Blockbuster drugs, budget busting cost: One Delray woman’s struggle

July 29--Tell us about prescription drugs Have high costs made it difficult or impossible to get prescription drugs? Does prescription drug advertising -- allowed in the U.S. in ways that most countries restrict -- help or hurt? Tell reporter Charles Elmore your story at [email protected] or call (561) 820-4811.

The front lines of a high-stakes battle over prescription drugs crossed Vickie Goldstein's 100-pound body in Delray Beach.

"I was terrified," she said.

Florida's Medicaid system was telling her she had to get sicker -- with liver scarring that could push her to the brink of a transplant -- before it would approve prescription medicine with a 90 percent or better chance of curing hepatitis C, she said. The viral infection can lead to liver failure, cancer or death.

"They told me I wasn't sick enough," Goldstein, 57, said. "That made me crazy."

It is a story tangled up with some of the toughest issues in U.S. health care. While new drugs to treat hepatitis C appear to be highly effective, they are also very expensive. Some health officials warned they risked becoming outright budget busters, blowing a hole in the system with tens of billions of dollars in costs to treat the nation's most common blood-borne infection. Treatment can cost between $24,000 and $31,000 a month.

Goldstein refused to give up. She requested a review of the decision with the help of groups including Legal Aid Society of Palm Beach County, Florida Legal Services and the National Health Law Program.

Effective in June, Florida officials changed their minds. For her and others with the virus, the system would make the medicine available sooner.

"We are thrilled that Palm Beach County Medicaid recipients will no longer hit a barrier when trying to access life-saving and curative hepatitis C treatment," said Bob Bertisch, Legal Aid's executive director in West Palm Beach. "Our client's life and the lives of many others will be improved by these corrective steps."

But the case only stokes an intensifying debate about costs.

The cost of manufacturing the pills for a 12-week course of treatment can be as little as $150 to $250, yet drug makers were charging about $84,000, calculated Andrew Hill, a researcher in the Department of Pharmacology and Therapeutics at the University of Liverpool.

"Never before has a drug been priced this high to treat a patient population this large, and the resulting costs will be unsustainable for our country," Express Scripts chief medical officer Steve Miller warned in 2014. Express Scripts is a St. Louis-based company providing pharmacy benefit management services.

Initial prices were "astronomic," Miller said this week. His organization says it reached agreements that helped cut the market price roughly in half, saving clients about $1 billion annually and others in the healthcare system about $4 billion in a ripple effect. "It was the right thing to do," Miller said.

Groups representing drug makers say predictions of financial ruin were overblown.

"A once incurable disease -- hepatitis C -- now has cure rates above 90 percent," said Priscilla VanderVeer, depty vice president of communications for the Pharmaceutical Research and Manufacturers of America. "This new generation of hepatitis C medicines cure patients of the disease, helping them avoid long-term liver damage like cirrhosis, which can require a liver transplant costing as much as $500,000 and years of costly follow-up care."

Advancements like hepatitis C drugs are possible because "we have a health care system that recognizes and rewards risk-taking," VanderVeer said. Government price controls on prescription drug prices would have a "devastating impact" on medical innovation, she said.

Goldstein's research, though, only made her more frustrated. It seemed she could get the drugs free if she lived in Egypt, or for about $500 in India. But the U.S. system imposes few limits on what drug makers can charge. In turn, many states, agencies and health plans, wary of a tidal wave of costs, imposed significant restrictions on who can get the medication and when.

Congress held hearings this year on price increases of more than 5,000 percent for some older life-saving drugs, casting an unflattering spotlight on current or former executives such as "Pharma Bro" Martin Shkreli. Memos that seemed to gloat about windfall profits "confirm what Americans across the country have experienced firsthand for years -- that many drug companies are lining their pockets at the expense of some of the most vulnerable families in our nation," said U.S. Rep. Elijah Cummings, D-Maryland.

Still, few expect sweeping changes as drug manufacturers have lobbied effectively against price controls.

