Diana Zuckerman, PhD, Cancer Prevention & Treatment Fund
Published in 19 newspapers including, Chicago (IL) Tribune, Sacramento (CA) Bee, McAllen (TX) Valley Voice, Bellingham (WA) Herald, Cambridge (OH) Sunday Jeffersonian, Lawton (OK) Constitution, Mason City (IA) Sunday Globe Gazette, Willimantic (CT) Sunday Chronicle, New Bedford (MA) Standard Times, Frederick (MD) News Post, and others from March 16-20, 2017

If you or a loved one were dying of a terminal illness and your doctor told you there were no proven treatments, would you take the risk of trying an experimental, unproven drug?

Many patients would say yes. But as with most medical decisions, the more you know, the more you realize the answer is not so simple.

As has been clearly shown, Congress is not very good at making complicated and nuanced decisions about medical care.

That’s reason enough to question the new federal Right to Try Act dozens of senators and representatives are pushing this spring.

The most important thing to know is that all terminally ill patients already have a right to try experimental drugs in this country.

The proposed new law, however, is much more dangerous to all patients, and not just those facing fatal illness.

Here’s why:

The current national expanded access program enables doctors to request experimental drugs for their patients. If the company that makes the treatment agrees, the patient will get the treatment for free or at cost; companies are not allowed to sell experimental drugs for a profit.

Many patients get access to experimental drugs through this existing program, and improvements are underway to further streamline the process.

In contrast, under the proposed new law, a drug company could charge desperate patients as much as they want to get access to an experimental drug.

Since insurance companies do not pay for experimental treatments, many patients would wind up with the right, but not the money, to try such regimens. Out of desperation, some would surely go into debilitating debt to try a drug that might harm rather than help them.

The current national program makes sure patients understand the risks of taking an experimental treatment and requires that the drug has been studied enough to know that the patient might possibly benefit from it.

Under the proposed new law, drugs that were only studied at a low dose on a small number of healthy volunteers could be sold to patients, and unethical doctors could receive kickbacks for persuading patients to try treatments that will not help them.

It’s easy to understand why every patient wants to have hope of a cure, and that’s the power of right-to-try laws.

So far, hype and false hope have convinced 33 states to pass right-to-try laws that provide no real advantage over the current national program. But rather than learning from the mistakes at the state level, patient activists and others are pushing Congress to pass a much more dangerous federal law.

In addition to encouraging the sale of unproven treatments at sky-high prices to desperate patients, the 2017 federal Right to Try Act would do the following:

• Allow the sale of almost all experimental drugs, even those never tested on patients before.
• Prevent patients and family members from suing the company if the treatment harms or even kills them.
• Prohibit doctors and scientists from evaluating the benefit or harm of the experimental drugs.

Desperate patients are lobbying for the bill, but do they realize what they are lobbying for?

Instead of getting access to free experimental drugs that have some evidence of benefit and are being tested to help all patients, this law would allow naive and desperate patients to be exploited by greedy companies and unethical doctors.

The right-to-try movement opposes the FDA for what’s described as “interfering” with the doctor-patient relationship. They do not understand that unbiased scientific evidence is needed to help physicians and patients make informed decisions – whether to save a life or make a patient’s last months as enjoyable as possible.

Patients already have a right to try through the FDA’s humanitarian expanded access program, which gives them hope while protecting them from greedy exploitation. The proposed federal Right to Try Act would not.

Liz Szabo, Kaiser Health News: February 9, 2017

Marlene McCarthy’s breast cancer has grown relentlessly over the past seven years, spreading painfully through her bones and making it impossible to walk without a cane.

Although the 73-year-old knows there’s no cure for her disease, she wants researchers to do better. It’s been years, she said, since she has found a drug that has actually helped. McCarthy said she’s frustrated that the Food and Drug Administration is approving cancer drugs without proof that they cure patients or help them live longer. […]

Pushed by patient advocates who want earlier access to medications, the Food and Drug Administration has approved a flurry of oncology drugs in recent years, giving some people with cancer a renewed sense of hope and an array of expensive new options. A few of these drugs have been clear home runs, allowing patients with limited life expectancies to live for years.

Many more drugs, however, have offered patients only marginal benefits, with no evidence that they improve survival or quality of life, said Dr. Vinay Prasad, assistant professor of medicine at the Oregon Health and Sciences University, who has written extensively about the FDA’s approval process for cancer drugs.

