Experimental or investigational

When a plan calls a treatment experimental, investigational or unproven, it says the evidence does not yet show that the treatment works well enough to cover. That can be true. It can also be a label the plan applies to a treatment your doctor and the published guidelines already accept. The appeal is a contest over the evidence.

What this means

The label comes from a definition in your plan document, so find it first. A plan’s definition of “experimental or investigational” may refer to things such as approval by a regulator, published studies or professional guidelines. Read the exact words, because your best argument is often that the evidence meets the plan’s own definition.

This is a medical-judgment denial. The federal external review rule lists “experimental or investigational status” among the denials that qualify, along with medical necessity, appropriateness, health care setting, level of care and effectiveness. The notice must either explain the scientific or clinical judgment, applying the plan’s terms to your circumstances, or say the explanation is free on request. On appeal, the reviewer must consult a health care professional with training and experience in the field, who was not consulted on the first decision.

Clinical trials. A plan that is not grandfathered may not deny a qualified person participation in an approved clinical trial. It may not deny or limit coverage of the routine patient costs, meaning the items and services it would typically cover for someone who is not in a trial.

Medicare Advantage. A plan must follow Original Medicare’s coverage rules. It may write its own criteria only when Medicare’s are not fully established, and any such criteria must rest on current evidence in widely used treatment guidelines or clinical literature and be posted publicly.

What to do

1. Get the definition. Ask for the plan document and the medical policy the plan used. The plan documents request letter and the guide on finding the medical policy help. (You.)
2. Get the plan’s evidence. Use the clinical criteria request. Ask what studies or sources the plan relied on, the reviewer’s specialty and the date of the policy. (You.)
3. Ask your doctor for a letter. It should state your diagnosis, why this treatment is chosen, what else was tried or ruled out, the doctor’s own experience with the treatment, and what is expected without it. If the treatment is a drug used for a purpose its label does not list, the clinician letter on off-label use is the model. (Your doctor.)
4. Gather the evidence. Collect peer-reviewed articles, professional society guidelines and regulatory approvals that support the treatment for your diagnosis. FAIR Health, a nonprofit that publishes cost and insurance information, advises asking the insurer how it defines “experimental”, then answering with journal articles, guidelines, regulatory approvals and the doctor’s experience. The Arthritis Foundation’s advice on “effectiveness not proven” is to cite peer-reviewed literature and name where you searched. (You and your doctor.)
5. Map each piece of evidence to the definition. Make a short table: the plan’s requirement on one side, your evidence on the other. (You.)
6. File the appeal. Use the experimental treatment appeal letter. Employer and non-grandfathered individual plans must allow at least 180 days after you receive the denial. Check the date on your notice, which controls. (You.)
7. Request external review. You have at least four months after the final denial. The independent reviewer looks at the case fresh, considers your records, your treating professional’s recommendation, the plan terms and evidence-based practice guidelines. (You.)
8. Ask for urgency if delay could harm you. Your doctor certifies it. (Your doctor.)

What the plan may say, and what to say back

“The treatment is investigational.” Ask for the plan’s definition and the specific studies it considered. Reply with the guidelines and articles your doctor cites and the point at which they meet the definition.

“It has not been approved for your condition.” Ask your doctor to explain the basis for the use and to cite the guideline or studies. See the off-label clinician letter.

“It is part of a research study.” If you are in an approved clinical trial and your plan is not grandfathered, ask the plan to cover the routine costs of care you would receive outside a trial.

The rule

A non-grandfathered plan may not deny a qualified individual participation in an approved clinical trial, and may not deny or limit coverage of routine patient costs. 42 United States Code (U.S.C.) 300gg-8

Watch for

• The plan’s definition governs. A published study that does not meet the definition may not persuade the reviewer. Quote the definition in your appeal.
• Grandfathered plans do not have the clinical trial rule. Ask the plan whether it is grandfathered.
• Routine costs only. The rule covers items and services the plan would typically cover for someone not in a trial. Ask the plan in writing how it will treat the rest.
• No guarantees. Strong evidence does not guarantee a decision in your favor.

What next

• Not medically necessary: the related clinical argument.
• Find guidelines and studies: where to look for evidence.
• How external review works: the independent review.
• Treatments, tests and equipment topic page: all related guides and letters.