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Advice and Resources for the Biotech Industry

Advice and Resources for the Biotech Industry

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We’ve Got Very Effective Disease Treatments. Too Bad They’re Not Being Used

“I want a new drug, one that does what it should, one that won’t make me feel too bad, one that won’t make me feel too good.” Huey Lewis and the News

Many people were desperately searching for a new drug in April of 1984 when “
I Want A New Drug” reached number one on the Billboard charts. In the midst of the AIDS epidemic, the world was looking for anything that could stop a rapidly spreading, poorly understood disease. It was a cruel diagnosis that, at the time, was tantamount to a death sentence. Drugs capable of successfully combating HIV were eventually discovered, but that hasn’t ended the AIDS crisis. Getting these powerful and expensive medicines in the hands of people who need them has turned out to be an equally daunting challenge.

Throughout history people have been praying for new treatments that would either cure them of their ailments, or prevent them altogether. Thanks to advances in science and medicine, we now have many of these solutions in hand. Huey wouldn’t be singing about a new drug these days. Instead, his lyrics would be shaped more along these lines:

How will I pay for the new drug?

Will my insurance cover it?

I’ve been reflecting on our advances in the prevention and treatment of diseases as I mark my 30th year in biotechnology. What I’ve learned over that time is that biology is the most complicated scientific discipline, bar none. However, with pluck, endurance, and a number of key insights along the way, we’ve actually been winning the war against a number of deadly diseases over the past century. The treatments are already in hand. Instead, the challenge has become enabling (or in some cases convincing) patients to purchase and use them. With medical costs rising rapidly and
real wages actually falling, many Americans can’t afford to fill their prescriptions. Even if they can pay, some are cutting their doses so that the prescriptions last longer, even if this might render the medicines ineffective.

The current problem can be illustrated by examining how diseases caused by three different viruses, HPV, HIV, and Hepatitis C, are treated. Scientists have been notching repeated victories in the fight against these tiniest forms of life while most people weren’t paying attention. However, factors beyond the scientist’s control have conspired to prevent their treatments from getting into the hands of those who need them most. Their heroic efforts have been thwarted by bad policy decisions, high costs, and “fake news” masquerading as facts on social media. Many of these problems are uniquely prevalent in the U.S. As Andy Slavitt, the former head of the Affordable Care Act during the Obama administration, put it recently, “
If you have to choose between health care and some other expense, chances are good you’re American.

Let’s focus on how the development of new drugs and treatments wound up being only one part of the ongoing struggle to defeat three different viruses.

Human Papilloma Virus
The human papilloma viruses are a family with more than 150 members. These viruses are incredibly common, with nearly 80 million Americans currently infected. A small percentage of these viruses are sexually transmitted, and a small number of these can give rise to seven different kinds of cancer. Only a tiny percentage of people infected with these cancer-causing HPV viral strains will actually go on to develop malignant tumors. Given the high infection rate, this translates into more than
30,000 cancer cases per year in the U.S. HPV infections cause more than 75 percent of cervical, vaginal, and vulvar cancers in women; penile cancers in men; and anal, rectal, and oral cancers in both men and women. Thankfully, efforts to develop a vaccine against these cancer-causing viruses have been successful. The current vaccine provides protection against nine different cancer-causing strains of HPV. It’s recommended for both girls and boys starting around age 11-12. The vaccine, which must be given before kids become sexually active, is both safe and highly effective. Widespread adoption of the vaccine will result in a drop in the number of infections, which in turn will lead to a large reduction in the incidence of these cancers. That’s the good news.

The bad news is that vaccination rates for boys and girls are nowhere near as high as they should be. Only about half of girls and 38 percent of boys have currently been vaccinated in the U.S. The rest remain at risk of someday developing HPV induced cancers. The rates at which parents have their kids vaccinated varies greatly from state to state. For example, only 20 percent of boys in Wyoming have been vaccinated compared to 69 percent in Rhode Island, the highest-ranking state. For girls, only 31 percent are current with their vaccinations in South Carolina, while Rhode Island again leads the way with 73 percent. Only three jurisdictions (RI and VA, along with the District of Columbia) currently 
mandate HPV vaccinations to attend school. The rates in the poorly vaccinating states are below the threshold of what is generally thought to be necessary to establish herd immunity against HPV, where uninfected people are generally protected from being infected by the vaccinated people around them. Immunization rates are much higher in a number of other countries, especially those where the vaccine is mandated.

