Clinical trials for the medication showed no significant improvement in patients.
Last week was a big week for those afflicted with Alzheimer’s disease, as the Centers for Medicare & Medicaid Services (CMS) has released its proposed national coverage determination (NCD) for coverage of medications for treatment of the condition.
There is currently one Food and Drug Administration- (FDA)-approved medication in this class, aducanumab-avwa, which is better known by its brand name, Aduhelm. As a reminder, Aduhelm is an intravenous medication given every four weeks that works to reduce amyloid plaque in the brain. The side effects include headache, confusion, and swelling and bleeding in the brain, known as amyloid-related imaging abnormalities edema (ARIA-E) and amyloid-related imaging abnormalities-hemosiderin deposition (ARIA-H).
The problem with Aduhelm is that the clinical trials that led to FDA approval did show reduction in amyloid plaque, but no clinically significant improvement in the patients’ dementia. The medical community was nearly unanimous in opposing this approval because of the reliance of the FDA on this surrogate marker without any data showing clinical benefit. And you may recall that the drug was priced at $56,000 a year, although the company did lower the price 50 percent in December. In fact, that initial price led CMS to drastically increase the 2022 Part B premiums to cover the potential costs to the Medicare Trust Fund if Aduhelm was widely used.
In the proposed NCD, CMS made the safe choice, stating that these medications would only be covered by Medicare as part of an approved clinical trial. Now, this is not unusual; there are several current treatments with an NCD that require a clinical trial, such as the use of leadless pacemakers or stem-cell transplant for treatment of myeloma. CMS also limited it to administration in a hospital-based clinic, prohibiting doctors from administering it in the office. CMS is also allowing coverage of one PET scan, which is currently not a covered benefit, but it is unclear how that will work; the PET scan will be covered for those enrolled in a trial, but you cannot enroll in a trial unless you have plaque, which is only detected with a PET scan or a lumbar puncture.
With this decision, CMS has managed to make almost no one happy. The drug company is disappointed, because a clinical trial now has to be designed, patients recruited and monitored, and then the data analyzed – and it could be years before there are results, so this will limit usage tremendously. Some advocacy groups are upset that patients won’t have access to a potentially effective drug, understanding that there are risks, but also potential benefit – and as they argue, patients should be able to make the choice of risk and benefit themselves. And most physicians are upset that CMS is even allowing any Medicare money to be spent on a medication with no proven efficacy that has significant side effects and may even cause death. If there is one bright spot to the NCD, it is that CMS is requiring that any clinical trial have enrollment that is diverse and inclusive, and include traditionally underrepresented groups.
It should be worth noting that since this is an FDA-approved medication, it can be ordered and administered to any patient now. Until the final NCD is released, if the patient has Medicare, coverage would be determined by the Medicare Administrative Contractor (MAC) when the claim is submitted. No MAC has made any public comment on how they will determine coverage, but most providers seem unwilling to accept the risk and are choosing not to administer the medication.
The other option would be for the provider to ask the Medicare patient to sign an Advance Beneficiary Notice (ABN) to indicate their willingness to pay out of pocket for the medication and any associated services if coverage is denied. The ABN should itemize every service, including imaging, if necessary, the medication, and the drug administration costs. The claim would then be submitted with the -GA modifier, indicating that a signed ABN is on file. If the patient is covered by a Medicare Advantage plan, the plan should be contacted prior to treatment to determine the correct way to shift potential financial liability to the patient.
Interestingly, if a patient elects to pay out of pocket for the treatment and does develop a complication requiring medical care, Medicare will cover that care in full. While Medicare does not cover continued hospital care provided for a complication that develops during an inpatient admission for a non-covered service, there is coverage for any necessary services to treat a complication that develops after a non-covered service which is provided as outpatient.
We are now in the midst of a 30-day comment period, and CMS will release its final decision in April. In the meantime, U.S. Department of Health and Human Services (HHS) Secretary Xavier Becerra has asked CMS to reevaluate the Part B premium increases it imposed and see if they can be adjusted mid-year, since it is clear that at least for 2022, there will not be a large Trust Fund expenditure on Aduhelm.