Amid the fierce backlash over the $56,000 annual price tag for its controversial new Alzheimer’s drug, Biogen is emphasizing another rationale beyond the benefits of reducing the staggering toll of the illness: Other novel medicines using similar technology are much more expensive.
Biogen’s medication, Aduhelm, is a monoclonal antibody made from the immune cells of healthier older people and is administered in monthly intravenous infusions. So for guidance on what to charge, Biogen spokeswoman Allison Parks said, the company looked to pricing of other antibody medicines in wide use that rely on infusions.
Cancer immunotherapies like Merck’s blockbuster Keytruda cost upward of $150,000 a year, and anti-inflammatory medicines such as AbbVie’s best-selling Humira cost more than $70,000 a year. In comparison, Biogen says, Aduhelm is reasonable.
“There is no perfect analog in this space, but we have priced Aduhelm at roughly 1/3 the level of cancer immunotherapies and roughly 25% below the average level of psoriasis biologics” such as Humira, she said in an e-mail.
Critics say the comparison would make sense if Biogen had submitted compelling evidence to the Food and Drug Administration that Aduhelm worked in clinical trials, as the makers of Keytruda and Humira did. But it didn’t.
“Keytruda is an expensive drug, but for patients with non-small cell lung cancer, you have clinical data showing it improves survival,” said Brian Skorney, an analyst at the investment bank Baird who contends drug regulators shouldn’t have approved Aduhelm. “The concern here with Aduhelm’s price is there’s no argument that it does for Alzheimer’s patients what Keytruda does for cancer patients.”
The FDA approved Aduhelm on June 7 over the objections of the agency’s scientific advisory committee, including three members who quit in protest, saying Biogen had failed to demonstrate its effectiveness. One of the most closely watched experimental drugs in recent history, it had generated contradictory results in two late-stage clinical trials. In one study, people given the drug declined 22 percent more slowly on average than those who received a placebo. The other trial failed to reach its goal.
Many analysts and scientific experts were stunned by Aduhelm’s approval, which came amid pressure from families of people with Alzheimer’s and their advocates. The FDA cleared the drug under a less common “accelerated approval pathway” reserved for medicines that fill a serious unmet need.
The FDA typically uses this approach when regulators are uncertain about the clinical benefits of a drug and rely on another yardstick that suggests it would help patients. In this case, that was Aduhelm’s reduction of a sticky substance called amyloid that clumps into plaques in the brains of people with Alzheimer’s. That reduction, Biogen said this week, is “reasonably likely to predict” that the drug will benefit such patients.
Considering the “unbearable expense” of treating roughly 6 million Americans with the most common cause of dementia, Biogen believes that $56,000 “reflects the overall value this treatment brings to patients, caregivers and society,” Parks said.
Critics, including drug-pricing experts, prominent scientists, a high-ranking insurance executive, and several lawmakers in Washington, vehemently disagree.
They say the evidence that Aduhelm was effective in clinical trials was muddled at best and that the drug is worth only a fraction of $56,000, if anything. They also say it remains an open question whether removing amyloid plaques from the brains of people with Alzheimer’s will slow cognitive decline.
“Based on the evidence we have today, this drug is nowhere near to being that effective, if it’s effective at all,” said Dr. Steven Pearson, president of the Institute for Clinical and Economic Review, or ICER, an influential Boston drug-pricing watchdog group.
Pearson’s group recently tried to calculate a fair price for a hypothetical drug that didn’t cure Alzheimer’s but kept it from progressing in patients with early symptoms. ICER concluded that such a drug would be a breakthrough and could command $50,000 to $70,000 a year, based on an estimated $500 billion in annual costs to the US health care system as a result of the mind-ravaging disease.
But in a May 7 draft report, the watchdog group said that Biogen had generated “insufficient evidence” that its medicine would fit the bill, if the FDA approved it, as the agency did four weeks later. In fact, ICER calculated that Biogen’s drug would deserve an annual price tag of only $2,500 to $8,300, based on the conflicting clinical trial results and evidence that it can cause worrisome side effects, including brain swelling that usually didn’t result in serious symptoms.
Many drug companies have charged jaw-dropping prices for revolutionary new medicines in recent years, including one gene therapy from Novartis that costs $2.1 million for a one-time treatment for a rare inherited disease. But Skorney, the Baird analyst, said that Biogen may have “poked a sleeping bear” by charging $56,000 for Aduhelm amid widespread skepticism that it works.
The price tag, he said, could lead Congress to pursue drug-pricing reforms, including seeking authority to negotiate the costs of medicines on behalf of the government-funded Medicare program, whose beneficiaries make up the bulk of patients with Alzheimer’s.
Senators Elizabeth Warren, Democrat of Massachusetts, and Bill Cassidy, Republican of Louisiana, this week called on Congress to investigate the implications of Aduhelm on the federal budget. Analysts have predicted that Medicare and its enrollees, who pay a share of their prescription drug costs, will spend anywhere from $5.8 billion to $29 billion on the drug annually, which could be more than what the federal government spends on some entire agencies.
Biogen and its Japanese business partner, Eisai, said in a news release Wednesday that they expect to market the drug to 1 million to 2 million Americans who have mild cognitive impairment, although the FDA drug label doesn’t limit it to those patients.
Biogen also said it was working with the Connecticut-based insurer Cigna and the Veterans Health Administration on “innovative access agreements” for patients that those organizations serve, but Biogen hasn’t provided details. A spokesman for the Veterans Health Administration said Friday he couldn’t immediately provide any information. Cigna did not respond to inquiries.
Michael Sherman, the chief medical officer for Point32Health, the insurance company formed by the recent merger of Tufts Health Plan and Harvard Pilgrim Health Care, agreed that the pricing of Aduhelm could fuel longstanding calls for the government to limit drug prices, anathema to the pharmaceutical industry.
His company, the second-largest health insurer in Massachusetts, is carefully weighing whether to cover Aduhelm because it is so expensive and experts whom Point32Health consulted have “uniformly ... been negative about their belief in the efficacy of the drug,” Sherman said in an e-mail.
Biogen, Sherman went on, has demonstrated that the current drug approval process “can create a blank check when it approves a drug for which there is no direct competition. That Biogen has chosen to fill it in with an unconscionably large number makes that point in a way that invites a strong regulatory response.”
Even the Alzheimer’s Association, which enlisted celebrities such as Samuel L. Jackson in a public campaign that lobbied the FDA to approve Aduhelm, has broken with Biogen over the price.
Joanne Pike, chief strategy officer for the nonprofit association, said Thursday that the group recognizes Aduhelm is not a cure but believes it will benefit patients and lead to better drugs.
“The association does not determine drug costs, but we’re adamant that the treatment be affordable and accessible,” she said. “The price as it is today is unacceptable.”
Jonathan Saltzman can be reached at firstname.lastname@example.org.