On December 9, CMS made a splash by posting a letter to Medicare Advantage plans, that they cannot classify an ALS drug (QALSODY) as "investigational" and therefore outside of covered drug benefits. The drug is approved under FDA accelerated approval.
Here's an ALS press release,
Here's the CMS letter,
https://www.als.org/sites/default/files/2024-12/HPMS%20Memo_Qalsody%2012-9-24%201.pdf
The CMS letter states that CMS does NOT MAKE A DISTINCTION based on regular or accelerated approval. (This has also been a talking point for former FDA commissioner Scott Gottlieb.)
Right? Wrong? Right...
When I read that "CMS does not make a distinction" based on accelerated approval, I thought, that's not literally true because the NCD on amyloid Alzheimer drugs treats accelerated approval and regular approval separately. I mis-remembered. Here's the policy:
The policy doesn't distinguish due to "accelerated approval" but rather, distinguishes because of drugs approved based on "surrogate endpoints." OK, well, "Surrogate endpoints" is almost a synonym for "accelerated approval," but not quite. In this NCD, the impact is that surrogate endpoints drugs must be covered only under a new RCT under an IND at the FDA. That's a high bar. On the other hand, drugs approved under "efficacy...clinical benefit" must only enroll in a simple registry.
So CMS doesn't literally distinguish, in this NCD, based on accelerated approval, although that was exactly the real impact.
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This Dec 9 CMS letter re Medicare Advantage coverage reminds us there have been several years of complaints, policy-making, and new regulations trying to better define and enforce Med Adv coverage:
https://www.discoveriesinhealthpolicy.com/2024/12/cms-edits-medicare-advantage-coverage.html
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Separately, CMS issued a Part D proposal regarding classifying obesity drugs like Wegovy as payable - this was a big splash and represents a position proposed by Biden and to be handled and finalized in the spring by DJT. E.g. see here.
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CMS can use NCDs to deal harshly with drugs approved on "surrogate endpoints," although we should remember there are some extremely well accepted ones (e.g. vanishing of HIV viral titers, etc.). That is, outside of the amyloid or ALS areas, a range of "surrogate endpoints" have been associated with regular, traditional approvals:
For example, a drug for Cushing's disease (elevated cortisol) that causes cortisol to fall to normal levels.
