If you’ve recently heard about a new cancer drug, you may be eager to talk with your doctor about whether it’s right for you. But even if the drug is a good fit, it may take some time and persistence to get access to that new treatment. The good news is, understanding the drug approval process can help you ask critical questions and take specific steps when talking to your doctor. Here are the questions you should keep in mind as you discuss your options with your health care team:
1. Is the drug FDA approved?
The U.S. Food and Drug Administration (FDA) has regulatory oversight of approving drugs in the United States to ensure that they have been determined in clinical trials to be both safe and effective. Drugs are approved by FDA for use in specific groups of patients with specific types of cancer. The “drug label” or “package insert” describes the approved use(s) of the drug. If the drug is approved for your kind of cancer, then your doctor can prescribe it for you if it seems like a good choice to include in your care plan. You can search for approved drugs on the FDA’s website.
You can also find drugs approved for specific diagnoses at the National Cancer Institute’s website, or explore details on dosing, side effects, and precautions of thousands of drugs on Medline Plus Drug Information.
If you’ve found a drug you’re interested in, you should ask your doctor if the drug is a good fit for you and whether they would recommend the treatment. Possible questions to ask your doctor include:
Has the drug been approved for my cancer or tumor type?
How effective is the drug?
What are the drug’s side effects?
Are you aware of whether insurance will cover this drug?
Are there other treatment options I should consider?
Before treatment, check with your health insurer to see if the drug is covered by your insurance plan. You can do this by checking your insurer’s website and reading your specific plan’s Summary of Benefits. You can also call the insurer directly to ask about coverage. If it’s not covered, you can pursue your insurer’s “drug exception” process, which can help you get access to the drug. Your doctor might need to contact the insurer directly to describe how the drug is the best treatment option for your cancer. Finally, you should always consider the out-of-pocket costs of any new drugs or treatment regimens. Even if the drug is covered, there may be a regular co-pay for it or other related costs.
2. Does your doctor recommend using the drug “off-label?”
If the drug is not approved by the FDA for your type of cancer, there may still be options. Ask your doctor whether they would prescribe the drug outside of its approved indication based on medical evidence that supports its use in a case like yours. This is called off-label use. Your doctor may need to read research studies and other clinical guidelines first to see if scientific evidence supports its use for your type of cancer.
Off-label use happens with all kinds of drugs but getting health insurance to cover off-label use of medications can be tricky. Check to see if your plan covers a drug’s off-label use by calling your health insurance company directly. You can also submit a claim to your insurer requesting that it reimburse you for the medication cost. If there isn’t a lot of research about the drug’s off-label use, your insurance may or may not cover the cost. Research whether drug makers or other programs for financial assistance can help cover the drug cost.
3. Is the drug investigational?
Investigational drugs—also called experimental drugs—are medications that are still being tested by the drug maker and have not yet received FDA approval to be marketed, sold, or prescribed. They are most commonly available to people taking part in clinical trials to demonstrate the drug’s safety and efficacy. If you are interested in joining a clinical trial, a good first step is to let your oncologist know.
4. How do I find a clinical trial to take part in?
Your oncologist can guide your search for a clinical trial. If you’d like to search online, the National Cancer Institute has a searchable database of its clinical trials. The U.S. National Library of Medicine also keeps an online database called ClinicalTrials.gov, which you can search by drug name, your type of cancer, and other parameters. Some patient advocacy groups maintain disease-specific clinical trial databases. These and other cancer clinical trial databases can be found here.
Once you’ve found a potential clinical trial, check if you’re eligible to participate. Your eligibility may depend on your age, sex, the type of cancer you have, how long you’ve had cancer, other treatments you’re receiving, and other medical conditions you may have. If you find that you’re eligible, the next step is to ask whether the clinical trial is still accepting new participants. Your doctor can help you navigate the eligibility criteria and availability of clinical trials.
5. What else should I consider in joining a clinical trial?
All clinical trials have specific goals, so try to determine whether those goals are in line with your goals for treatment. The location of the facility offering the clinical trial is another thing to think about, as that will affect your time and travel expenses. Find out how often you would need to travel to the study site’s location if you participate. If necessary, evaluate the cost of taking off work or arranging for childcare. You should also consider whether your insurance will cover your routine care costs while you’re on the trial. For help covering the costs, inquire about any financial assistance the sponsor of the clinical trial may offe
If you have any questions about the clinical trial process, ask your doctor to contact the team running the clinical trial. You can also contact the team directly.
6. If I’m not eligible or the clinical trial is closed, what are my options?
If you can’t participate in a clinical trial for a specific drug you are interested in, then you have 2 options for drug access: “Expanded Access Programs” or “Right to Try.”
Expanded Access Programs, which are also called compassionate use programs, help patients with serious illnesses get access to investigational drugs. Your doctor applies to the drug manufacturer for access. If approved by the manufacturer, your doctor must submit an application to the FDA seeking permission to treat you with the drug. FDA typically approves about 99 percent of the applications it receives for expanded access. Then, your doctor will oversee your treatment with the investigational drug.
Recently, the FDA announced a new pilot program called Project Facilitate to help patients and health care providers find access to investigational cancer drugs through Expanded Access.
Another route you can follow is Right to Try. The Right to Try Act is a 2018 law allowing patients with life-threatening illnesses who have tried all other treatment options to access investigational drugs. Through Right to Try, you and your doctor contact the drug maker directly to see whether they can provide the investigational drug. The FDA is not involved in reviewing or approving these requests, and the manufacturer is not required to grant access to the investigational therapy.
7. Are the risks of taking an investigational drug worth it to me?
Taking an investigational drug is always a risk because the drug has not been approved by the FDA as a safe and effective medication. That means there could be unknown side effects, or it might not work at all. At the same time, the drug might be your best chance for successful treatment. Or, you may have tried all other treatment options. Consider what’s most valuable to you in terms of your quality of life, life expectancy, and other treatment options. Balancing these risks with potential benefits is an individual choice—one that you should discuss with your family and health care team.