The People's Perspective on Medicine

Should Desperate Patients Have the Right To Try Unproven Drugs?

Many people with a terminal illness are willing to take almost any risk to improve their odds. Will the new right to try bill passed by Congress help? What are the downsides?

Most people empathize with the plight of desperate patients. Those who have exhausted all available treatments are willing to try almost anything. That is why Congress has passed the “right to try” bill. Dying patients will be allowed to obtain experimental medications even if they have not been approved by the FDA. President Trump is an enthusiastic booster of this legislation. He is expected to sign the bill into law promptly.

Challenges to Right To Try:

On the surface it would seem that this is a patient friendly initiative. There are challenges, however.  First, a doctor has to be on board with the experimental therapy. Many physicians will be reluctant to embrace experimental drugs that have little evidence of effectiveness or safety.  Second, the drug company has to be willing to supply it to desperate patients. That is not a given.

Who Will Pay?

Will drug companies agree to supply an experimental medicine? Will they charge? If so, how much? Will Medicare, Medicaid or insurance companies pick up the tab? Probably not. Most payers refuse to pay for expensive medications that have not been approved for a specific illness.

In other words, a new cancer drug may have FDA’s blessing for one kind of cancer, say lymphoma. The insurance company may cover that particular disease. There may be experimental data to suggest it would also work against breast cancer, but the insurance company could well refuse to pay if the FDA has not yet approved the drug for that use. If the drug costs $150,000 a year but a patient cannot get financial support, access may be meaningless.

The Dark Side of Right to Try:

Even though desperate people are willing to try almost anything to extend their lives or the lives of those they love, there is a downside to “right to try.” Keep in mind that these drugs will not have passed FDA review. That means there may be little, if any, evidence that they are safe or effective for the condition in question.

The counter argument is that if you are drowning, you need a life saver, even if it has not been proven effective. The trouble with this position is that if you are thrown an anvil instead of a life preserver, you could well sink very quickly without a trace. Some experimental drugs may actually make the condition worse and speed death. It’s entirely possible that the end game could be far worse than normal.

Who’s For Right to Try?

The Senator who sponsored the right to try bill says that it provides:

“hope for terminally ill patients who have nowhere else to turn.”

That sounds noble. Critics say that such patients already have the right to request experimental drugs through a compassionate use program that the FDA already supervises. According to experts, the overwhelming majority of requests are approved.

We find it fascinating that the chief umbrella organization for drug companies, The Pharmaceutical Research and Manufacturers of America, has not taken a stance in favor of the right to try bill.

The legislation provides legal protection against litigation. If an unapproved or experimental medicine harms a patient or shortens his life, it is unlikely there could be a lawsuit against the drug company. Even so, there is no certainty that a pharmaceutical firm would provide an exciting new drug to anyone at an affordable price.

People’s Pharmacy Perspective:

We understand what it means to be desperate. When I (Joe) was young, I had polio. I was surrounded by dying children in an isolation ward in a Philadelphia hospital. My parents would have done anything to save my life or speed my recovery. (I did recover even without a medical breakthrough).

Close friends and family members have died from cancer and severe neurological diseases. We recognize that people are willing to take huge risks when faced with terminal illnesses. Sadly, there are few true miracle medicines. The overwhelming majority of drugs tested for Alzheimer’s disease have flamed out. Not only haven’t they helped, some have caused substantial harm.

We do believe in the FDA’s fast track process that accelerates the approval of true breakthrough medicines. We want some evidence that a new drug will work for its intended purpose and will not make a patient worse. The expanded access (aka compassionate use program) already provides desperate patients a way to get experimental and unproven drugs.

We believe that drug companies should provide such medicines to patients at either no cost or an affordable price. Careful records of both benefits and risks should be made available to everyone–doctors, patients, researchers and FDA officials. Only then can people make informed decisions about these experimental compounds.

What Do You Think?

Please share your thoughts about the right to try legislation. Is this a good idea or a potential boondoggle? We would love to read your opinion in the comment section below.

