Aduhelm is the first drug to be approved by the FDA that claims to treat Alzheimer’s disease rather than solely address its symptoms. However, since its approval earlier in June, the decision has been met with much scrutiny and continued reports of unusual events. Here’s the story so far:
June 7th: Aduhelm is approved with a $56,000 annual list price
Aduhelm is the first drug in 18 years to be approved by the FDA for the treatment of Alzheimer’s disease.
The drug was approved via the accelerated approval pathway, with an additional requirement for the drugmaker to conduct a follow-up study confirming Aduhelm’s benefits to patients.
Aduhelm works by clearing beta-amyloids from the brain but it is not the first drug that has been developed to do so. Previous drugs were not reported to deliver significant benefits to patients, such as improving the patient’s ability to think, care for themselves or live independently, and thus were not approved.
June 11th: 3 FDA advisers resign over the approval
Three FDA advisers resign in protest of the Aduhelm approval which was granted despite FDA’s own science advisors almost unanimously not supporting it:
“The committee had found that the evidence did not convincingly show that Aduhelm could slow cognitive decline in people in the early stages of the disease — and that the drug could cause potentially serious side effects of brain swelling and brain bleeding. None of the 11 members of the committee considered the drug ready for approval: Ten voted against and one was uncertain.” – The New York Times
June 25th: Investigation into Aduhelm’s approval announced
Following reports of off the books meetings between the drugmaker’s and FDA staff, Carolyn B. Maloney, Chairwoman of the Committee on Oversight and Reform, and Frank Pallone Jr., Chairman of the Committee on Energy and Commerce announced that they will be investigating the approval of Aduhelm.
Later, on July 12th, a letter informing the CEO of Biogen of the investigation is sent. One of the requests made within the letter is for the drugmaker to submit all of the dates, times, notes taken and materials prepared for all meetings between them and the FDA.
June 30th: ICER suggests a fair price for Aduhelm would require an 85% – 95% discount on the annual listed price
The Institute for Clinical and Economic Review (ICER) assessed the clinical effectiveness and value of Aduhelm in the treatment of Alzeheimer’s disease. Their report concluded:
“Accepting the uncertainty associated with the positive estimate obtained from pooled data from both pivotal trials, the revised report finds the price range needed to reach standard cost-effectiveness thresholds is $3,000-$8,400, representing an 85%-95% discount from the announced list price” – ICER
July 8th: Aduhelm approval is backtracked
The FDA backtracks its decision to label Aduhelm for use in all Alzheimer’s patients. The updated label states that the drug should only be used to treat patients with mild cognitive impairment due to Alzheimer’s disease or mild Alzheimer’s dementia – the patient population that was studied across the three Aduhelm clinical trials that supported approval.
July 9th: Expert suggests that costs of Aduhelm could be as high as $2 billion per year, not $56,000
Experts investigated the packaging of drugs. They reveal that drug packaging does not provide much flexibility in terms of size. One package often contains more substance than is required to administer a single dose, and thus leads to high quantities being wasted over time. The report estimates that packaging inefficiencies could result in over $2 billion more expenditure on the drug.
July 9th: FDA acting commissioner launches independent investigation into Aduhelm approval
On July 9th, Janet Woodcock, FDA acting commissioner requested that the Office of Inspector General conduct an independent investigation into the regulator’s decision. Although Woodcock admits concerns have been raised, she reassures that she has ‘tremendous confidence’ in the leadership at the FDA.
July 13th: Push for pricing reforms strengthens, Aduhelm used as a primary example
A letter requesting the empowerment of Medicare and the Department of Health and Human Services is sent to the congress. The call for support regards 3 key points:
- empowerment for drug price negotiations,
- capping price increases to the rate of inflation,
- and redesigning Medicare part D to lower prices and costs for beneficiaries and taxpayers.
The letter, signed by 39 groups including Families USA and Patients for Affordable Drugs, uses Aduhelm to exemplify why change is necessary:
“the current system, in fact, is (…) yielding the development of high-cost, but clinically-marginal drugs. A current and deeply troubling example comes in the form of Aducanumab (marketed as “Aduhelm”), a recently approved drug to treat Alzheimer’s disease.”
Summary
The approval of Aduhelm has triggered a yet another pressure on tightening drug pricing legislation in the US. Similar initiatives could be observed when other highly priced drugs have been introduced to the market, such as Gilead’s Sovaldi, or large price increases were put in place, such as Turing’s 5,000% rise in the price of Daraprim.
Although the short-term gains for these companies could be fruitful the long term impacts may not be as positive for the wider pharmaceutical industry due to the resulting reductions in pricing freedom.