A drug, once free, now costs $375k

[ochotona]

Health care robber barons.

https://www.kitco.com/news/2019-02-04/U ... -375k.html

[Kriegsspiel]

TLDR- This doesn't seem like robber barons/capitalism run amok, it seems like government regulations showing their potential to fuck shit up.

There is no relationship between patients obtaining drugs under the FDA's Expanded Access (Compassionate Use) program and the drugs being free. This article said that the reason the drug was free was that it was being manufactured by a small, family-run pharm company and prescribed for off-label use:

The cost of the treatment will likely skyrocket — from next to nothing to potentially more than $100,000 without insurance — now that the Food and Drug Administration has approved a medicine to treat their disease, Lambert-Eaton Myasthenic Syndrome.

“There has been a long-standing need for a treatment for this rare disorder,” the FDA said in a statement this week announcing the approval.

The reality is that physicians have been prescribing 3,4-DAP, a nearly identical treatment, off-label, since the 1980s. It was never formally approved by the FDA; instead, LEMS patients pay about $300 to $500 a month to get the drug from a compounding pharmacy. That, or they’ve been supplied with the drug — for no charge — from the New Jersey-based Jacobus Pharmaceutical Co.

With an FDA approval in hand, however, Florida-based Catalyst Pharmaceuticals now has the ability to charge what could be hundreds of thousands of dollars — for a medicine that costs almost nothing to make. What’s more: Catalyst could block older forms of the drugs from being given to patients, even though they didn’t invent the formulation in the first place.

Jacobus doesn't actually just give it to patients, it's ordered by Duke University for $100/g, which then distributes it to 350 patients, who pay the postal costs. If the people who have that disease aren't connected with the Duke program, that's when they pay compounding pharmacies $300 for it.

In the 1970s, Scottish scientists discovered 3,4-DAP — also called amifampridine — and by the 1980s, Swedish scientists were demonstrating that it worked in LEMS patients. The Muscular Dystrophy Association later asked Jacobus to manufacture 3,4-DAP because doctors wanted to study its use in LEMS, but didn’t have a steady supply of the chemical.

Jacobus agreed, and ever since has provided the drug to more than 1,000 of patients with LEMS, asking them only to cover postage costs, said Laura Jacobus, who runs the company with her father, David.

In 2000, Parisian researchers developed a shelf-stable version of 3,4-DAP by adding a phosphate group onto the amifampridine compound. The older version required refrigeration, which could be inconvenient for patients.

Amifampridine phosphate was licensed to a biopharma company, and the drug switched hands from one to another — until it was acquired by Bay Area-based Biomarin in 2009. Amifampridine phosphate was approved by the European Medicines Agency that year, and was given a new name: Firdapse.

It was also given a new price tag: $60,000.

So the more convenient form is the one that Catalyst owns, not the type that needs refrigeration:

Shell managed to get the drug from a compounding pharmacy, but was never instructed that the drug required refrigeration. So it quickly denatured, and never worked for her. Shell also tried to get the drug from Jacobus, but came up short.

“Nobody responded to us, because we didn’t have a doctor that was connected with Jacobus to get it for free,” Shell said.

(Jacobus says it currently provides 3,4-DAP for about 350 LEMS patients.)

The family eventually enrolled in a clinical trial Catalyst was running.
. . .
They are grateful that Catalyst offered her the drug when they felt they had no options. Shell isn’t concerned about the cost: She called her insurer, Blue Cross Blue Shield, and they assured her they would pay for the drug.

“Our insurance company said they view this as a specialty drug — saying that because so few people need it, they’ll cover it,” Shell said. “They told us our out-of-pocket cost would only be $60 per month.”

Catalyst has said it plans to set up programs to help patients navigate insurance questions and apply for financial assistance once the drug is launched in the first quarter of 2019.

Catalyst is banking on insurance paying for the drug. If they refuse to, they said they'll have to lower the price. The NHS said in 2010 that they won't require physicians to prescribe the Catalyst version, because the dirt-cheap version is available. Unless the FDA requires physicians to do otherwise, they would be free to do the same.

Finally

“From a commercial point of view, we didn’t do it right,” [Jacobus] added. “We thought we were doing the right thing by always making our drugs affordable — making this drug available in a compassionate use basis — and we got stung.”

Pretty sure that's what a free-marketer would have told her.

[Kriegsspiel]

To add one thing, Bernie said, "Catalyst’s decision to set the annual list price at $375,000 is not only a blatant fleecing of American taxpayers, but is also an immoral exploitation of patients who need this medication."

First, it seems Catalyst is trying to fleece insurance companies, not tax payers (as long as we're not forced to bail out said insurance companies by the government).

Second, it seems the patients need the medication, but they don't need Catalyst's medication, as long as the government doesn't force physicians to prescribe it in lieu of the dirt-cheap version.