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

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Most often, the way that people access an experimental treatment for cancer is by enrolling in a clinical trial. Clinical trials are research studies in which people volunteer to try a new drug under careful supervision to help doctors find out if it’s safe and effective. By joining a clinical trial, you can potentially try a treatment that isn’t otherwise available and that might be beneficial for you.

In relatively rare cases, you might not be able to find a clinical trial that you can join because, for example, you don’t meet the eligibility criteria for any trials or you live too far away from the nearest medical center that has a trial that is appropriate for you.

If you find yourself in this situation and have run out of other treatment options, you and your doctor may be able to identify an experimental medication that could be beneficial for you and request it from the drug company.

Be sure to carefully consider and 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 pathway 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, potential costs, and how to begin researching experimental treatments that might 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 will make changes to the treatment plan, such as changing the dosage of the medication.
  • The drug company will let you know if you will be charged for any of the costs associated with providing the drug.
  • If the FDA tells your doctor the request may proceed, your doctor will submit the Expanded Access plan to an IRB.
  • After explaining the possible risks and benefits of the experimental treatment, your doctor will have you sign an informed consent form to certify that 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 will ship the medication and treatment can begin.

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

In May 2018, President Trump signed into law Senate Bill 204 (officially called the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017). The new 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 1 clinical trial and is still in clinical testing and under active 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 will work with the company to develop a treatment protocol for your unique situation.
  • The company will let you know if you will be charged for any of the costs associated with providing the drug.
  • After explaining the possible risks and benefits of the experimental treatment, your doctor will ask you to sign an informed consent document to certify that you understand the risks associated with taking the investigational drug.
  • If the request is approved by the company, they will provide the drug to your doctor and treatment begins.

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

The Goldwater Institute, a free-market public policy and legal organization, developed the model legislation that the 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, Ph.D., MPH, MA, an assistant professor in the division of medical ethics at NYU 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. For example, 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.”

What are the potential costs of receiving an experimental drug outside of a clinical trial?

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 will charge for the drug (and if so, how much) at some point before you sign an informed consent document and begin treatment.

Bateman-House says that 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 medication, such as extra clinic visits, lab tests, and imaging may not be covered by your 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 will cover some or all of these costs.

How to find out if there’s an experimental drug that could be beneficial for you

First, 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 also recommend enrolling in a trial if it’s an option since you’ll be participating in research that helps advance treatments for breast cancer and you’ll receive extra medical attention and monitoring of side effects during the trial.

To find out if there’s a clinical trial that’s right for your unique situation:

  • Search for trials on Clinicaltrials.gov.
  • Ask your doctors if they know of any clinical trials that would be suitable for you.
  • Contact the large cancer centers in your region to find out about their clinical trials.
  • Consider traveling to meet with an oncologist at a major cancer center that has a lot of clinical trials to discuss your options.

If you find a clinical trial that might be suitable for you but you’re not sure if you meet the eligibility criteria, don’t hesitate to reach out to the trial’s research coordinator or principal investigator. In some cases, they may be able to make exceptions to certain criteria so you could enroll in the trial. They may also know whether you could instead request the treatment they are studying through Expanded Access.

If you can’t enroll in a clinical trial and have exhausted other treatment options, where can you search for investigational treatments that may be available through Expanded Access or Right to Try?

You can search for treatment options on the following sites:

  • 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 the FDA’s sites about Expanded Access and Right to Try. Also, the Reagan-Udall Foundation for the FDA’s Expanded Access Navigator has step-by-step guides for patients, caregivers, and physicians on how the Expanded Access process works.

Written by: Jen Uscher, contributing writer

References

Right-to-Try.org. Right to Try In Your State. Available at: http://righttotry.org/in-your-state/.

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)

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)

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.

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

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

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.


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