Specialty drugs transform lives — but at a cost

Expensive new specialty drugs offer hope to patients with rare and chronic illnesses but also leave many worrying about paying for them

“If I didn’t have this medicine I’d probably be on oxygen all the time,” Cora Higson (with husband Bob at their Framingham home) says of Tracleer, a drug that lowers blood pressure in the lungs, easing Higson’s pulmonary hypertension.Michele McDonald for the Globe

Every year, Cora Higson fills out a sheaf of forms and waits several long, anxious days to learn whether a charity will pay for the drug she needs to breathe.

The medication — Tracleer — is so essential , Higson says, that she is supposed to call her doctor immediately if she misses a dose. But her share of the cost is a whopping $1,111.47 per month .

Tracleer is the brand name for bosentan, a drug that lowers blood pressure in the lungs, easing the pulmonary hypertension that otherwise would leave her gasping for air.

So far, Higson hasn’t forgotten a pill, nor has she been turned down by the charity, Caring Voice Coalition of Mechanicsville, Va., which provides a grant to cover her share of the drug’s cost. But each time she fears that she won’t qualify for help.

Higson, 56, a former hairdresser, has health insurance through her husband, Bob, an air-conditioning technician, but his union-based plan requires a 30 percent copay on certain drugs.

“I wouldn’t mind paying. I mean, it’s not like we’re destitute,” Higson said. “But I can’t afford $1,100 a month. . . . If I didn’t have this medicine I’d probably be on oxygen all the time.” (An alternative drug she tried had severe side effects, and the other options aren’t any less expensive, she says.)

Born with a hole in her heart, Higson had open-heart surgery at age 7, but her heart didn’t trouble her again until her 40s, when heart failure and pulmonary hypertension struck. She now receives disability payments.

Throughout Massachusetts, people like Higson worry they might no longer be able to afford an essential medication as prices rise and insurance policies change, and some are even skipping or splitting pills, patient advocates say.

A quarter of the 40,000 Massachusetts residents who sought help last year from the National Patient Advocate Foundation said their biggest problem was affording the out-of-pocket cost of pharmaceuticals, said Larry Lanier, who leads state outreach and community advocacy for the foundation.

This is happening even though — on average — patients nationwide pay less than $10 out of pocket for their prescription medications, 86 percent of which are generics, according to the IMS Institute for Healthcare Informatics. Higson, for example, has no trouble paying for her five other medications, all generics that together cost her less than $20 a month.

Higson’s outsize copay for Tracleer exemplifies a widening gap between the lower-cost generics that most people use and new “specialty” drugs with eye-popping price tags. For example, Sovaldi, a new treatment for hepatitis C, cures the vast majority of patients, but at a price of $84,000 per person for the full course of treatment.

Specialty drugs are medications that work by complex mechanisms and often require special handling or monitoring. They cost a lot but can transform or save lives, treating such illnesses as cancer, rheumatoid arthritis, multiple sclerosis, HIV/AIDS, and other chronic conditions. Nineteen of the 28 drugs approved by the FDA last year were specialty drugs, including 10 new cancer treatments and 17 drugs for rare illnesses. And many more are in the pipeline.

Fewer than 4 percent of patients use specialty drugs, but they account for 25 percent of total drug spending in the United States; and the growth of specialty drugs is a key factor driving up health care spending, according to PricewaterhouseCoopers.

Faced with these soaring costs, and trying to control premium increases, insurance companies and employers have started shifting the burden to the minority of patients who need the costly drugs. Some plans group all specialty drugs into a separate “tier,” requiring patients to pay 20 to 50 percent of the cost, rather than the flat dollar amount required for other drugs.

“The impact of the specialty tier benefit design falls disproportionately on patients who are living with diseases and conditions that are serious and life-threatening,” said Marialanna Lee, Northeast Region state government affairs director for the Leukemia & Lymphoma Society. Often the specialty drug is the only one available for these conditions, she said.

“It’s going to cost more in the long run,” predicts Patricia Ferland Weltin, executive director and founder of the Rare Disease United Foundation. If denied drugs that keep them well, she says, “They’re going to end up in the E.R, they’re going to end up sick, costing more money than if they took the drug. . . .

“It just breaks my heart to see. We’re finally making headway with treatments, and the insurance companies’ lack of foresight is going to change that.”

In Massachusetts, the health insurers say they have not segregated specialty drugs into separate tiers. “It’s not a strategy overall that Blue Cross has adopted,” says Dr. Tony Dodek, vice president of medical quality and strategy for Blue Cross Blue Shield of Massachusetts. “Our first priority is to make sure that our members have access to these drugs.”

But some plans do require percentage copays, including several sold on the Massachusetts Health Connector. Additionally, Medicare Part D drug coverage does allow specialty tiers. And others may see specialty tiers if their employer is self-insured — that is, the employer designs the benefit and assumes the risk, hiring the insurance company merely to process claims. (Cora Higson’s coverage, through her husband’s union, is one such plan.)

Six states have adopted legislation that in one way or another puts a cap on consumers’ costs for specialty drugs. A bill in Congress also would limit specialty tiers.

The Massachusetts Legislature is considering a bill, modeled after a law in Vermont, that would cap out-of-pocket costs for prescription medications at $1,250 a year for an individual and $2,500 for a family. The cap would apply to all plans sold on the Massachusetts Health Connector as well as to commercial plans that people obtain through their employers. But people whose employers are self-insured — about half the commercially insured population — would not be affected, because such plans are exempt from state insurance rules.

Eric Linzer, senior vice president of public affairs and operations for the Massachusetts Association of Health Plans, says that capping out-of-pocket drug costs will only raise premiums and force insurers to remove some low-premium plans from the market, without addressing what he calls the source of the problem: drug prices. “We need to take a good hard look at what pharmaceutical companies are charging for prescription drugs,” he says.

The bill’s sponsor, Senator Anthony W. Petruccelli (D-East Boston), says the bill has been well-received, winning approval in two key Senate committees. But there may not be enough time to move it out of the Senate and through the House before the July 31 end of the legislative session.

That will mean another year of worry for people like Diane Lima of Acushnet, whose 14-year-old son depends on a hemophilia treatment that costs $25,000 to $30,000 a month. Years ago the boy needed a wheelchair from time to time because of frequent bleeds into his muscles and joints. Now, thanks to injections every other day, her son can play sports at his high school, where hardly anyone even knows he has hemophilia.

Lima works for a hospital group whose health insurance policy covers her son’s care. But she knows that she’s expensive to insure and worries every year that her employer will switch to a less generous plan.

“We have been super-fortunate,” Lima says. “But you don’t know what’s going to happen tomorrow.”