‘Orphan drugs’ no lifeline if you can’t afford them

February 27, 2017

State Sen. Jeff Steinborn, D-Las Cruces, says state government spending on pharmaceuticals is a “runaway train,” topping $679 million in 2016.

He and state Rep. Joanne Ferrary, D-Las Cruces, are co-sponsoring Senate Bill 354, which would create an “interagency pharmaceuticals purchasing council” to review and coordinate cost-containment strategies for the procurement of pharmaceuticals and pharmacy benefits and pool purchasing by state agencies.

“By coordinating purchases, we should see some cost efficiency,” Steinborn said in a phone interview.

Driving the high cost of pharmaceuticals, according to the National Council on State Legislatures, are specialty medications approved under the Orphan Drug Act.

Specialty medications for inflammatory conditions, multiple sclerosis and cancer top the list, according to a 2015 report by IMS Health, a pharmaceutical data analytics company.

Alarming, too, is that some drug companies are following a business model to acquire off-patent sole-source drugs, which allows them to obtain a monopoly under the Orphan Drug Act and then impose and protect astronomical price increases, according to a special report by the U.S. Senate Committee on Aging.

In April 2016, the committee found that the mean price of insulin, a lifeline therapy for the 29 million Americans with diabetes, increased from $4.34 per milliliter in 2002 to $12.92 per milliliter in 2013, almost a 300 percent increase.

Drugs for multiple sclerosis, which cost $8,000 to $11,000 a year in the 1990s, now sell for about $60,000 a year, according to an April 2015 article in the journal Neurology.

Humira was approved by the FDA in late 2002 to treat millions of people who suffer from rheumatoid arthritis. Three years later, it was designated as an “orphan drug” to treat juvenile rheumatoid arthritis.

Humira subsequently was approved for four more rare diseases, including Crohn’s and uveitis, an inflammatory disease affecting the eyes, according to a January 2017 report by National Public Radio.

The results of a six-month investigation by Kaiser Health News found more than 70 of the drugs approved by the Food & Drug Administration were reapproved as orphan drugs in the treatment of rare medical conditions.

The Kaiser investigation found that about a third of orphan approvals by the FDA since the program began have been either for repurposed mass-market drugs or for drugs that received multiple orphan approvals.

Medications such as the recently FDA-approved Marathon Pharmaceutical’s deflazacort, which is used to treat Duchenne, a rare form of muscular dystrophy causing muscle degeneration and weakness, had been available for decades in Europe for $1,600.

Marathon has delayed commercialization efforts after protests from consumer advocates and a congressional inquiry, yet its pricing of the drug at $89,000 per year was not out of the ordinary.

The top 100 orphan drugs in the U.S. cost an average of $111,820 a year per patient in 2014, according to a report by Evaluate Pharma, a market research firm.

Besides the extraordinarily high pricing for newly approved specialty medications, there are also restrictions on purchasing drugs outside of the U.S. once they have been approved by the FDA.

“What we are seeing is a system that was created with good intent being hijacked,” said Bernard Munos, a former corporate strategy adviser at drug giant Eli Lilly and Co. in a recent interview on NPR’s Morning Edition.

It’s “quite remarkable that it has gone on for so long,” Munos said.

Andy Winnegar has spent his career in rehabilitation and is based in Santa Fe as a training associate for the Southwest ADA Center, 800-949-4232. He can be reached at a@winnegar.com.