When step-therapy steps your patient into a rabbit hole

Neil Baum, MD

I had an older female patient with overactive bladder, and I prescribed a a beta-3 adrenoceptor agonist, mirabegron. I received a response from her insurance company that this was a “third-tier” medication and suggested that I wrote for an anticholinergic medication. I wrote a letter (with the assistance of AI) which explained that anticholinergic medication in the elderly is associated with cognitive impairment. I soon received permission that the insurance company would approve the third-tier medication. This process required more than one hour on my part and there was nearly a month delay for the patient to receive authorization for the more expensive medication. This situation is a common problem impacting most physicians that results in using uncompensated time or even pajama time to obtain authorization and delays treatment for patients. This blog will discuss step therapy and suggestions for helping our patients receive the treatment\medication that they deserve in a timely fashion.

Step therapy imposed by insurance companies requires a patient to try less expensive medication first before they can receive a more expensive medication that will be covered by the insurance company. We have labeled this situation as “fail first” therapy. If we read between the lines, this forces patients to "fail first" on the cheaper option before moving to the preferred treatment. Yes, this is a cost-saving measure by the insurer but is an expensive administrative task for physicians and may cause a significant delay in patients receiving proper treatment.

Step therapy protocols not only delay necessary treatment but can lead to adverse reactions that ultimately increase rather than lower health care costs. As in the case of the patient with overactive bladder, using the less expensive medication can result in the unwanted side effect of cognitive decline.

Another example of the disadvantage of step therapy occurs when a patient changes insurance companies, they may be required by their new insurer to go off a successful treatment and take a less effective medicine that is not effective or accompanied by adverse effects.

Step-therapy works as cost saving to insurance companies but the “fail first concept” occurs at the detriment to the patient.

"Fail first" concept:

Patients must demonstrate that a lower-cost medication is not effective or is associated with adverse effects for their condition before allowing coverage for the medication their doctor first prescribed.

Cost control:

The main goal of step therapy is to reduce healthcare costs by encouraging the use of less expensive drugs.

Formulary tiers:

Insurance plans often have drug formularies with different tiers. Step therapy dictates which tier a patient must start with before moving to a more expensive medication.

Potential concerns:

Step therapy can delay access to necessary medications, potentially impacting patient health, especially if the initial "step" drug is not suitable for their specific needs as in the overactive bladder patient I described.

Perhaps the government will recognize that the fail-first or step-therapy is not in the best interests of our patients. The Safe Step Act introduced in Congress on March 3, 2023 and was reintroduced in the House and Senate in 2024. This legislation has bipartisan support. The bill requires a group health plans to make readily available a clear process for a patient to request an exception to the use of step therapy. The bill further specifies timelines under which insurance companies must respond to requests for an exception of step therapy. Patients also can find out whether their state has passed legislation related to step therapy by going to the StepTherapy.com website. There are national organizations that have supported legislation, which ensures step therapy is based on current clinical data and that it is available to the patient in a transparent manner in a non-medical jargon. This legislation strikes a fair balance between cost containment and the health of the patient.

There are five automatic exceptions from step therapy that states may use that prevent abuses by the insurance industries. The five exceptions include: (1) the required prescription drug is contraindicated or will likely cause an adverse reaction or physical or mental harm to the patient; (2) the required prescription drug is expected to be ineffective based on the known clinical characteristics of the patient and the known characteristics of the prescription drug regimen; (3) the patient has previously tried the required prescription drug while under their current or a previous health insurance or health benefit plan, or another prescription drug in the same pharmacologic class or with the same mechanism of action and such prescription drug was discontinued due to lack of efficacy or effectiveness, diminished effect, or an adverse event; (4) the required prescription drug is not in the best interest of the patient, based on medical necessity; and (5) the patient is stable on a prescription drug selected by their health care provider for the medical condition under consideration while on a current or previous health insurance or health benefit plan.

Bottom line: Step therapy requires so much extra work by the physician and the medical staff and it takes time away from patient care. We must stand up to insurance companies and put the focus back on the patient and not participating in bean-counting by the insurance companies.

Neil Baum, MD, a Professor of Clinical Urology at Tulane University in New Orleans, LA. Dr. Baum is the author of several books, including the best-selling book, Marketing Your Medical Practice-Ethically, Effectively, and Economically, which has sold over 225,000 copies and has been translated into Spanish.