Overall cancer survival has barely changed over the past decade. The 72 cancer therapies approved from 2002 to 2014 gave patients only 2.1 more months of life than older drugs, according to a study in JAMA Otolaryngology-Head & Neck Surgery.

And those are the successes.

Two-thirds of cancer drugs approved in the past two years have no evidence showing that they extend survival at all, Prasad said.

The result: For every cancer patient who wins the lottery, there are many others who get little to no benefit from the latest drugs. […]

In a November study published in JAMA Internal Medicine, researcher Diana Zuckerman looked at 18 approved cancer drugs that didn’t help patients live longer. Only one had clear data showing that it improved patients’ lives, such as by relieving pain or fatigue.

Two drugs harmed quality of life. For example, thyroid cancer patients taking the most expensive drug, cabozantinib, scored worse on a scale measuring five symptoms: diarrhea, fatigue, sleep disturbance, distress, and difficult remembering, Zuckerman said. […]

“We cannot have a system where drugs that may not even work are being sold for these amazingly crazy amounts of money,” said Zuckerman, president of the National Center for Health Research, a nonprofit in Washington that aims to explain research to consumers.

Recognizing the slow pace of progress, the American Society of Clinical Oncology has set goals for new cancer drugs of extending life or controlling tumors for at least 2.5 months. The bar was set relatively low because “it’s not very often that we come across a transformative treatment,” said Dr. Sham Mailankody, an assistant attending physician and myeloma specialist at Memorial Sloan Kettering.

Yet in a study published in September in JAMA Oncology, Mailankody found that only one in five cancer drugs approved from 2014 to 2016 met those standards. […]

The FDA wants to give patients the chance to benefit as soon as possible, rather than waiting for definitive proof of improved survival, Pazdur said. In some cases, the FDA requires pharmaceutical companies to perform long-term studies after drugs are approved, to measure whether drugs live up to their early promise.

But many of these studies never provide an answer, Zuckerman said. Once a drug is approved and is available to anyone, patients have no incentive to participate in a clinical trial. So studies can end with no clear conclusion. […]

Unless the FDA requires companies to provide survival data before approving a drug, “we may never have answers,” Zuckerman said. “We will have all of these expensive drugs on the market and we will never have the information we need about how well they work or even how safe they are.”

President Donald Trump has vowed to cut regulations at the FDA and recently told pharmaceutical industry leaders that he wants to further speed up the drug approval process. […]

Read the entire article here.

Carolyn L. Johnson, The Washington Post: January 31, 2017

President Trump met with leaders of some of the world’s biggest pharmaceutical companies Tuesday and emphasized the need to lower “astronomical” drug prices, decrease regulations and bring more drug manufacturing into the United States.

Trump offered no specific policies, but mentioned increasing competition and “bidding wars” as a way to bring down prices. In the past, he has lashed out at the pharmaceutical industry for “getting away with murder” and threatened to use the government’s bargaining power to force down drug prices for programs like Medicare.

Most of Tuesday’s meeting was held behind closed doors, but Trump spoke to the media beforehand while surrounded by executives from a half-dozen large drug companies. He struck a less combative tone and didn’t mention government intervention directly.

“We have to get prices down for a lot of reasons. We have no choice,” Trump said, flanked by chief executives Kenneth Frazier of Merck and Robert Hugin of Celgene. “For Medicare, for Medicaid, we have to get the prices way down.”

In the past, pharma companies have railed against government intervention in pricing, saying those prices fund development of future, lifesaving drugs. […]

There are many empty positions at the FDA, said Diana Zuckerman, president of the National Center for Health Research. The recently enacted 21st Century Cures law provided for additional hiring authority.

“But with the hiring freeze, will they be able to hire anyone?” she asked. […]

Read the complete article here.

Diana Zuckerman, PhD, Spectrum: December 1, 2016

Why would anyone vote against “cures,” especially “21^st century cures?” That question is the key to understanding how the U.S. House of Representatives, whose members usually can’t agree on anything, overwhelmingly passed a 996-page health bill yesterday — just a few days after the bill, the 21^st Century Cures Act, was written behind closed doors.

