Experimental Treatments and Clinical Trials
Most health plans don’t cover treatments they regard as “experimental.” Sometimes, they may deny a claim for such a treatment. But, you and your doctor may think the treatment is well supported by evidence. Then, you have grounds for an appeal.
Appealing a decision. If you choose to appeal, follow the advice in If Your Plan Doesn’t Pay (Appealing a Reimbursement Decision). Find out how the insurer defines “experimental” and why it believes your treatment is experimental. Then, make your case with evidence showing that the treatment is safe and effective. Support your case as appropriate with a letter from your doctor.
Clinical trials. In other cases, an experimental, or investigational, treatment may be just what you want. For example, you may have tried standard treatments without success. You may want to take part in a clinical trial—a study in humans—of a promising new treatment. To start looking for such a trial, ask your doctor. You also can search for trials yourself at ClinicalTrials.gov.
If a trial interests you, read the protocol summary, a document with basic information about the trial. See if you’re eligible, where the trial is located and whether its purpose fits with yours. If you decide you want to know more, call the trial team at the phone number in the protocol summary. Ask questions, such as what the possible risks and benefits are of the treatment being studied, and how they compare to those of standard treatments.
If you join a federally approved trial, most health plans can’t refuse to let you take part. They usually won’t pay for the investigational treatment. But, often, the trial sponsor will supply that treatment for free. Usually, you’ll keep on getting routine care from your own doctor, and your insurer should continue to pay for that. In the trial setting, most plans are required to pay for routine care costs under certain conditions.
Expanded Access. If you can’t get into a clinical trial but are seriously ill, you still may be able to get an investigational treatment through the FDA’s Expanded Access program.
Most health plans only cover treatments they think are medically proven to work. But, medicine is always changing. Opinions can differ about whether there is enough evidence to support a treatment. In such a case, an insurer may say no to paying for a treatment that it calls “experimental,” even though your doctor thinks the treatment is well supported. That can leave you with a big bill—unless you successfully appeal the insurer’s decision. In other cases, an experimental, or investigational, treatment may be just what you want. You may have tried standard treatments without success. You may hear of a clinical trial—a study in humans—of a new treatment that has shown promise. You may want to take part in the trial, in hope of getting better. But, how do you learn more about such trials? And, will your insurance cover any of the costs?
This guide will show you:
- How to appeal an insurer’s denial of your claim on the grounds that the treatment is experimental;
- How to find out about clinical trials;
- How to decide whether to enroll in a clinical trial;
- How much you can expect your health insurer to pay for your participation in a clinical trial; and
- What to do if you can’t enroll in a clinical trial but still want an investigational treatment.
Appealing Decisions That a Treatment Is Experimental
If your insurer denies your claim because it says the treatment is experimental, follow your insurer’s appeals process. Also, follow the advice in Appealing a Reimbursement Decision. Get as much information as possible about the decision in writing from the insurer. You’ll want to know how the insurer defines “experimental” and what specific grounds it has for believing your treatment is experimental.
Then, make clear to the insurer in writing why you believe your treatment is not experimental. Support your case as appropriate with a letter from your doctor. There are several kinds of evidence that might be persuasive. Cite journal articles showing that the treatment is safe and effective. Your doctor can detail his or her experience treating patients successfully with the treatment. Cite guidelines recommending the treatment and approvals by the Food and Drug Administration (FDA). Sometimes insurers might use data or documents that are out of date to rule a treatment experimental. If that’s the case here, point that out.
You’ll generally have three chances to appeal. Two phases are internal (appealing to people inside the insurance company) and one external (appealing to people independent of the company). If you need the treatment urgently, ask for an expedited review. That will get your appeal heard faster.
Learning More about a Clinical Trial
If you’ve decided you want to know more, call the trial team at the phone number listed in the protocol summary. Ask for the trial coordinator. If you prefer, have your doctor make the call.
Now, you’ll have another set of questions to ask—this time asking the trial team. These questions include:
- Why do you think the treatment you’re studying is better than standard treatments? What tests has it already undergone?
- What are the possible risks of the treatment being studied?
- What are the possible benefits?
- How do the risks and benefits of the new treatment compare to those of standard treatments?
- For what costs will the trial pay? (For example, the cost of the treatment; travel expenses.)
- Who’ll pay if I’m injured during the trial?
- If the treatment helps me, will I be allowed to keep getting it after the trial is over?
Once you’ve gotten answers to all your questions, consider whether to enroll. If you decide you will enroll, make an appointment with the trial team.
If You Can’t Get into a Clinical Trial
You may not be able to get into a clinical trial. Perhaps it’s because you don’t meet eligibility criteria or you live too far away. But, you may still be able to get the treatment under study.
The FDA has an Expanded Access or Compassionate Use program. Through that program, manufacturers make investigational medical products (such as drugs or medical devices) available to people who are seriously ill but can’t participate in a clinical trial. The patient must be someone for whom standard approved treatments don’t work or can’t be used. And, the probable risk from the investigational product can’t be greater than the probable risk from the illness. You may have to pay for the product, or the manufacturer may give it free of charge. For more information, see the FDA’s Expanded Access page.