So for many people, access to drugs comes down to what private health plans or government agencies decide to approve and pay. The outcome affects not only individual patients, but virtually everyone -- because overall drug and other health costs become baked in to insurance premiums and taxes people pay.

It has been a long, exhausting fight for Goldstein. When the box of prescription medicine finally arrived, she could not initially bring herself to tear it open, she said.

"It took me three years to get," she said. "I wasn't going to stop until I did. I looked at the box for a whole day. I just couldn't open it."

Now she is about two-thirds of the way through a 12-week treatment, which she said can leave her feeling tired and irritable. At the end of the journey, she hopes, is a cure.

Hepatitis C virus is the most common blood-borne infection in the United States, affecting about 2 percent of the population, health officials say.

An estimated 300,000 Floridians are infected, with approximately 2,000 new cases occurring each year, according to the Florida Department of Health.

The infection can persist over decades or come on strong in a burst of acute symptoms, but about 10 percent to 20 percent of those with a chronic infection will develop cirrhosis of the liver, and 1 percent to 5 percent will develop liver cancer, officials say.

The virus is transmitted primarily through contact with contaminated blood that is injected -- commonly though drug use with needles, though blood transfusions and other causes have been cited.

Goldstein, who has the support of parents who live with her, said she has undergone blood transfusion during medical procedures and used intravenous drugs in the past, but has not done so for many years.

"Most people with hep C don't want to raise their hand," she said.

Molina Healthcare, which manages some Medicaid patients in Florida's system, denied Goldstein access to Viekira Pak, a direct-acting antiretroviral drug used to treat the virus, she said. A denial notice said the medication was "not medically necessary," she said.

Molina Healthcare declined comment on an individual case, but said it adheres to the new state policy.

"While we do have concerns about the high costs associated with Hepatitis C drugs, our top priority continues to be providing the most compassionate, supportive care possible to our members," a company spokeswoman said.

Goldstein's persistence caught the attention of lawyers who worked with her.

"She really kept pushing forward for her health needs and her rights," said Vicki Tucci Krusel, an attorney with Legal Aid Society of Palm Beach County. "She's a woman of great courage. She wasn't backing down. She wanted to live."

Effective June 1, Florida's Agency for Health Care Administration discontinued a requirement for evidence of hepatic fibrosis, or liver scarring, before approving the drugs. Officials said it was a policy change not necessarily tied to any one case but came after a review that included a look at trends around the country.

Health plans, who oversee Medicaid patients in Florida under managed-care programs, "may choose to continue to review for proof of abstinence of illicit drug and alcohol use or that patients are receiving substance or alcohol abuse counseling services," state officials said.

Beginning Sept. 1, the state agency "will make adjustments" to the health plans' rates to reflect this policy change. At this point, officials said they were unable to estimate how much this change will affect Medicaid costs in the state.

The agency is still reviewing data to address a question from The Palm Beach Post about how many Medicaid patients might have been denied curative treatment with hep C drugs in recent years, an AHCA spokeswoman said.

Last year the agency approved a 7 percent increase for Medicaid plans, and Florida's chief economist Amy Baker said drug costs were a driving factor not only in Medicaid but also state employee health plans.

For Goldstein, the reasons for delaying treatment made little sense.

Was it really better to let someone get so sick they might need a liver transplant or get liver cancer, and then agree to cure their hep C?

"If I had Stage 1 breast cancer, would they say wait until Stage 3 or 4 to do anything about it?" she said. "No. And this is a cure."

It troubled her, she said, to know a prospective cure was out there, only to be told it was "not medically necessary" for her.

One thought gives her comfort, she said -- that she might have helped other patients get medicine sooner.

"That made all the difference in he world," she said.

___

(c)2016 The Palm Beach Post (West Palm Beach, Fla.)

Visit The Palm Beach Post (West Palm Beach, Fla.) at www.palmbeachpost.com

Distributed by Tribune Content Agency, LLC.