Hefty Pricetags On Orphan Drugs

The market for drug therapies for rare diseases is becoming increasingly crowded with expensive new products, a Modern Healthcare review of FDA approval and company pricing data show.

Most of the new orphan drugs cost more than $7,500 a month, which has prompted some experts to call on drugmakers to reduce prices and take smaller profits.

Two drugs approved by the Food and Drug Administration during the past year treat a rare type of high cholesterol called homozygous familial hypercholesterolemia, a genetic disorder that affects one in a million people. Aegerion’s Juxtapid was approved in December and costs $295,000 a year. Genzyme’s Kynamro, which was approved a month later, is less expensive but still one of the priciest drugs to come to market during the FDA’s fiscal 2013, which ended Sept. 30. It costs roughly $162,600 a year.

All nine new oncology drugs approved in 2013 cost more than $5,500 a month. Three of the treatments were also approved with companion diagnostics, which have separate costs associated with genetic testing.

“I don’t see (prices) going down,” said Joshua Cohen, research assistant professor at the Tufts Center for the Study of Drug Development. “There’s still a lot of unmet needs.”

The drugs reflect the industry’s ongoing focus on the orphan-drug market, one of the biggest growth areas for drug approvals. One-third of the 30 new molecular entities and new therapeutic biological products approved by the FDA last year are orphan drugs.

More than half the newly approved orphan drugs received standard review by the FDA. Drugs that receive priority review are considered “major advances in treatment, or provide a treatment where no adequate therapy exists,” according to the FDA.

Drug companies and physicians say better science is leading to more targeted therapies and new treatments. But the patients and insurers responsible for covering the drugs’ costs face new challenges in their efforts to get the best treatment at the lowest cost. “We’re going to see pushback from payers and hospitals,” Cohen said.

Unlike in some European countries, where the national health systems have said they will not cover the costs of certain drugs, U.S. payers generally are unwilling to deny coverage. Competition with other private insurers and pressure from elected officials and patient groups—some of which receive funding from drug companies—make out-and-out denial difficult even if the science doesn’t stack up.

“The divided private market makes that kind of pushback harder,” said Dr. Aaron Kesselheim, assistant professor of medicine at Harvard Medical School.

Payers are using higher patient cost-sharing as one strategy to limit their costs. Tufts researchers found that the coinsurance rate for biologics has doubled to about 30% over the past decade. Prior authorization, quantity limits and step therapy—a policy that requires patients to first try a less costly drug—are other examples of payer pushback.

“As new therapies are developed for limited patient populations based on individual genetic variants, the market for each product will become smaller and smaller; accordingly, prices may become higher and higher,” Dr. Brian O’Sullivan, a pediatric pulmonologist, wrote Oct. 2 in the Journal of the American Medical Association. “The cumulative effect will place strain on the healthcare economy.”

Last year, O’Sullivan and about 25 other clinicians sent a letter to the CEO of Vertex Pharmaceuticals, which manufactures Kalydeco, a drug that treats a subset of patients with cystic fibrosis, requesting that the company lower the price of the $295,000-a-year drug. Dr. Jeffrey Leiden, Vertex’s president, CEO and chairman, met with some of the physicians and listened to their concerns. However, “nothing substantial happened,” O’Sullivan said. The drug, which was approved in January 2012, now costs $311,000 a year.

Most manufacturers of pricey drugs provide patient-assistance programs. Kadcyla, a new oncology drug that treats patients with HER2-positive, late-stage breast cancer, costs about $94,000 for a roughly 10-month course of treatment. The company provides financial support to people who make less than $100,000 a year.

About 80% of the drug pipeline has been developed by smaller companies, Cohen said. Experts expect orphan drugs to make up a larger portion of drug approvals during the next several years.

Some traditional drug companies also have turned out targeted new drugs, with GlaxoSmithKline getting three orphan drugs approved by the FDA in fiscal 2013. On Oct. 1, Merck said it was shifting to a “more agile operating model,” sharply reducing costs to focus, in part, on developing drugs for unmet medical needs.

In the JAMA article published this month, O’Sullivan wrote that drug companies should become more transparent about pricing. “A model of reduced profitability, particularly for lifelong therapies, is ethically responsible and institutionally plausible but will require pharmaceutical companies to develop new models and educate investors about the long-term advantage of regaining the trust of the public,” he wrote.

Source: Crain Communications Inc.

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