Experimental or investigational

What it means

Your plan says the treatment, test or use of a drug is experimental or investigational. That usually means the plan thinks the evidence is too thin to cover it. Plans define these words in their documents, so the definition is the first thing to read.

Two situations look alike. A treatment can be new, or used off-label (a drug prescribed for a condition its label does not name), and still be supported by guidelines and studies. Or a plan can apply the word to a service it would rather not pay for. Either way the plan has made a judgment about evidence, and evidence can be answered.

When a denial rests on experimental treatment, the notice must either explain the scientific or clinical judgment applied to your circumstances, or say that the explanation is free on request.

Who has to act first

You and your doctor, with different jobs.

• You: ask the plan in writing for its definition, the policy it used, and the claim file, all free. File the appeal on time: at least 180 days after you receive the notice for a job-based plan or a non-grandfathered individual plan (check the date on your notice, which controls). Medicare, Medicaid, TRICARE, Veterans Affairs and federal employee plans have different, often shorter, deadlines.
• Your doctor: sets out the clinical case: what has been tried, why this treatment, and what published evidence supports it. Your doctor can also ask for a peer-to-peer call (a doctor-to-doctor conversation with the plan’s reviewing physician).

If delay could seriously harm your health, your doctor can say the claim is urgent, and the plan must decide within 72 hours.

Arguments that work

• Fit the plan’s own definition. Show, using the words of your plan document, that your treatment does not meet the definition of experimental, for example because a professional society guideline recommends it.
• Guidelines and published studies. The independent reviewer considers evidence-based practice guidelines along with your records and your treating doctor’s recommendation. Ask your doctor to name them.
• Ask what the plan relied on. Request its policy on this treatment for your diagnosis. For a group health plan, the claim file includes statements of plan policy about the denied treatment, whether or not the plan relied on them. You can also ask who advised the plan.
• Your own circumstances. Standard treatments you have tried, failed or cannot take belong in your doctor’s letter.
• Clinical trials. A non-grandfathered plan may not deny you participation in an approved clinical trial, and may not deny or limit coverage of routine patient costs, meaning items and services the plan would typically cover for someone not in a trial. If the denied item is routine care during a trial, say so.
• Medicare Advantage. The plan must follow Original Medicare’s coverage rules. It may write its own criteria only where Medicare’s are not fully established, must base them on current evidence, and must publish them with a summary of the evidence.

Evidence to gather

• The plan’s definition of experimental or investigational, and the policy it applied (the plan, in writing).
• The claim file and the names of any experts consulted (the plan, free on request).
• A letter from your treating doctor (your doctor).
• Guidelines and peer-reviewed studies that support the treatment (your doctor).
• Records of earlier treatments and their results (your provider or pharmacy).
• If you are in a trial: proof of enrollment (the trial site).

What to send

Send one packet to the address in the denial notice, by a method that proves delivery: an appeal letter that quotes the plan’s definition and says why you meet it, your doctor’s letter, the guidelines and studies, and your records. Send copies, never originals. Ask for the decision in writing, and ask about external review if the denial is upheld.