Donanemab In Medicare: Quick Reversal Of Alzheimer’s Coverage Limits Doubtful, Lilly Says
Executive Summary
Lilly execs caution that even if FDA approves donanemab in 2023, no uptake of any significance is expected until 2024 and beyond. An accelerated approval would essentially preclude Medicare reimbursement, and a CMS policy change or more persuasive trial data could take some time.
Eli Lilly and Company expects its donanemab to get an accelerated approval from the US Food and Drug Administration in Alzheimer’s disease early next year but is not counting on much in the way of sales during the balance of 2023 because Medicare coverage restrictions will be a continuing challenge, according to company executives.
Lilly has expressed confidence in the past that it will have the data that can convince the Centers for Medicare and Medicaid Services to reconsider its seven-month old national coverage determination for amyloid-directed monoclonal antibodies for Alzheimer’s disease, which restricts Medicare coverage to patients enrolled in a study. (Also see "Lilly Phase III Alzheimer’s Data Will Set Stage For Unrestricted Medicare Coverage Request" - Pink Sheet, 28 Apr, 2022.)
Donanemab has a projected user fee date of 4 February for an accelerated approval, and the company expects to announce results from its Phase III Trailblazer study in mid-2023, but it is unclear when the drug might achieve the conversion to traditional approval that would smooth Medicare reimbursement even somewhat.
The NCD imposes coverage with evidence development restrictions. For drugs with an accelerated approval based on a biomarker and no clinical evidence of cognitive improvement – like Biogen, Inc./Eisai Co., Ltd.’s Aduhelm (aducanumab-avwa) – that means sponsors must conduct an additional randomized clinical trial.
Drugs with a traditional approval and clinical evidence of cognitive improvement can satisfy the CED requirement with a study using real-world data from sources such as a patient registry.
Many stakeholders believe a study using real-world data, though less cumbersome than a clinical trial, would still significantly curtail access to the drugs. (Also see "Medicare Registry-Based Studies For Alzheimer’s Drugs Still ‘Challenging’ Prospect – Former CMS Official" - Pink Sheet, 8 Apr, 2022.)
CMS has pledged to move quickly to reconsider the coverage restrictions imposed by its NCD. Most recently, CMS Administrator Chiquita Brooks-LaSure emphasized the agency is open to considering new data when it becomes available at the Milken Institute Future of Health Summit on 8 December.
‘Our Door Is Open,’ CMS Administrator Brooks-LaSure Says
“We certainly welcome, as new products come to market, manufacturers coming to speak to us and we’ll certainly take in new data,” Brooks-LaSure said. “Our current decision is based on the information available at the time and as new products come to market we’ll take a look and make our determination [about revising the NCD] based on that. … I can’t speak to any specifics but will just say that our door is really open.”
“CMS has said publicly that they can quickly reconsider it if new data becomes available; it certainly is our hope that they stand by that. However, the track record is that most likely it’s going to take quite a bit of time to work through this” – Lilly’s Ann White
However, Lilly cautioned that such a reconsideration may take several months to complete. “We expect extremely limited use of donanemab in 2023 and the reason is that … it really comes down to CMS and the reconsideration process,” Lilly neuroscience president Ann White explained during the company’s 2023 financial guidance presentation on 13 December.
“CMS has said publicly that they can quickly reconsider it if new data becomes available; it certainly is our hope that they stand by that. However, the track record is that most likely it’s going to take quite a bit of time to work through this,” White continued.
“What we’re planning to do is apply for reconsideration upon positive Phase III data in mid-’23 [and] as you’ve heard us say before, we feel very strongly that that data should meet the high level of evidence that CMS is seeking,” she said. After that, “the process forward is less clear.”
White noted that Eisai and Biogen are also likely to get an accelerated approval early next year for their lecanemab in Alzheimer’s, which will increase pressure on CMS to rethink the NCD. (Also see "Lecanemab Data On Benefit In Alzheimer’s Might Satisfy FDA – But What About Medicare?" - Pink Sheet, 30 Nov, 2022.)
Lecanemab might be able to win full approval by the end of September (Also see "Lecanemab Conversion From Accelerated To Full Approval Could Be Among Fastest Ever" - Pink Sheet, 28 Sep, 2022.), and the best-case scenario for other products is that the strength of that data makes CMS reconsider its opinion on the amyloid biomarker in general.
“We certainly hope that over the next month, certainly with two accelerated approvals in early 2023, that we’ll get more clarity from CMS on what the next steps are and when we can expect movement from them,” White said. “As we have more insight and information, we’ll share that for the years beyond.”
Sponsors might also get some help on getting CMS to change the policy from the incoming Republican-controlled House. (Also see "As US House Changes Hands, CMS May Face More Oversight Than FDA" - Pink Sheet, 22 Nov, 2022.)
Reconsideration of NCD For Amyloid PET Scans A Model For Drugs?
CMS’ ongoing reconsideration of its NCD for amyloid PET scans in dementia suggests the process may take at least nine months. That NCD restricts coverage of the diagnostic to patients enrolled in a clinical trial and allows only one PET scan per lifetime.
Lilly is well aware of the process because it has worked within the confines of the NCD in its clinical trials on donanemab and it also markets two imaging agents used with amyloid PET scans – Amyvid and Tauvid. The NCD has been in place since 2013 and CMS opened a reconsideration of it in June. A draft decision is due 16 December and a final decision is scheduled to be issued by 16 March.
As the reconsideration process plays out, Lilly will focus in 2023 on educating physicians about the use of amyloid-directed MABs and helping to build a diagnostic infrastructure to complement treatment, White said.
“Our [medical science liaisons] that we already have out there, as well as our other efforts on education, will really be focused on diagnostic and therapeutic infrastructure,” she explained. Lilly wants to “make sure that the system is ready for these medicines so that as soon as reconsideration occurs, we can make sure that patents get access to these medicines quickly. … We’ve already started working on it and we will continue to do that after approval.”