At the same time, the decision focused on the accelerated approval process by the Food and Drug Administration and highlighted the strength of the CMS – so that it could have an impact on how quickly other new medicines hit the market.
“With this decision, the CMS simply does not say it does not trust Alzheimer’s treatments that have been approved by the FDA through accelerated approval. “It also undermines confidence in the FDA’s approval of traditional medicines more broadly,” said Michelle McMurray-Heath, president and CEO of the Organization for Biotechnology Innovation.
The CMS ordinance applies to all Aduhelm-like medications that target amyloid plaques in the brain that are likely to contribute to Alzheimer’s disease.
Lee Fleischer, chief medical officer at CMS, said the agency “looked into the very unique circumstances of this class of treatment and made a decision that balanced the patient’s benefit potential with the importance of serious unknown factors that could cause harm.”
The agency, which decides what to cover more than 60 million Americans on Medicare, has not sold on drug efficacy and needs additional clinical trials or treatment studies – once they receive FDA approval. At least three similar Alzheimer’s medications are in development. The two move forward with accelerated approval.
“Accelerated approval, as we know it, is gone,” said George Wradenburg, who heads the advocacy group UsAgainstAlzheimer’s. “Medicare believes that it has both the legal authority and the responsibility to ensure that medicines that are covered for Medicare’s population have clinical benefits. “And because accelerated approval is only a ‘reasonable probability of clinical benefit,’ they are not going to release a drug to the medical population that does not show clinical benefit.”
Following Adhelm’s approval – which led to the resignation of three members of the FDA’s Neurological Drug Advisory Panel in protest – the approval process has been further explored. Congress is considering the changes, launched an investigation into Adhelm’s approval, and Oregon has demanded that it not be required to cover drugs that are given the green light by expedited approval.
By definition of coverage, antiamyloid antibody drugs approved by the FDA-accelerated approval process, such as Aduhelm, must undergo additional randomized clinical trials at the FDA or the National Institutes of Health. Eli Lilly has one such drug in the accelerated approval process and said the results of his prospective study should lead to repayment if approved by the FDA – calling the additional requirements “unnecessary”.
But the CMS does not stop there by requesting a new Alzheimer’s treatment for accelerated approval for further study. Those who go through a more intensive, traditional FDA approval process also need to provide more evidence that their medications are effective – causing outrage among lawyers.
Companies such as Genentech, which has an Alzheimer-like drug in the process of being developed and plans to go through the traditional process, still need further – but less rigorous – further research into CMS-sanctioned further approval. Even medicines that “[demonstrate] The effectiveness of the direct measurement of clinical benefit is promising, but does not meet … a reasonable and necessary statute, ”the CMS said. Memorandum of Understanding.
Nevertheless, advocates say that even less rigorous studies – such as patient registers – take some time to recover, and patients may progress to further cognitive impairment during this time, making Audihelm-like medications unavailable.
“The impact of this final decision will be felt beyond Alzheimer’s disease,” said Su Peshin, executive director of the Aging Research Alliance. “Medicare senior officials have launched this aggressive process to care for the beneficiaries who are otherwise required to serve.”
The agency’s actions could also slow down investments in both Alzheimer’s drugs, which promise to go through the long, traditional process of FDA approval, and in the treatment of other complex diseases.
“If you were a drug manufacturer and had the choice to invest in therapeutic areas, you would be investing in an area where – even demonstrating clinical benefits and traditional FDA review and approval – you would still have to go through a CMS. Research to demonstrate the same result in the case of real-world populations? Why would you do that? ” Said Wradenburg.
The CMS disagreed with comments that this would stifle innovation in other Alzheimer’s drugs.
“We believe that defining the criteria by which future therapy for Alzheimer’s disease can be met will actually contribute to innovation. “The final decision of the CMS gives the public a clear definition of where the goal line is, what constitutes success in the endeavor (and therefore meets the ‘reasonable and necessary’ standard required by law),” the agency’s decision memorandum said.
Chris Mickins, a former employee of the Department of Health and Human Services who is now a policy analyst for Raymond James, said it was too early to say what impact the CMS decision would have on investing in new medications. He points to an agency that calls this class of drug “unique.”
“The CMS shows that it has a tool in the toolbox,” he said. “In order for people to really change their investment behavior, you will need one or two more examples in the future.”
The agency, however, “does not specify what will be sufficient in terms of a new future product. So it’s a bit like a negative statement, “said John Murphy, chief policy officer at the Biotechnology Innovation Organization. “You are leaving the industry in question, whether there will be any new product or product class subject to it at the discretion of the CMS. This is where you will find some coldness about investing in new categories of disease or new therapeutic areas. ”
More than six million Americans have Alzheimer’s disease, according to the Centers for Disease Control and Prevention, and mortality from this disease has increased by 33 percent from 1999 to 2019. It is the seventh leading cause of death in the US, according to the NIH.
The CMS proposal was the subject Intensive advocacyAnd the CMS reviewed more than 10,000 comments, including thousands of letter forms. About 65 percent of respondents agree that there was insufficient evidence to cover anti-myeloid antibody drugs such as Aduhelm.
Aduhelm, manufactured by Biogen, was the first treatment aimed at slowing down cognitive impairment. However, there have been mixed results on whether the drug effectively reduces memory loss, and some patients have experienced swelling or bleeding in the brain. Whether medications that target amyloid plaques in the brain can reduce cognitive impairment is also a matter for consideration.
“Now that the CMS has made that decision, I can say it was a defining moment for the White House and they went through the flying colors,” said Diana Zuckerman, president of the National Center for Health Research, who called the move science-based. “Everything that has happened in the last year or so, or maybe even more than a year, has sent a message to the industry and patient groups, as well as to the FDA, that standards have been lost too much.”
Narrow coverage of Aduhelm has sparked debate about the viability of future treatments
