How to appeal an experimental or investigational denial

Federal claims rules treat a decision that care is experimental or investigational as a denial you can appeal. Each plan writes its own definition of the term, usually in the definitions or exclusions section of the plan document. Your appeal has to answer that exact wording, so find it first.

The label can be applied to a new device or procedure, a test with little published evidence, or a drug prescribed for a use the FDA has not approved. Doctors may prescribe an approved drug for an unapproved use when they judge it medically appropriate.

Ask the plan in writing for:

• The plan document's definition of experimental or investigational, and the exclusion it applied.
• The clinical policy or guideline the reviewer used, which employer plans must give you or provide free on request.
• The plan's explanation of the scientific or clinical judgment for the decision, applied to your medical circumstances.
• Your full claim file, which you can get free of charge.

These rules cover employer plans and, through 45 CFR 147.136, most individual plans. On appeal, an employer plan must consult a health care professional with training and experience in the field of medicine involved. For deadlines, see the pages for employer plans, Marketplace plans, Medicare Advantage, Original Medicare and Medicaid.

The central question in these appeals is whether the evidence supports the treatment for someone in your situation. Ask your doctor's office to gather:

• FDA status. Whether the drug or device is FDA-approved, and for which use. For an off-label use, FDA has not determined that the drug is safe and effective for the unapproved use, so the appeal needs other evidence for that use.
• Drug compendia. Listings in standard reference compendia such as the American Hospital Formulary Service drug information, which the insurance commissioners' model law counts as medical or scientific evidence.
• Clinical practice guidelines from medical specialty societies that recommend the treatment for your condition.
• Peer-reviewed studies, ideally controlled trials, in patients like you.
• Your history. Standard treatments already tried, with dates and results, and why they failed or cannot be used.

A letter from your doctor should tie this evidence to your case and to the plan's definition, point by point.

In most private plans, an experimental or investigational denial can go to an independent reviewer. The federal external review process covers a plan's determination that a treatment is experimental or investigational, and HealthCare.gov lists these denials among those eligible.

State review programs must handle these cases in a way substantially similar to section 10 of a model law written by the National Association of Insurance Commissioners. Under that model, the treating physician certifies that standard treatments have not worked, that standard treatments are not medically appropriate for the patient, or that no covered standard treatment is more beneficial. The reviewer then considers whether the treatment has been approved by the FDA for the condition, or whether medical or scientific evidence shows it is more likely than not to be more beneficial than standard treatments.

The model allows a faster review if your doctor certifies in writing that the treatment would be significantly less effective if not promptly initiated. It also applies only when the service is not explicitly listed as an excluded benefit. Your state's version may differ, so follow the instructions in your final denial letter.

Federal law, 42 U.S.C. 300gg-8, says a group health plan or insurer covering a qualified person may not deny or limit coverage of routine patient costs connected with an approved clinical trial. Routine costs are items and services the plan would typically cover for someone not enrolled in a trial.

They do not include the investigational item, device or service itself, or items provided only to collect data for the study. The rule covers phase I through IV trials for cancer or another life-threatening disease or condition that meet federal criteria, and a plan may require you to use an in-network provider if one is taking part in the trial.

Medicare covers some costs for certain clinical research studies. Medicaid programs must cover routine patient costs for beneficiaries participating in a qualifying clinical trial on or after January 1, 2022. If a plan denies routine care because you joined a trial, say so in the appeal and cite the rule that fits your coverage.