Just days after President Donald Trump unveiled his vague and widely panned plan to curb pharmaceutical prices, the Food and Drug Administration quietly approved a biosimilar to compete with a drug that has cost taxpayers at least $50 billion over the past quarter century.
Alas, this follow-on biologic will arrive long after use of the original drug, which prevents anemia in cancer and dialysis patients, has faded. A newer, still-patented version of the same protein, whose only improvement is the need for less frequent dosing, has already superseded most uses of the original product.
In carrying out the president's wishes, FDA Commissioner Dr. Scott Gottlieb vowed to crack down on the "shenanigans" that impede generics and biosimilars from coming to market. If he wants a primer on those shenanigans, he need look no further than the sordid saga behind Epogen/Procrit (they're the same drug, epoetin alfa, and are marketed by Amgen and Johnson & Johnson for dialysis and oncology, respectively) and the newly approved biosimilar Retacrit, which is manufactured by Pfizer.
The story reveals everything you need to know about how industry lobbyists and lawyers, working through Congress, the FDA and the patent system, have undermined biosimilars' money-saving potential. It also shows why the torrent of words pouring from HHS, the FDA and the White House won't affect the soaring cost of the most expensive products in healthcare—biologic specialty drugs.
The stakes are growing rapidly. Biologics, man-made versions of naturally occurring or bioengineered proteins, now make up the lion's share of specialty drugs that constitute nearly half the $450 billion U.S. drug market. Their soaring prices are driving higher drug spending. Most new chemotherapy regimens are priced at $20,000 or more a month. Drugs for rare cancers are edging close to $1 million a year.
If you buy the argument that those high prices are necessary to promote innovation (I don't, but that's a different story), then your only hope for long-term relief is to suck it up, pay the bill and patiently await patent expiration and the arrival of biosimilar competition.
You will wait a long time. The U.S. has set patent life at 20 years from the time of filing, which can occur as much as a decade before a new drug receives FDA approval. To offer incentives for investment, the 2010 Biologics Price Competition and Innovation Act guaranteed innovators 12 years of exclusivity no matter when the patent expires.
What happened in this case? The original Epogen/Procrit patents were filed in the mid-1980s. The drug received FDA approval in 1989. Three decades passed before competition arrived.
How did that occur? First, patent litigation held up biosimilar development. Pending lawsuits will further delay Retacrit's marketing.
Second, after the BPCIA created a pathway for biosimilar development, the FDA established much stricter efficacy and safety standards for biosimilars than for other generics. In fact, the agency went well beyond the standards set for the original biologic's approvals.
To meet the FDA's new standards, Pfizer wound up testing Retacrit in 2,458 patients who were enrolled in two randomized controlled trials. The drugmaker needed to show it had the same effect on patients as Epogen/Procrit and didn't cause an unacceptable level of immune reactions, which is a problem with all versions of the drug. The original protein's trials, conducted from 1987 to 1989, had involved just 358 patients.
Even with seven times more data, the FDA didn't grant Pfizer the right to sell its drug as interchangeable with Epogen/Procrit. Instead, it will be marketed like a new drug. Biosimilars (the FDA has now approved five) are generally priced around 85% of the original, not the 20% to 40% typical of most generics.
So, Dr. Gottlieb, unless Congress changes the law or you change FDA guidance on biosimilar development, the shenanigans will continue and patients and payers can expect to pay exorbitant prices for biologics long after their patents have expired.