another example is acthar gel. it was approved in the 1950s as a "miracle drug" for dozens of diseases. it is a hormone extracted from pig pituitary glands that stimulates corticosteroid production, and the research around the clinical use of this drug and corticosteroids won a nobel prize
in the 1950s, synthesizing steroids was expensive, so doctors used the pig-derived acthar (which was actually developed by armour & co, the chicago meat packing company). then cheap generic synthetic steroids gained use and acthar use declined, as they basically had the same effects.
except that acthar worked better than corticosteroids to treat a rare form of epilepsy in infants called infantile spasms, even though the drug was not FDA approved for this disease. however, there were too few patients for the drug to be profitable in that indication, so the manufacturer of acthar decided to take it off the market. physicians and patients revolted, as this was the only drug that worked for a serious disease, so the manufacturer kept selling it though they lost millions a year on it
enter questcor pharmaceuticals, which bought the rights to the drug for $100K. they cobbled together some existing publications and got FDA approval for infantile spasms. they increased the price of the drug from $30 / vial to $30K / vial, and then started marketing it in other indications like multiple sclerosis. there was no real evidence it worked in these indications, but because it was approved in the 1950s before modern FDA standards, it retained many approved indications
questcor was eventually acquired for $6B, and to this day there is no competition for acthar because there is no FDA pathway for making a generic version of a pig-derived drug
there is little backwards compatibility for FDA regulations
in the 1950s, synthesizing steroids was expensive, so doctors used the pig-derived acthar (which was actually developed by armour & co, the chicago meat packing company). then cheap generic synthetic steroids gained use and acthar use declined, as they basically had the same effects.
except that acthar worked better than corticosteroids to treat a rare form of epilepsy in infants called infantile spasms, even though the drug was not FDA approved for this disease. however, there were too few patients for the drug to be profitable in that indication, so the manufacturer of acthar decided to take it off the market. physicians and patients revolted, as this was the only drug that worked for a serious disease, so the manufacturer kept selling it though they lost millions a year on it
enter questcor pharmaceuticals, which bought the rights to the drug for $100K. they cobbled together some existing publications and got FDA approval for infantile spasms. they increased the price of the drug from $30 / vial to $30K / vial, and then started marketing it in other indications like multiple sclerosis. there was no real evidence it worked in these indications, but because it was approved in the 1950s before modern FDA standards, it retained many approved indications
questcor was eventually acquired for $6B, and to this day there is no competition for acthar because there is no FDA pathway for making a generic version of a pig-derived drug
there is little backwards compatibility for FDA regulations