There’s an often-forgotten unapproved drugs initiative from the Food and Drug Administration that contributes to the ratcheting up of drug prices. There are still a number of drugs in use that haven’t actually been approved by the FDA, but are waiting for some enterprising biopharma entrepreneur to snap ’em off the shelf, submit them for review, and get marketing exclusivity on the drugs.
This rule contributed to a recent price hike in vasopressin, a long-used drug for cardiac arrest. Since its FDA approval, a vial of neostigmine has increased from $5 to $90.
In the midst of this Martin Shkreli mess, a rather thoughtful article emerged from the Los Angeles Times on this issue. It says this FDA stipulation “underscores an enormous flaw in our drug-approval process that rewards a few clever manufacturers at the expense of patients.”
The LA Times writes:
The most notorious case is that of colchicine, which was first used to treat gout 3,000 years ago and has been in generic use in the U.S. since the 1800s. Following a 2009 FDA order awarding exclusive rights to a branded formulation of colchicine to a Philadelphia drug maker and barring competing generics from the market, its price rose 50-fold.
In many of these cases, the new manufacturers had done little to alter, much less improve, the traditional drugs before claiming FDA-mandated exclusivity periods ranging from three to seven years. Some performed new safety or efficacy testing, but often these trials merely confirmed what doctors had known about the drugs after decades of use.
The article adds that the FDA’s orphan designations abet these steep pharmaceutical cost hikes:
As Aaron S. Kesselheim and Daniel H. Solomon of Harvard Medical School reported in 2010, that’s what turned colchicine from a traditional medication for gout into a branded monopoly for the firm URL Pharma, which put colchicine on the market as Colcrys.
The firm claimed the drug’s effectiveness against the orphan condition of familial Mediterranean fever, a genetic condition affecting only 100,000 patients in the world, and also in a low-dose formulation for gout. URL sued to bar other companies from selling colchicine, and raised the price from nine cents per pill to $4.85. State Medicaid programs, which had been spending about $1 million a year on colchicine, were now facing bills totaling $50 million.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
In essence, the impetus regulators give to drugmakers to make the drugs patients rely on — market exclusivity — plays a huge role in barring the drugs from reaching patients who can’t afford them.
