With the demise of the Alzheimer’s drug aducanumab after the FDA’s accelerated approval and CMS’s actions to raise Medicare premiums in anticipation of huge demands based on its proposed pricing, we may now face another similar situation and that is with a drug for amyotrophic lateral sclerosis (ALS). After a 7-2 favorable vote by the FDA’s advisory committee a few days ago (after it reviewed newly mined study data, which turned the tables on the 6-4 negative vote by the committee in March 2022), we must now see how the FDA will react to the new “lease on life” of the product.
Clearly, there are differences between the two products and disease states. There were other approved treatments for Alzheimer’s disease, though aducanumab was to be the real advance. There are no such products approved to treat ALS. The drug’s sponsor went so far as to agree to withdraw the ALS product from the market if it confirmatory study (required for accelerated approval) was not positive. No question, ALS is a horrendous disease, and we need hope where there is none for ALS patients, but other questions come to mind.
And that puts us back to the reimbursement and drug cost issues that sunk aducanumab as well as the time it would take to complete the confirmatory study. There had been talk of CMS lowering the cost for Medicare part B premiums it had raised in anticipation of the significantly increased cost associated with the Alzheimer’s drug, but it did not do that; instead, Medicare said undoing the premium increase mid-year would be too difficult and indicated that it would revisit lowering the premium costs for the next calendar year. In addition, CMS’s agreement is to cover drug costs only for patients who were enrolled in the Alzheimer’s confirmatory clinical study.
Now, here we are, back at the foot of the mountain, but with significantly fewer ALS patients than Alzheimer’s patients, waiting to see whether the FDA will take the committee’s advice. If the FDA does approve the product, then we will be waiting to see what the medication costs will be. As we get further up the foothills, the next climb will be to see what CMS does with this product in terms of requirements for reimbursement. Maybe we should be glad that CMS did not reverse the Part B premium increases? Hopefully we will have a better outcome.