Fast-track Too Fast? 

Medicare Part B premiums will remain the highest they’ve ever been for the rest of 2022. CMS made efforts to reduce Part B premiums in response to the drama surrounding Biogen’s Alzheimer’s Disease drug Aduhelm, but operational and legal barriers prevented such efforts from succeeding.

Biogen’s Timeline

Within the past year:

• Jun ’21: The FDA granted Aduhelm accelerated approval despite its shaky efficacy and strong recommendation from an independent review board not to approve it.

• Jul ’21: FDA calls for a federal investigation into Aduhelm’s approval. FDA narrows drug’s target population to those with mild cognitive impairment. Prestigious medical centers like Mount Sinai and Cleveland Clinic announce they’ll not have Aduhelm on their formulary.

• Nov ’21: CMS increased Medicare Part B premiums by 15% from $148.50 to $170.10, the largest increase in history. This was done to proactively set aside money to pay for Aduhelm, previously set at $56,000 per year.

• Dec ’21: Biogen slashes drug price in half from $56,000/year to $28,000/year after continuous complaints from hospitals (and much of the medical society) that the drug’s high cost was not worth its benefits.

• Jan ’22: Medicare restricts coverage of Aduhelm to patients in clinical trials (read: this drug isn’t for everyone with Alzheimer’s). HHS Secretary Xavier Bercera says CMS will review reducing Part B premiums, given the drug’s price was slashed in half and the treatment population restricted.

• May ’22: Medicare Part B premiums will remain unchanged for the rest of the year and any savings will be applied to 2023 premiums. Legal and operational hurdles (as you’d expect) prevented CMS from reducing premiums mid-year.

My Thoughts

The FDA’s accelerated approval of Aduhelm has caused so much drama on the insurance side of things. It makes you wonder if the accelerated approval track needs a second look or a revamp. I say, “it does.”

The accelerated approval program fast-tracks drugs that meet unfulfilled needs for hard-to-treat diseases like cancer and Alzheimer’s. These drugs get to the market faster than if they followed the regular approval route. As a result, the accelerated approval program is becoming increasingly popular. Last year, over 20% of the 50 new drugs approved came from this program.

The fast track works by allowing the drug’s approval to be based on surrogate endpoints. These are endpoints that, by proxy, are likely to show the drug works clinically, avoiding the need for clinical results right away (but you still have to run a confirmatory trial after approval to show the drug actually has clinical benefits).

For Aduhelm, the decrease in amyloid-beta plaque was a surrogate for improved cognitive function. The amyloid-beta plaque alone is not itself a measure of clinical benefit. But, studies actually showed that there was minimal cognitive benefit. The FDA ended up granting Biogen nine years to run confirmatory trials that their drug works in the clinical setting.

Nine years is a long time for a drug to be on the market without confirmatory results showing that the drug works clinically. The timeline needs to be shortened to raise the standards of the fast-track program. At the end of the day, it’s not the doctor taking the drug—it’s the patient. Patients deserve to know that what they’re taking is safe and clinically effective. 

While the fast-track program needs to be more exclusive, it’ll be difficult for the FDA to add stricter requirements for the program. From my experience researching healthcare, once stakeholders get a taste of something sweet (fast-track approval with long confirmatory trial timelines), it’s extremely difficult to take that sweetness away, whether due to operational barriers or hard-core lobbying (which Pharma does well at).

Lastly, Eli Lilly’s Alzheimer’s drug donanemab functions similarly to Aduhelm. Lilly will also apply for fast track approval, given the reduction in amyloid-beta plaques (surrogate endpoint). I think it’s highly likely the FDA will approve it, given it approved Aduhelm. It’s the surrogate endpoint that matters in these cases, according to the “rules,” not if there’s a clinical benefit (yet). Either way, the precedent for how the FDA approves these fast-tracked drugs needs to be updated.