Should Congress allow the terminally ill to buy experimental drugs not yet approved by the FDA?

Yes: Right-to-try laws give last-minute reprieve to many on death’s doorstep

After baby Eli was born, his family received the devastating news that he had a fatal type of spinal muscular atrophy.

With a disease that damages the motor nerve cells in the spinal cord, ultimately taking away the ability to eat and breathe, Eli was not expected to live long enough to celebrate his first birthday.

Eli, however, is now 2 years old. He talks and sings and, with the help of a walker, can walk.

He was among a fortunate group of 15 children who gained access to a clinical trial. While Eli received a partial dose of the investigational treatment used on the group, the infants who received full-dose treatments are not showing any disease symptoms today.

Terminal patients like Eli don’t have time to wait for the bureaucratic sanctioning of promising new treatments, which take, on average, more than a decade to obtain Food and Drug Administration approval.

That is why a federal version of what’s called a right-to-try law is needed. Under these laws, terminally ill patients under the care of a doctor who has exhausted other treatment options, are permitted to seek treatments that have successfully completed basic safety testing on humans in Phase 1 FDA trials and are continuing on the path to full FDA approval.

Such laws are now on the books in 33 states and under consideration in the other 17. Terminal patients understand that these laws don’t offer guarantees; but they do offer hope.

A Texas oncologist told a U.S. Senate committee last year he had already treated 78 patients under Texas’ right-to-try law. Some had only three months to live when they began treatment. Today, months later, most are doing very well.

Unfortunately, there is fear in the medical and manufacturing communities that the FDA could retaliate against those using these laws.

That is why a federal right-to-try law is needed. A federal law would, simply, require the federal government to abide by the state right-to-try laws.

Some opponents fear such laws are too risky. But these laws work in tandem with the human safety requirement used by the FDA. If the treatment is safe enough to use on others in clinical trials, why isn’t it safe enough for a terminal patient under their own doctor’s care?

Others fear that manufacturers could use such laws to sell their treatments. But federal law already makes it illegal to provide investigational treatments for more than the actual documented cost.

In reality, most manufacturers that participate elect to provide the treatment for free because they are reluctant to make their cost information public.

Other critics point out that the FDA’s compassionate use program, which is supposed to give patients access to investigational treatments, already provides such access to terminal patients. But the FDA granted fewer than 1,300 such requests in 2015.

That same year, more than 1.3 million died from the three leading disease killers: heart disease, cancer and chronic obstructive pulmonary disease. That does not include other devastating diseases, such as Alzheimer’s, Lou Gehrig’s disease and Parkinson’s.

Something is clearly amiss in a system that is so bureaucratic and time-consuming that fewer than one-tenth of 1 percent of terminal patients can take advantage of the FDA’s compassionate use exception.

Federal right-to-try legislation would make compassionate use the rule not the exception in all 50 states – not just the 33 that already have passed it.

No one should be forced to beg the federal government for permission to try to save their own lives. Families like Eli’s or maybe even yours are why we need a federal right-to-try law.

No: Right to Try laws allow Big Pharma to exploit false hope

By Diana Zuckerman

Tribune News Service

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.

This story was originally published March 17, 2017 at 4:46 AM with the headline "Should Congress allow the terminally ill to buy experimental drugs not yet approved by the FDA?."