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Expanded Access and Right to Try: Alternative Paths to Experimental Treatments for Metastatic Breast Cancer

If there are no clinical trials you can join, There are two main pathways for requesting an experimental treatment from a drug company outside of a clinical trial: the U.S. Food and Drug Administration Expanded Access and Right to Try.
 
 

Expanded Access and Right to Try: Alternative paths to experimental treatments for metastatic breast cancer

If you would like to find out whether there’s an experimental drug that could be beneficial for you, talk to your medical team. Make sure that you’ve thoroughly researched whether there are any clinical trials you can join. In most cases, you won’t be eligible to request an experimental drug through Expanded Access or Right to Try unless participating in a clinical trial is not possible for you.

Doctors recommend enrolling in a clinical trial if you’re eligible since your participation helps advance treatments for breast cancer and, by participating, you receive extra medical attention and monitoring of side effects during the trial.

Still, if there are no clinical trials you can join and you have run out of other treatment options, talk to your medical team to see if they may be able to identify an experimental medication that could be beneficial for you and request it directly from the drug company.

It’s smart to talk with your doctor about the possible risks, benefits, and out-of-pocket expenses that may be involved with accessing an experimental drug outside of a clinical trial. The drug may cause unexpected serious side effects and may not be effective for you even if it helps others. The earlier a drug is in the development process, the less is known about it.

There are two main pathways for requesting an experimental treatment from a drug company outside of a clinical trial: the U.S. Food and Drug Administration (FDA) Expanded Access and Right to Try. In both pathways, your doctor makes the request on your behalf and the drug company gets to decide whether to provide the drug. The drug company can also decide which pathway you must use to make a request.

Here’s what you need to know about pursuing each pathway, as well as potential costs and how to begin researching experimental treatments that may have promise for you.

Expanded Access

The FDA’s Expanded Access pathway (also known as “compassionate use”) is a more established way to request an investigational drug. The FDA has helped people gain access to experimental treatments outside of clinical trials since the 1970s, but it formalized the Expanded Access pathway in 1987.

Expanded Access also provides safeguards for patients that Right to Try does not — a review of the request by the FDA and by an Institutional Review Board (IRB). An IRB is a committee — typically connected with a hospital or research institution — that reviews research plans and protects the rights and welfare of human subjects enrolled in clinical trials.

To be eligible to use the Expanded Access pathway, you must:

  • be diagnosed with a serious or life-threatening disease or condition

  • have no comparable or satisfactory alternative therapy

  • be unable to enroll in a clinical trial

Then you and your doctor must follow these steps:

  • Your doctor (who must be a licensed physician) works with you to find an investigational drug that is suitable for you. The drug can be at any stage in the development process or no longer under development.

  • Your doctor contacts the drug company to ask if it will provide the drug to you outside of a clinical trial.

  • If the drug company agrees to provide the drug, your doctor submits an Expanded Access request to the FDA. In some cases, the FDA may make changes to the treatment plan, such as changing the drug’s dosage.

  • The drug company lets you know if there are any costs you need to cover for access to the drug.

  • If the FDA tells your doctor the request may proceed, your doctor submits the Expanded Access plan to an IRB.

  • After explaining the possible risks and benefits of the experimental drug, your doctor has you sign an informed consent form to certify you understand the risks associated with taking the experimental drug.

  • Once your doctor receives the necessary permissions from the drug company, the FDA, and the IRB, the drug company ships the drug so you can start treatment.

Although there are no statistics available on the total number of Expanded Access requests that are received or approved by pharmaceutical companies, the pathway has worked for many of the eligible patients who pursued it. According to a 2018 report from an external assessment of the Expanded Access program, the FDA received approximately 9,000 Expanded Access requests between 2013 and 2018 and authorized 99% of those requests. Also, Pfizer, Novartis AG, and Janssen have all publicly reported that they approve more than 90% of the compassionate use requests they receive.

Right to Try

A federal law gives people who have a life-threatening disease or condition the right to work with a licensed physician to request an experimental treatment from a drug company outside of a clinical trial, with minimal oversight from the FDA and without IRB review (unless IRB review is required by the drug company or the institution where the patient is seeking treatment).

To be eligible to use the Right to Try pathway, you must:

  • be diagnosed with a life-threatening disease or condition

  • have tried all FDA-approved treatment options

  • be certified by a licensed physician to be unable to participate in a clinical trial involving the eligible investigational drug

Then you and your doctor must follow these steps:

  • Your doctor works with you to find an investigational drug that is suitable for you that has completed a Phase I clinical trial and is still in clinical testing and under active development.

  • Your doctor contacts the drug company to ask if it can provide the drug to you outside of a clinical trial.

  • If the drug company agrees to provide the drug, your doctor works with the company
    to develop a treatment protocol for your unique situation.

  • The drug company lets you know if there are any costs you need to cover for access to the drug.

  • After explaining the possible risks and benefits of the experimental treatment, your doctor asks you to sign an informed consent document to certify you understand the risks associated with taking the investigational drug.

  • Once the drug company approves your doctor’s request, it ships the drug so you can start treatment.

