A 1992 commercial starts with a young student coloring as the teacher drones on: “Stay within the lines, the lines are our friends.” The girl gleefully draws a stray mark with her crayon. The ad then cuts to a scene of a joyful young woman (presumably the same person several years later) turning off a lined road to go off-roading. The voiceover says: “Isuzu Rodeo… good to go way outside the lines.”
We typically see payers cover evidence-based treatments. But evidence does not always serve as coverage guardrails. Sometimes payers go off-roading, covering experimental or investigational treatments because they are forced off the road of evidence.
FDA accelerated approvals
The FDA sometimes approves drugs with weak evidence of efficacy. Just because the FDA determines a product to be safe and effective does not necessarily mean it is so. Are products that are approved on the basis of surrogate endpoints under the Accelerated Approval Program effective? Time will tell. Payers often cover products lacking more conclusive evidence to avoid noise, appeals, and negative public relations.
But sometimes, payers push back and reject what the FDA approves. Such was the case with Medicare’s National Coverage Determination for Aduhelm. It will be interesting to see which, if any, payers push back against high-priced gene therapies receiving accelerated approval on the basis of surrogate endpoints if their efficacy and durability are uncertain. If approved by the FDA, Sarepta Therapeutics’ gene therapy for Duchenne muscular dystrophy is one to watch.
State mandates
Insurers are regulated by the states, which can mandate certain types of coverage. Some state mandates are less about evidence than appeals to populist sentiment. In 2011, the Connecticut General Assembly proposed mandating coverage of breast cancer screening by thermography in both individual and group policies. Maybe some politicians in Connecticut thought they knew something that payers and other states didn’t know. Insurers view thermography as experimental due to insufficient evidence and, therefore, not medically necessary. And in a 2019 safety communication, the FDA wrote: “There is no valid scientific data to demonstrate that thermography devices, when used on their own or with another diagnostic test, are an effective screening tool for any medical condition, including the early detection of breast cancer or other diseases and health conditions.” Fortunately, the thermography mandate was not enacted in Connecticut. It is self-evident but important to remember that mandates are political.
Court orders and legal settlements
Court orders and legal settlements may compel payers to cover care that is experimental or investigational. For example, in 1994, Health Net lost a case for denying a bone marrow transplant (BMT) for a woman with breast cancer. The verdict apparently led other insurers to reverse denials of coverage of BMT for breast cancer. The prosecuting attorney said, “The jury spoke and because of this case many people are being helped.” Actually, this case may have hurt many people. Randomized clinical trials in the 1990s showed that high-dose chemotherapy was not more effective than standard-dose chemotherapy in treating breast cancer, and some of these trials showed that treatment-related death rates were greater for high-dose chemotherapy. Health Net’s president was right back in 1994 when he said, “We maintained, and still do, that the procedure in question would have potentially done more harm than good to the patient.”
External reviews
In theory, payers do not cover experimental or investigational treatments. But members of nongrandfathered plans can appeal denials for treatments that payers deem experimental or investigational, including drugs, devices or therapies not approved by the FDA or those not approved for a particular indication. Determinations by an independent third party, regardless of whether evidence published in peer-reviewed medical literature is conclusive, can be somewhat subjective. Even so, if the plan’s denial is overturned on grounds of medical necessity, then the plan must cover such care.
Administrative overrides
MCOs and PBMs crave predictability and strongly prefer to align coverage decisions to the formulary or medical policies. All too often, self-insured employers “request” coverage for care that is initially denied. The resulting administrative override not only breaks the rules, but circumvents the normal process for making exceptions to the rules. The hazards of doing so include: A) establishing a new precedent for expanded coverage, B) giving the stop-loss provider the right to deny coverage for the claim, and C) making benefits claims in federal court subject to the de novo standard of review (as opposed to the “Firestone standard,” which presumes the plan’s determination was correct). The safest path is for self-insured employers not to intervene and let the formal appeals processes play out. Despite the risks, some senior executives can’t resist the temptation and push for coverage of care lacking evidence.
Clinical trials
The ACA requires most payers (other than grandfathered plans existing on or before March 23, 2010) to cover routine costs incurred during participation in a federally approved or funded clinical trial. Similarly, Medicare and Medicaid cover routine care costs associated with cancer clinical trials. As a result, payers wind up subsidizing some care associated with experimental or investigational care that may not have otherwise been delivered.
The lines of evidence
For several reasons, experimental or investigational treatments should be limited to clinical trials. Patients benefit from an informed-consent process, an approved protocol, and close monitoring. While real-world evidence is what payers really, really want, payers also want sufficient evidence of safety and efficacy from clinical trials prior to coverage and utilization in the real world. Importantly, manufacturers absorb much of the financial risk.
Payers attempt to make it clear that they don’t cover experimental or investigational treatments. But it is not always clear which treatments are experimental or investigational. And even when treatments clearly lack strong evidence, payers may be forced to cover them. There are economic and clinical downsides to this: Costs and premiums will rise, and some patients may be harmed.
Approvals of treatments that lack strong evidence is a vexing problem that, increasingly, will challenge payers and plan sponsors as they try to stay within the lines of evidence. More and more, payers will be forced off the road of evidence-based care. Such off-roading is neither fun nor safe.
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