:
- Oversee your treatment.
- Work with the product manufacturing company and FDA.
- Obtain the medical investigational drug, biologic, or medical device.
- Monitor you during the course of treatment.
- File the necessary paperwork.
Although patients may contact a company to explore whether the company will provide treatment access, usually only a licensed physician can make a formal request for the product from the company. In addition, only a licensed physician can file an application to FDA’s expanded access program or access the investigational product through the right-to-try process.
Your physician must first consult with the company that makes the product to see whether the company is able and willing to make the investigational drug, biologic, or medical device available outside its clinical trial. Neither FDA’s expanded access program nor right-to-try requires a company to provide its product to patients.
The role of the IRB
When an investigational medical product is provided through FDA’s expanded access program, an institutional review board (IRB) helps protect you, the patient, by ensuring that the risks are reasonable in relation to the potential benefit. The IRB will require and review an informed consent document to make sure you are aware of the following:
- The product that will be used for treatment is investigational.
- The product has known risks—and you have been told what they are.
- There might be additional, unknown risks—and you are willing to accept the level of risk associated with the investigational drug, biologic, or medical device.
Carefully read the informed consent document and make sure you understand the risks associated with the investigational product before signing the document. Be sure to discuss with your physician any details you do not fully understand, including medical terms or procedures described in the consent document, and what you might realistically expect from being treated with an unapproved investigational drug, biologic, or medical device. Be sure you understand how your treatment will proceed, what costs are involved, and who will be responsible for them.
Under FDA’s expanded access program, at a minimum, the consent document should:
- State that the product is investigational.
- Describe the treatment procedure and expected duration.
- Specify what part of the treatment is investigational.
- Describe reasonably foreseeable benefits.
- Describe reasonably foreseeable risks and discomforts and include a statement that there might be unknown risk.
- Describe alternative options/treatments, if any.
- Explain whether compensation or treatment will be provided in case of injury caused by the treatment.
- Describe whether some or all of the treatment will be covered by insurance.
- Provide contact information in case you have questions about the treatment, injury, complaints, etc.
For example, the consent document should tell you if you will have to pay medical costs for the physician’s time, and whether any of the costs will be covered by insurance. The document might include information such as whether a procedure such as an MRI will be required during treatment, and who will be responsible to pay the cost. You should have adequate time to review and ask questions about the treatment, and you should receive a copy of the final, signed document.
Under the right-to-try law, although patients are required to sign an informed consent document, the law does not specify what information must be included. Nor does the right-to-try law require that an IRB review the consent document to ensure that information is included for the patient to make an informed decision whether to accept the treatment. However, if the investigational drug or biologic will be administered at a hospital or clinic, or if the physician is affiliated with an institution (hospital/clinic), the institution may still require IRB review, even if the investigational drug or biologic is provided through the right-to-try pathway.
Reasons why access might be denied
Even if you meet the basic criteria for treatment under FDA’s expanded access program, access to the investigational treatment is not guaranteed. Other factors affect access, such as:
- Your physician might not be able to obtain approval for expanded access for you because of your medical history or the risks associated with the investigational product.
- Your physician might determine that the risk from the investigational product is greater than the risk from the underlying disease.
- Your physician might not agree that an investigational product is your best or only option.
- Your physician might not be able or willing to manage the use of an investigational product.
- The company that makes the investigational drug, biologic, or medical device is not required to offer it outside their clinical trials and may not be able or willing to do so.
- Some companies have stated that their policy is to not provide access outside their clinical trial. You can often find a company’s policy on its website.
- Even if the company has a policy to make an investigational product available, the company might not have enough of the product available for all patients requesting treatment under expanded access.
The process for requesting access
Your physician must submit a formal request to the company asking for treatment access to an investigational product. The request usually includes the details of your medical history, such as age, your overall health picture, former treatment(s) for the condition, other conditions that might affect you, and what other medications you may be taking.
Different companies have different requirements and forms for applying for treatment access and have different time frames for responding. Recent federal legislation (The 21st Century Cures Act) requires companies to provide publicly information about their policy on treatment access, contact information at the company for inquiries, and approximate turnaround time on requests.
If it agrees to provide its product, the company will provide the physician with information called a Letter of Authorization, which is then sent to FDA along with the request for the treatment use. For treatment uses under expanded access involving investigational drugs and biologics, there is a standard, short-form (FDA Form 3926) application the physician prepares for FDA. If the treatment use involves an investigational device, the physician submits a written treatment plan or protocol to FDA.
FDA usually completes its review of the application within a few days and allows nearly all requests to proceed. The few that are denied are usually based on safety concerns that stem from having inadequate information about the product’s safety, or known serious medical issues—such as giving a product known to have serious adverse effects on the liver to a patient with existing severe liver problems. It’s beneficial to have the expert review from FDA because agency staff are experts in product safety. They often have significant insight into the product in question or other products in the same product class. Not all physicians are familiar with investigational drugs, biologics, and medical devices. Unless physicians are involved in the research of the product, they are likely to be unfamiliar with how to administer and monitor its use.
After it approves the request, FDA will issue an IND or IDE number and send that number to your physician. Once your physician informs the company that he or she has the IND or IDE number, the company will ship the product to your physician.
Investigational products are expensive to make. Some companies do not charge for the product or ancillary services, such as shipping. However, the company may request authorization from FDA to charge you the direct costs (such as manufacturing and shipping) and indirect costs (such as reporting and IRB approval) of making the investigational drug, biologic, or medical device available.
Most insurance companies will not pay for access to an investigational product. There may be additional costs for administering the investigational drug and for monitoring your condition by your hospital or healthcare provider. It is important for you and your physician to consider the cost of the investigational drug, biologic, or medical device and/or the medical services associated with its use that are not covered by third-party payers such as insurance or Medicare.
If you or a loved one is critically ill and has exhausted standard treatment options, you face some difficult choices. To help you make informed decisions, Clinical Research Pathways has developed educational materials on clinical trials and the process for requesting expanded access to experimental medicines.
From Clinical Research Pathways
- Intermediate-size Patient Population Expanded Access Use
- Responsibilities of a Sponsor-Investigator (Treating Physician)
- How to Request Treatment Use via FDA’s Expanded Access Process (Non-Emergency)
- Points for Patients to Consider, PDF version, printed brochure, printing instructions
- The Right-to-Try Pathway
- Streamlining IRB Procedures
- FDA Guidance: Expanded Access to Investigational Drugs for Treatment Use – Questions and Answers (October 2017)
- FDA Guidance on IDE Policies and Procedures, (January 20, 1998)
- FDA website
- FDA Form 3926 *Right-click and select “save link” to save the file to your computer)
- For drug-related expanded access: FDA Division of Drug Information, 301-796-3400 or email@example.com
- For biologics-related expanded access: FDA Biologics Office of Communication, Outreach, and Development, 800-835-4709 or firstname.lastname@example.org
- For medical device-related expanded access: FDA Device Division of Industry and Consumer Education, 301-796-7100 or DICE@fda.hhs.gov