It began with a phone call.
“Doc,” my CEO stated, “are you familiar with this physician?”
I inquired further.
“He prescribed a medication that costs $250,000 for a single treatment course.”
“You must be referring to $25,000,” I replied.
“No,” she insisted. “The insurer has already disbursed two hundred fifty thousand dollars.”
I presumed it must be a groundbreaking treatment, a rare life-saving intervention. Then she revealed the name: Acthar Gel. I was taken aback. Acthar: ACTH, adrenocorticotropic hormone, the same pituitary extract utilized by doctors in the 1950s. And now it was marketed as a “specialty biologic” for multiple sclerosis relapses, lupus, and nephrotic syndrome, priced at a quarter-million dollars per treatment course. As I delved into its background, regulatory loopholes, and pricing maneuvers, I uncovered something more sinister than mere corporate avarice. This was institutionalized corruption: polished, credentialed, and embedded within the framework of contemporary medicine. A decay so profound it could outshine a glioblastoma.
The genesis and demise of a drug
Acthar (repository corticotropin injection, ACTH) received its initial FDA approval in 1952. At that time, a drug could be approved if deemed safe; evidence of effectiveness was not necessary. The Kefauver-Harris Amendments of 1962 later mandated proof of efficacy. Acthar Gel was a basic pituitary extract suspended in gelatin for gradual release. It was affordable, fairly effective, and extensively utilized throughout the 1950s-70s for MS relapses and inflammatory conditions. By the early 1980s, synthetic corticosteroids (prednisone, methylprednisolone, and dexamethasone) rendered ACTH obsolete. By 1995, Acthar’s rights came under Rhône-Poulenc Rorer (subsequently Sanofi-Aventis). Demand had dried up. In 2001, they sold it to a small biotech, Questcor Pharmaceuticals, for $100,000. At that moment, a vial was approximately $40.
The revival
Questcor recognized that Acthar’s FDA approval from 1952 was still in effect, eliminating the need for new clinical trials. They did not require investments in science, safety, or efficacy; only marketing. A handful of minor, unblinded “open-label” studies followed: uncontrolled, self-funded, and published in lesser-known journals. A few dozen patients, a few laudatory graphs, and suddenly Acthar was reintroduced as a “specialty biologic.” The price skyrocketed from $40 to $34,000 per vial. Then came the ultimate tactic: the biologic loophole. Because Acthar was an older, animal-derived extract instead of a modern synthetic compound, Questcor could categorize it as a biologic, protecting it from generic or biosimilar competition. This regulatory classification justified outrageous pricing under the guise of complexity. To fortify the monopoly, Questcor acquired and obscured Synacthen, the sole potential synthetic ACTH competitor. An outdated hormone transformed into a secured monopoly. In 2014, the Irish pharmaceutical giant Mallinckrodt purchased Questcor for $5.6 billion. The Federal Trade Commission subsequently fined Mallinckrodt $100 million for antitrust infractions, and the Department of Justice charged it with kickbacks and Medicaid rebate fraud. Mallinckrodt declared bankruptcy twice, paid mere fractions of these penalties, and continues to market Acthar at the same exorbitant rate. Ironically, in 2021, ANI Pharmaceuticals reintroduced a claimed competitor, Purified Cortrophin Gel. In a typical market, prices would plummet, but here they remained stationary. Both drugs continue to hover around $40,000 per vial.
The world’s priciest obsolete medication
To comprehend the absurdity, juxtapose Acthar with actual biologics. Medications like Keytruda have transformed medicine; they are intricate, engineered molecules that have undergone years of research, billions in investment, and extensive clinical trials. A single dose of Keytruda, one of the world’s leading cancer immunotherapies, costs about $10,000. Acthar, on the other hand, is a 1950s pig-pituitary extract. No recombinant technology. No modern trials. No innovation. Yet each vial is sold for $30,000-40,000, even though its production cost is likely only a few hundred dollars.
Reasons for prescriptions by doctors
ACTH and steroids both elevate cortisol levels. Once IV Solu-Medrol became the standard, rational justification for using ACTH ceased to exist. Questcor enlisted “key opinion leaders” with funding, speaking fees, and advisory roles. Academic institutions, eager for corporate funding, echoed the same presentations created by corporate marketing teams. Even reputable journals published ghostwritten articles that recycled the same unfounded assertions. Physicians, occupied, trusting, and sedated