So why aren’t kids getting this vaccine? There are several reasons, which I’ve discussed at length in a
previous article. While some of the problem is simply ignorance about the vaccine by parents, much of the blame for this failure to immunize lies at the feet of the anti-vaxxers. I’m not only talking about folks who are specifically focused on the HPV vaccine, though they exist. I’m focusing on a growing movement, fueled by celebrities and misinformation spreading out of the dark corners of the Internet, that threatens the progress we’ve made in fighting viral diseases. Dr. Paul Offit, a vaccine expert, has recently written an entire book about this phenomenon: Bad Advice: Or Why Celebrities, Politicians, and Activists Aren’t Your Best Source of Health Information. These people have bought into the (thoroughly discredited) idea that modern vaccines are unsafe and will injure their children. Not being doctors, they’ve never seen the harms that these viruses can cause. Scare stories on social media fan the flames of vaccine resistance. Some parents, not knowing whom to trust, take the path of least resistance or succumb to these worrying tactics and don’t have their kids immunized.

Bottom line: instead of wiping out HPV, which should be within our grasp, it will continue to spread and result in thousands of men and women being diagnosed with life-threatening cancers that could have been prevented. We need to do a better job of ensuring that kids get vaccinated against this virus.

Hepatitis C Virus (HepC)
This virus, which was only
discovered in 1989, causes liver damage over a long period of time (sometimes decades). Hepatitis C infections often lead to liver cirrhosis, and the virus is thought to contribute to about half of all cases of liver cancer. Most people don’t know this, but hepatitis C kills more Americans than any other infectious disease (nearly 30,000 people a year). It kills more people than 60 other infectious diseases (including HIV and tuberculosis) combined! About 3.5 million Americans are infected with hepatitis C, though only about half of these people know they carry the infection. Seventy-five percent of those infected are baby boomers, many of whom were infected when they were young and there was no way to screen for this virus. That’s why you’re seeing all of those pharma ads begging boomers to get tested. Here’s the good news: highly effective drugs to treat and clear the infection have been developed and have been on the market since 2014. If everyone who had the virus was treated with these drugs, we could eliminate a large number of fatal cases of cirrhosis and liver cancer each year. A recent clinical trial suggested that these drugs could also be used to cure kidney transplant patients that obtained organs from donors who had HepC, thereby expanding the pool of available, life saving kidneys. “Why are so many Americans dying of this preventable, curable disease?” asked Jonathan Mermin, M.D., director of CDC’s National Center for HIV/AIDS, Viral Hepatitis, STD, and TB Prevention.

The answer to this question is that finding and getting people treated is challenging. There’s no requirement that people be tested, so many infections go undiagnosed. The other part of the problem is that these new stand-alone, hepatitis C-curing drugs are incredibly expensive. When they first came on the market they cost $84,000 and up per course of treatment for each patient. Competition has cut that price by at least half, but these remain very costly drugs. Insurance generally covers the treatment. However, the millions of Americans who don’t have insurance remain at risk of developing cirrhosis and liver cancer, and also serve as a reservoir for spreading the infection further.

Most hepatitis C infections arise these days from the sharing of contaminated needles by drug users, many of whom are imprisoned. There are nearly
two million inmates in US prisons and jails. It’s estimated that about one in seven prisoners are infected with hepatitis C, and about one in three Americans with the disease go through a correctional facility every year. Despite this, less than one percent of those inmates are treated with these new drugs. It’s estimated that the annual cost to house each inmate in a state prison is about $29,141. These new drugs would cost more than twice that amount per prisoner. Inmates have filed lawsuits to get the drugs, arguing that withholding the drugs constitutes cruel and unusual punishment. Some states, like Florida and New York, will provide treatment for some prisoners, but most do not get this. As one expert pointed out, “It’s an impossible situation that the prison administrators are put in. You can’t buy something you don’t have any money for.

