A ‘game-changing’ Hepatitis C drug and the expensive price tag that comes with it

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    Cure. It’s a beautiful word, one that medicine doesn’t take lightly and one that doesn’t come up very often for Hepatitis C.

    Cure. 

    It’s a beautiful word, one that medicine doesn’t take lightly and one that doesn’t come up very often for Hepatitis C.

    That is, until recently.

    A much anticipated line of treatments recently gained FDA approval, and more are in the pipeline. They boast a higher likelihood, 90 percent or more, that a patient will clear certain strains of the virus.

    The development has put Hepatitis C, a serious liver disease affecting tens of millions of people around the world, in the spotlight. But the price of the main drug, Sofosbuvir or Sovaldi, at $1,000 a pill has also taken center stage, spurring serious questions, conversations and in some instances, decisions to limit treatment in Philadelphia and beyond.

    A common but silent virus

    Paul Yabor, a Philadelphia native and community activist, learned he had Hepatitis C, or HCV, before it even had a name.

    “I was diagnosed with Hepatitis non A non B in 1990 as a result of a follow up to a diagnosis of HIV and AIDS,” he recalled.

    Hepatitis C didn’t really surface in the medical community until the ’90s. Today it’s considered the most common blood borne infection in the U.S. An estimated three to four million people nationwide are chronically infected, but most don’t know it.

    The infection is more common among baby boomers, who may have gotten a transfusion or been exposed to tainted blood in the health setting before it was screened out of the supply starting in 1992. The virus is also incredibly tough, with researchers believing it can survive (in blood) on an outside surface anywhere from a few hours to a few days. For that reason, anyone who has ever injected drugs, even once, is at very high risk of being infected. It can also be transmitted through sex.

    Some people, a quarter or less, naturally clear Hepatitis C, but for the many who are chronically infected, obvious complications may not surface for years or even decades. That’s why many call it a silent disease. Over time, however, the virus eats away at the liver and may lead to severe scarring and liver disease. It’s a common cause of liver failure and cancer.

    “I’ve seen people die, people I admire, people who were doing things with their life,” said Yabor. “And I’ve also seen the disastrous effects of long term liver disease. And it’s a horrible way to die. I wouldn’t wish it on anyone.”

    HCV-associated deaths have surpassed that of HIV infection.

    Difficult treatment options

    Hepatitis C has no vaccine, and only a small portion of those infected previously ever entered treatment and then cleared the virus. The main option has long involved a daily injection of a substance called Interferon, taken for six months, sometimes more, in combination with other drugs. People liken its side effects to chemo: intense fatigue, fevers and suicidal thoughts, to name a few. On top of that, there’s no guarantee it will clear the virus, with chances of cure ranging from 60 to now about 80 percent for the more common strains of the virus.

    On the flip side, those who develop advanced liver disease may need a transplant. That wait list has been growing. While a liver transplant can be lifesaving, it typically doesn’t rid a person of the Hepatitis C virus.

    Over the years, Yabor, with his doctor, had ultimately decided to monitor his virus and postpone treatment with the hopes that something better would come along. It’s a strategy some in medicine refer to as ‘warehousing.’

    But, for Yabor, it meant a constant “tape running in the background that said, ‘you know, you’re running out of time, you know what this thing is going to do to you.'”

    A game changer

    Yabor, who’s 52 years old now, may soon get to hit stop on that tape. That is in part thanks to a Bucks County scientist, Michael Sofia, and more specifically to a new hepatitis C drug he co-invented called Sofosbuvir (its brand name is Sovaldi).

    “It hit me because we were just drawing chemical structures on a piece of paper,” he said, recalling one of the ‘aha’ moments during the early development stages. “I looked at one structure and said, ‘this is the direction we need to take this program.'”

    Sofia has spent the last few years zeroing in on the Hepatitis C virus at a small biotech company, Pharmasset, based in Princeton, N.J. He says one challenge that has stumped scientists for decades is how to formulate a drug so it only targets the liver without negatively affecting other parts of the body. That’s why other hepatitis C treatments require certain additional drugs to combat the severe side effects. His lab essentially figured out how to mask the drug when it’s ingested and program it to only go to the liver.

    “And then once that drug gets to where you want it to go, this mask that’s on this drug is now able to fall off,” he said. “Because it takes advantage of some property within that cell or organ that allows that to happen.”

    Once in the liver with changed properties, the drug can’t get out. It stays there and attacks the virus, Sofia says.This is a concept that a wave of new Hepatitis C drugs still in the development pipeline are incorporating.

    Sofia says the introduction of Sofosbuvir in December has meant that for the very first time, Hepatitis C has an Interferon-free treatment option. It still has to be taken with other drugs (and some strains respond better than others), but so far it boasts fewer side effects, only needs to be taken for three months, and has shown cure rates surpassing ninety percent in phase three clinical trials.

    “That set the HCV world on fire,” said Sofia.

    It generated excitement among patients, providers and advocates around Philadelphia.

    “What we have on our hands is possibly the greatest public health accomplishment of our generation with the new treatments of Hepatitis C,” said Dr. Stacey Trooskin, a community co-chair of Hepatitis C Allies of Philadelphia and an infectious disease doctor at Drexel University College of Medicine Partnership Comprehensive Care Practice, which has several hundred Hepatitis C patients.

    “I think ‘cure’ is our new favorite word,” she said.

    Trooskin says the development has spurred increased testing efforts in the community, since a lot of people don’t know they’re infected. On a community level, getting more people tested and into treatment, she says, could in turn prevent further spread of the virus and new infections.

