FDA approves much-debated Alzheimer’s drug panned by experts – Honolulu, Hawaii

Honolulu, Hawaii 2021-06-07 12:00:00 –

Washington >> Government health officials today approved the first new drug for Alzheimer’s disease for the first time in about 20 years.

The Food and Drug Administration said it has approved a drug developed by Biogen for patients with Alzheimer’s disease.

It is the only drug that US regulators have said is likely to treat underlying illnesses rather than managing symptoms such as anxiety and insomnia.

This decision can affect millions of older Americans and their families and is certain to cause disagreements among doctors, medical researchers, and patient groups. It also has a wide range of implications for the criteria used to evaluate experimental therapies, including those that show only gradual benefits.

A new drug co-developed by Biogen with Eisai in Japan did not reverse mental weakness, but only delayed it in one study. The drug, named Aduhelm, is given by intravenous drip every four weeks. ..

In a statement, the FDA’s chief executive admitted that “residual uncertainty” surrounds the drug, but Aduhelm’s ability to reduce harmful plaque clumps in the brain is “to delay dementia.” Expected. “

Under the terms of approval, the FDA requires pharmaceutical companies to conduct follow-up studies to confirm benefits to patients. If the study shows no efficacy, the FDA may withdraw the drug from the market, but the FDA rarely does.

Biogen didn’t immediately reveal the price, but analysts estimate that the drug’s annual treatment costs will be between $ 30,000 and $ 50,000.

A preliminary analysis by a group found that the value of the drug should be increased from $ 2,500 to $ 8,300 per year, based on the “small overall health benefits” suggested by a company study. It was. The non-profit Institute for Clinical Economic Review added that “price is too high” if follow-up studies do not confirm the benefits of the drug.

About 6 million people in the United States and many more around the world have Alzheimer’s disease. Alzheimer’s disease gradually attacks areas of the brain that are needed for memory, reasoning, communication, and basic day-to-day operations. In the final stages of illness, suffering people lose the ability to swallow. The global burden of the disease, the most common cause of dementia, is expected to increase as millions of baby boomers move into their 60s and 70s.

Aducanumab (pronounced “add-yoo-CAN-yoo-mab”) helps remove Alzheimer’s disease-related protein called beta-amyloid from the brain. Other experimental drugs have done it before, but there was no difference in the ability of the patient to think, care for himself, and live independently.

The pharmaceutical pipeline in the pharmaceutical industry has been messed up for years with billions of dollars worth of research funding for failed treatments for Alzheimer’s disease. FDA approval could bring back investment in similar therapies previously shelved by pharmaceutical companies.

New drugs are made from living cells and need to be given by IV in a clinic or hospital.

Researchers do not fully understand the cause of Alzheimer’s disease, but it is widely accepted that the plaques in the brain targeted by aducanumab are only one factor. There is increasing evidence that family history, education, and chronic illnesses such as diabetes and heart disease may be involved.

“This is just one piece of the puzzle. I think we need to investigate and expand all these other options,” said Mayo Clinic’s dementia expert at Biogen and other pharmaceutical companies. Dr. Ronald Petersen, a consultant, said.

Patients taking aducanumab had a 22% slower decline in thinking ability than patients receiving quack treatment.

However, it meant a difference of only 0.39 in the 18-point score for cognitive and functional abilities. And it is unclear how such metrics are transformed into practical benefits such as greater independence and the ability to remember important details.

The FDA’s review of this drug has sparked a long-standing debate about the criteria used to evaluate treatments for difficult-to-treat conditions. On the one hand, a group of people with Alzheimer’s disease and their families say that any new treatment — even the slightest benefit — is worthy of approval. However, many experts say that approval of this drug is dangerous. It sets a clear precedent and warns that it may open the door to the treatment of suspicious interests.

Approval was given despite a rigorous November evaluation by an external committee of FDA neurological experts. The group voted “no” to a series of questions about whether reanalytical data from a single study submitted by Biogen showed that the drug was effective.

Cambridge, Massachusetts-Biogen discontinued two studies of the drug in 2019 after disappointing results suggesting that aducanumab could not meet its goal of delaying the mental and functional decline of patients with Alzheimer’s disease. ..

A few months later, the company reversed its policy and announced in a new analysis of one study that the drug was effective at high doses and the FDA advised that it needed to be reviewed. Company scientists said the initial failure of the drug was due to some patients who did not receive high enough doses to delay the disease.

However, dose changes and post-company analysis have made it difficult to interpret the results, creating skepticism among many experts, including members of the FDA committee.

The FDA does not have to follow the advice of outside panelists and has previously ignored their views in making similar high-profile drug decisions.

In 2016, authorities gave the go-ahead for the first drug for muscular dystrophy, even though an expert committee voted that a study of 12 patients did not show efficacy.

In that case, the internal dispute between the institution’s scientists was finally decided by Dr. Janet Woodcock, the institution’s longtime drug director. Janet Woodcock is currently the commissioner. Her official The decision concluded that it was “reasonable” that the drug could benefit some patients. Approximately five years later, the benefits of this drug have not yet been confirmed by FDA-mandated follow-up.

Approximately 600 US healthcare professionals will participate in biogen drug research, with more healthcare professionals planning to launch the drug. Many practical questions remain unanswered: how long will the patient benefit? Does the doctor decide how to discontinue the drug? This drug is for patients with more advanced dementia Is it effective?

With FDA approval, adducanumab is guaranteed to be covered by virtually every insurer, including Medicare, a government plan for the elderly that covers more than 60 million people.

Both the FDA and Medicare are forbidden to consider costs when considering new drugs and treatments.

Even qualifying for a drug can be expensive. It has only been tested in people with mild dementia due to Alzheimer’s disease, or in less severe conditions called mild cognitive impairment. A brain scan, which costs more than $ 5,000, may be required to confirm the diagnosis. Insurers, including Medicare, do not cover scans because their benefits are unknown, but that could change if scans are the gateway to treatment. Additional scans are needed to monitor potential side effects such as swelling and bleeding of the brain.

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