Here’s why many health policy and consumer advocacy groups — including the National Center for Health Research, where I work — strongly oppose the bill and are asking senators not to pass the bill next week: On the bright side, the bill promises more than $6 billion dollars over the next few years for medical research and to fight the opioid epidemic. On the other hand, the ‘promise’ of that money does not include anything resembling a guarantee that the money will be provided for that purpose.

If the bill passes, those 996 pages of mostly unintelligible legislative language will influence important issues that affect all of us. Most importantly, the bill instructs the U.S. Food and Drug Administration (FDA) to help drug and device companies get their products on the market more quickly. Unfortunately, it does that by loosening and lowering the very scientific standards that have made FDA approval the gold standard for countries around the world.

A Perfect Storm of Lobbying:

More than 1,450 lobbyists pushed to get this bill passed, including many patient groups and others funded by the companies that make prescription drugs and medical devices. Universities and medical schools also lobbied for the bill because they want more funding for medical research.

But physician groups concerned about patient safety, such as the National Physicians Alliance, the American Medical Women’s Association and the American Medical Student Association, oppose the bill. Dozens of nonprofit groups such as the Consumer Reports Safe Patient Project and National Consumers League also strongly oppose the bill. In one surprising twist, some of the same HIV/AIDS activists who have criticized the FDA in years past for being too rigid are lobbying against the bill because it doesn’t do enough to make sure new treatments are safe and effective.

None of these groups oppose all aspects of the bill. They just want Congress to take the time to fix it. That would mean waiting until next year instead of rushing through a bill that nobody has time to read.

The FDA’s job is to review new medical products to make sure they are safe and effective. ‘Safe’ doesn’t offer a 100 percent guarantee: Any treatment can harm some people; if the benefits are substantial enough, the FDA may even approve a treatment that can be lethal.

But on balance, the treatment’s benefits must outweigh the risks for most people. The company that makes a treatment provides all of the studies and information about it to the FDA, and scientists there review that information. For prescription drugs, that review usually entails the FDA scientists scrutinizing data from clinical trials that compare people taking the new drug with those taking a placebo or a different treatment, such as behavioral therapy.

Lower Standards:

Traditionally, the goal of clinical trials is to see if a treatment improves the outcome for the participants. Depending on the condition being treated, better can mean living longer (for cancer, for example) or losing a few pounds (for a weight-loss drug) or being more able to enjoy life (for many psychotropic drugs and devices).

However, the new bill wants the FDA to make it easier for companies to prove a ‘benefit,’ by relying on ‘surrogate markers’ of effectiveness. Surrogate markers don’t measure benefits directly, but rather a secondary trait that is believed to predict the benefits. For diabetes, for example, blood glucose level is the surrogate endpoint, but the treatment’s goal is to avoid amputation or extend life. Better glucose levels don’t always predict those health outcomes.

Cancer drugs are a perfect example. Last December, researchers reported that the FDA approved 36 of 54 new cancer drugs more quickly by basing approval on surrogate markers such as tumor shrinkage, rather than on survival benefit¹. Years after approval, only 5 of the 36 drugs had been proven to help people live longer, 18 definitely did not, and the manufacturers of the other 13 had not made the results of their studies publicly available (a sign that the drugs probably don’t work).

Our center looked at those same 18 ineffective cancer drugs and found that only 1 improves quality of life, 15 have no proven impact on quality of life and 2 makes quality of life worse. The most expensive of these drugs, costing $170,000 per person, is one of these last two.

Spiraling Costs:

The standards for medical devices, including implants and devices that send electromagnetic pulses to the brain, are even lower than for cancer drugs. Device companies are rarely required to conduct clinical trials to prove their product is safe or effective. More than 90 percent of the time, all they need to do is explain to the FDA that their device is similar to another device already on the market.

Even when device studies are required, the companies rarely use a placebo group as a comparison to the new device. Wishful thinking can make almost any new treatment seem effective at first. That’s why so many people pay so much for treatments that don’t seem to do any good at all.

The new bill would make that situation worse, by pressuring the FDA to make it even easier for new, unproven treatments to be sold in the United States. People might end up paying billions of dollars for a wide range of treatments that were approved based on shorter studies of fewer participants and surrogate endpoints. That would increase the already-spiraling cost of insurance coverage and challenge the financial stability of Medicaid and Medicare even more than is the case today.

Don’t be distracted by the false promises of funds for medical research. The reality is that there is too much in this bill that would dismantle the structures that help physicians make informed decisions and keep us all safe.

Read original post here.