    The industry saw the drug’s potential, too. Before it gained FDA approval, the California drug company Gilead Sciences bought Pharmasset, a company of less than 100 people, for $11 billion. The move set in motion what some describe as one of the most successful drug launches in U.S. history.

    For Paul Yabor, it set in motion the start of treatment he’d been holding out for, for years.

    Difficulties obtaining an expensive treatment

    At its retail price, the new drug is about $1,000 a pill, commonly taken once a day for three months, in combination with another costly drug. Insurers, medicaid agencies and other payers have been questioning the price. Some senate leaders recently called for a congressional investigation. On the ground, many patients report having an extremely hard time getting approved for coverage of the drug. Yabor, who has Medicaid, was denied at first.

    “I don’t want to make a patient feel like they don’t deserve to be treated with expensive medication, but I do warn the patient, ‘it is very likely that we are going to get denied,'” says Kenneth George, the pharmacist at Drexel’s partnership clinic.

    George says from there, he appeals, often with success, but not always. Either way, it requires perseverance. He also says treatment guidelines and eligibility criteria from the varying payers are constantly changing right now, complicating matters more.

    As one example, Pennsylvania’s fee-for-service pharmacy program through Medicaid requires that patients show signs of liver disease to qualify for the new treatment. 

    An estimated 20,000 people with Medicaid in Pennsylvania have Hepatitis C, according to the state’s Medicaid pharmacy director, Terri Cathers, and less than five percent have previously started any treatment. She says it’s too soon to know how many people are on the new treatment, but each managed care agency the state contracts with has its own eligibility criteria.

    She acknowledges it’s a “real conundrum.” Before treatment of Sofosbuvir combined with other drugs, “we didn’t really question whether the patient had advanced liver disease, so in those cases it’s possible people who were maybe Hepatitis C positive but not really sick might have received treatment at that time because we weren’t screening for who had the right treatment. But the cost of this treatment has forced us to look at who are the best candidates to get biggest bang for your buck.” 

    Dr. Trooskin calls it a sign of the times.

     

    “There needs to be conversations that happen between payers and insurance companies, so as providers we can do what we love to do,” Trooskin said. “And that is care for patients and give them treatment they deserve and that so many of us have been waiting so long to give them.”

    Why the high price?

    Gilead is in the sweet spot right now, according to Eric Keuffell, a professor of health economics at Temple University’s Fox School of Business, with the drug bringing in $2.3 billion in sales during the first quarter alone.

    “Whenever an expensive product comes out it raises eyebrows,” he said.

    Keuffel explains that drug pricing in the U.S. is based on free market principles. Companies have more leverage to set prices if they’re first to develop and patent a drug, but he also says that encourages them to take risks to develop a product over years that may very likely fail and not get approved.

    “Now they can price it as high as they want. at some point there’s pushback , and managed care or payers will not pay for it,” he said. “Their game is usually to try to get up to that particular point and price it there.”

    Countries like the U.K. have a governing agency that negotiates price.

    As far as the retail price of Sovoldi, Keuffel says the big challenge lies in how many people could benefit. Other even less effective specialty drugs, such as in oncology, already cost two, three, four times more than Sofosbuvir, but may not apply to that many people.

    Those with hepatitis C, on the other hand, comprise large portions of the Medicaid population, the prison population and beyond.

    “So the question becomes how are we going to pay for all that?”

    Gilead defends the price, believing that it “reflects the value of the medicine,” a spokesperson wrote in a statement. The company points to the long term benefits from a short term treatment. It’s a one-time regimen that can rid a person of Hepatitis C, without the need for a costly liver transplant down the road. The company also stresses that existing Hepatitis C treatments are already expensive, costing upwards of $100,000. Additionally, for those without insurance and meet certain criteria, the company has a patient assistance option to obtain the medicine free of charge.

    Keufull thinks once a new wave of drugs makes it through the pipeline, and that is expected soon, the price of Sofosbuvir may go down.

    A personal ‘long battle’ nearing an end

    On a recent morning, Yabor took a seat in the office of LaNelta Nobles, a pharmacist at the Jonathan Lax Treatment Center in Philadelphia. They’ve known each other for a long time. Nobles reviewed Yabor’s labs and said she’s seeing signs of success already.

    “Remember what you were before you started treatment? Three million copies,” she told him. “After two weeks of treatment, it went down to less than 15 copies.”

    “Wow, that’s fantastic!” he responded. 

    Nobles said adherence is critical in order for the drug to work. She handed him a pill box containing his next month’s supply of Sofosbuvir, along with other drugs. 

    Yabor thanked Nobles, placed that next round of expensive medicine in his backpack and headed out the door. He said for him, waiting all those years for the new medicine has finally paid off.

    “I’m now going to be living a life without the threat of death by hepatitis C,” he said with a laugh. “I’m laughing because I’m happy. I’m laughing out of joy. This has been a long battle.”

    Yabor said just the act of taking the drug has already had a positive effect. He feels younger.

    “I get up in the morning and, like everyone else, you drag yourself, you do your routine, whatever that might be. And I just [have] felt like somebody put a bigger battery in me,” he said. “There was a little more pep in my step.”

    Yabor has four weeks left of treatment before he hopes he’ll be totally free of Hepatitis C.

    Cathers acknowledges it’s a “real conundrum.” Before treatment of sofosbuvir combined with other drugs, “we didn’t really question whether the patient had advanced liver disease, so in those cases it’s possible people who were maybe hepatitis C positive but not really sick might have received treatment at that time because we weren’t screening for who’s the right treatment. But the cost of this treatment has forced us to look at who are the best candidates to get biggest bang for your buck.”

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