Since 2014, 41 states have passed Right to Try laws that are fairly similar to the federal bill. Experts say that, in general, federal law should apply when you use the Right to Try pathway no matter where you live in the United States.

The Goldwater Institute, a free-market public policy and legal organization, developed the model legislation that state and federal Right to Try laws are based on. Naomi Lopez Bauman, director of healthcare policy at the Goldwater Institute, says that a key advantage of the Right to Try pathway is that it’s less burdensome on physicians than the Expanded Access pathway.

“Right to Try can dramatically reduce the paperwork and administrative burden on a physician who might spend dozens of hours completing FDA paperwork and is not compensated for that time,” she says. “Right to Try also provides additional liability protections for physicians and drug companies who use the pathway. Expanded Access does not.”

While Right to Try laws were created with the intention of helping more people access investigational treatments, some experts say that they are unlikely to increase access in a meaningful way.

Alison Bateman-House, PhD, MPH, MA, an ethicist and assistant professor in the Department of Population Health at NYU Grossman School of Medicine, studies Right to Try laws. She says she is only aware of two patients who have received treatments through the Right to Try pathway since the federal law was passed.

“I don’t think that Right to Try will ever be a mainstream path for accessing investigational drugs,” she says. “Most of the time, drug companies will ask you to follow the Expanded Access path instead. They usually prefer to have the extra layers of oversight.”

Critics also say that Right to Try lacks important safeguards for protecting patients.

A number of major cancer organizations opposed the passage of the federal Right to Try law, including the American Society of Clinical Oncology (ASCO), the American Cancer Society Cancer Action Network, The Leukemia & Lymphoma Society, and Susan G. Komen.

ASCO published a brief stating that federal and state Right to Try bills are “crafted under the incorrect assumption that FDA is slowing down the expanded access process” and that the bills “remove critical patient protections from the process for no clear benefit.”

By law, drug companies cannot make a profit on any treatment that has not been approved by the FDA. But when they provide an experimental drug through Expanded Access or Right to Try, they are permitted to charge the patient for direct costs associated, for example, with manufacturing the drug.

If a drug company agrees to provide a drug through Expanded Access or Right to Try, they must let your doctor know whether they plan to charge for the drug — and if so, how much — at some point before you sign an informed consent document and begin treatment.

Dr. Bateman-House says her research has shown that, in practice, most pharmaceutical companies do not charge for the drugs they provide through Expanded Access.

Other costs associated with taking an experimental drug, such as extra clinic visits, lab tests, and imaging, may not be covered by your health insurance. Insurance companies, Medicaid, and Medicare are not required to cover costs related to investigational treatments used outside of a clinical trial. It’s best to talk with your insurance company before starting an investigational treatment to find out if they are willing to cover some or all of these costs.

If you can’t enroll in a clinical trial and have run out of other treatment options, you can search for alternative treatment options:

  • Go to Clinicaltrials.gov, click on “Advanced Search,” enter your condition or disease in the first search field, and then select “Expanded Access Studies” under the “Study Type” pull-down menu.

  • Visit the Company Directory that is part of the Reagan-Udall Foundation for the FDA’s online Expanded Access Navigator. The directory allows you to search for investigational drugs and for the Expanded Access programs and policies of various drug manufacturers.

  • Once you’ve identified an investigational drug you’re interested in, you can visit the website of the manufacturer of that drug and look for their “compassionate use,” “preapproval access,” or “expanded access” policies. If you’re having trouble finding that information online, call the company.

For more information on how to request an experimental drug outside of a clinical trial, visit Expanded Access and Right to Try. The Reagan-Udall Foundation’s Expanded Access Navigator also has step-by-step guides for patients, caregivers, and physicians on how the Expanded Access process works.

 
References
  1. RighttoTry.org. Right to Try In Your State. Available at: https://righttotry.org/in-your-state/.

  2. CancerAdvocacy.org. Cancer Leadership Council letter. May 21, 2008. Available at: https://www.canceradvocacy.org/wp-content/uploads/2018/05/CLC-to-House-Leadership-on-Right-to-Try-May-22-2018.pdf. (PDF)

  3. FightCancer.org. February Right to Try Coalition letter. February 6, 2018. Available at: https://www.fightcancer.org/sites/default/files/National%20Documents/February%20Right%20to%20Try%20Coalition%20Letter%20-%20Final.pdf. (PDF)

  4. ASCO.org. ASCO in Action Brief: Right-to-Try and Expanded Access to Investigational Drugs. May 9, 2018. Available at: https://www.asco.org/advocacy-policy/asco-in-action/asco-action-brief-right-try-and-expanded-access-investigational-drugs.

  5. FDA.gov Expanded Access Program Report. May 2018. Available at: https://www.fda.gov/media/119971/download.

  6. Pfizer.com. Compassionate Use & Expanded Access. Available at: https://www.pfizer.com/purpose/medicine-access/compassionate-use.

  7. Usdin, Steve. FDA to facilitate access to unapproved drugs. Available at: https://www.biocentury.com/biocentury/regulation/2018-12-14/how-fda-plans-help-patients-get-expanded-access-unapproved-drugs

— Last updated on February 9, 2022, 11:48 PM