In a new and promising development, the state of Louisiana is looking at a “Netflix model” where it will pay Gilead a set amount for a period of years in exchange for an unlimited amount of the company’s HepC medicine. This would then be used to treat Medicaid patients as well as those in prison. Taking a different approach, China has invalidated Gilead’s patent on it’s HepC medicine in order to widen access to the drug it their country.

Bottom line: we have a cure for this disease, but not the money to pay for it, especially for those without insurance or in prison. A way needs to be found to get these drugs to all who need them.

Human Immunodeficiency Virus (AIDS)
This virus, which burst onto the world’s stage in the 1980’s, has
killed about 35 million people around the globe since the start of the epidemic (about 700,000 in the U.S.) Years ago, a diagnosis of HIV infection was akin to a death sentence, often a slow and painful one. Fortunately, things have changed markedly since then. We now have multi-drug cocktails (HAART, highly active anti-retroviral therapies) that transformed a once lethal diagnosis into a chronic, manageable disease for many. And the good news doesn’t end at treatment. Notably, Gilead Sciences, Inc. has reversed an earlier decision and decided to market their HAART drug Truvada as a pre-exposure prophylaxis (PrEP) treatment. The drug is about 92 percent effective at preventing people at high risk of acquiring this infection from doing so. It’s aimed at people who either have an infected partner or are intravenous drug users. As one doctor put it, “it’s the closest thing we have to an HIV vaccine.” These extremely effective therapies provide a lot of good news in the management of this disease.

Sadly, there’s bad news to balance this out. Once again, cost and other factors often prevent HAART and PrEP drugs from getting into the hands of all those who need them. HAART therapies have an average wholesale
price of about $3,000/month. Gilead’s PrEP drug Truvada is not reaching anywhere near as many people as are needed to wipe out HIV, in large part because it costs a hefty $2,000 per month. The CDC estimates that nearly 1.1 million Americans should be taking the drug, although the actual number is only about 167,000. Activists are targeting Gilead and asking that the company either lower the price, or allow another company to sell a generic version of the drug. Another idea that bounces around the Twitterverse is that the Government should buy Gilead and provide its drugs at substantially lowered prices. I’ve written about the pros and cons of this approach, and why it would face numerous challenges to enact. It won’t happen anytime soon because there’s simply no political will to even consider this idea under the current administration.

While Truvada is remarkably effective at preventing HIV infections, many gay men have been
denied life, disability, or long-term care insurance specifically because they are taking this drug. Insurance company decisions discouraging use of the drug force those wanting to take this medicine into having to make a difficult choice. Prevent HIV infection, or do without insurance products that most people can obtain easily. This policy only serves to spread the infection further. Cost and insurance issues aren’t the only problem here. So is compliance. Inconsistent use of Truvada (e.g. forgetting to take it every single day) can reduce its efficacy to near zero. To address this non-compliance issue scientists have developed special sensors that can be directly imbedded into the pills. These give patients feedback as to how well they are managing their medications, and with the goal being a boost in compliance rates.

Bottom line: high costs, insurance company policies, and patient non-compliance issues are impeding great progress in both preventing and treating HIV. Education campaigns and cost savings are needed to reverse these trends, along with new regulations for the insurance industry.

New Drugs Are Simply Not Enough
In the three examples above, the scientists have done their jobs extraordinarily well. They’ve discovered real solutions to help prevent, treat, or cure each of these infectious diseases. It’s not the science that’s holding us back at this point. It’s the politics, social policy, and money being spent in the wrong places (e.g. big tax breaks for corporations, most of which have gone to funding stock buybacks). We desperately need a healthcare system that meets the needs of all of us. We need to make better efforts to combat the mountains of misinformation and prejudice against certain groups that prevent people from taking full advantage of the drugs that prevent and treat these diseases. Educational campaigns illustrating just how safe and effective life-saving vaccines are need to be ramped up. Helping the poor, the disadvantaged, and even prisoners will bring benefits to all corners of society when these diseases have been, for the most part, eliminated. Most of us don’t know anyone who’s had smallpox, polio, or rabies. Let’s hope our children get to add three more viruses (HPV, hepatitis C, and HIV) to that list as well.