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About the Author
Joe Graedon is a pharmacologist who has dedicated his career to making drug information understandable to consumers. His best-selling book, The People’s Pharmacy, was published in 1976 and led to a syndicated newspaper column, syndicated public radio show and web site. In 2006, Long Island University awarded him an honorary doctorate as “one of the country's leading drug experts for the consumer.” .
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I think people in a terminal or hopeless situation should have the right to try something experimental.

It’s my body and my choice what I do with it. If I want to try any non-FDA approved drug to try to extend my life, I should be able to take it. If it lengthens my life, great! If it does not, so be it. We are all going to die anyway and we should learn to accept it. Remember, the FDA has approved drugs which have turned out to not be so great and in some instances, been detrimental to one’s health. Doctors used to give Zoloft to many people, but they discovered that it interacted horribly with alcohol and other drugs and caused confusion and psychosis.

If an unapproved drug was given to a desperate person and found to help (beyond a placebo effect), the company would likely be flooded with requests for the drug when they lacked the ability to supply it, and certainly before they knew how to price it.

Hi,
I read most of the articles in this on-line publication, great. The best thing is the print is big enough to read with out a magnifying glass.
Thanks

Allowing patients who are desperate to live, is in my opinion, with informed consent, and I don’t mean signing a piece of paper without knowing what it means, a program that I support.

Pharmaceutical companies will eventually, after trials, either be granted the stamp of approval by the FDA or not, depending on the outcome of the drugs efficacy. Therefore, the experimental drugs should be paid for by companies who are in the process of receiving FDA approval, not the patient(s), and not Medicare or Medicaid, nor should anyone who can’t afford the drug be charged for it. The new law will give manufacturers yet another loophole to hasten the approval/non-approval of a drug, something they enjoy under other circumstances at present, i.e. promising to do an additional study after the patent is up on a particular drug, that results in the company not having to start at step one with the FDA again.

Informed consent is critical, and I believe there should be an impartial MD with the patient, who deals with the patients disease, to make certain they understand what the informed consent covers, what it may or may not do, and what costs, if involved, such as overnight or longer stays at hospitals, charges for administering the drug, and care for the patient while in the hospital. Some people are medically savvy, while others depend on MD’s for information – this is why an independent evaluation of the patients disease is necessary, with the patient present to ask and have questions answered.

I see Compassionate Use of drugs as a right, as I believe in the Right to Die program in the State of WA is a right that all who wish to die in this manner have access to, at present. A Volunteer Oversight Committee should be put into place where non-FDA approved drugs will be administered, and should be charged with evaluating each drug and patient on an individual case basis, and if this isn’t possible, then the patient must know what he/she may experience, both good and bad, if used to prolong or extend life.

The drug/medication companies have a massive influence on congress and the medical practice itself. The TV ads for medications are constant. This reveals that the drug companies are making massive profits from the sale of these medications. Some advertisements even state “once you take our product, you cannot stop” This is analogous to the ruthless drug cartels that get people hooked on their poison. Most TV ads proclaim “Ask, your doctor,” “Ask your doctor.” Then they quickly go through the deadly “side affects” on the ad. Those are just summaries of the “side affects” People have the responsibility to stay informed about the side affects of the prescribed and not prescribed medications.

For example: The ads for the drug for dry mouth: The natural alternative is to drink water; however, the drug companies will not make billions from people who drink water.

It is the rule rather than the exception that the drug/medication industry is primarily motivated by avarice and greed. And the “new” medications without thorough testing and final approval are a roll of the dice.

I am in favor of trying any drug which might save a life. These drugs should be provided at no cost and if they are successful, the drug Companies will prosper. If one has a terminal illness, that person should have the right to make a decision with or without a Doctor’s say.

I think this is smart move and that every person should have the right to try anything they choose in the hopes of trying to save their own life. I am 100% for this.

The Seattle Pacific NW magazine had an article on cancer research from Fred Hutchinson research center. An extremely positive result has been achieved with enhancing the body’s own T-cells. I would want to try that if I had a grim prognosis.

“We believe that drug companies should provide such medicines to patients at either no cost or an affordable price. Careful records of both benefits and risks should be made available to everyone–doctors, patients, researchers and FDA officials.”

I agree wholeheartedly!

If I had a terminal disease and there was an opportunity to try an unapproved drug for a cure, I would ask myself two questions. 1. If the drug worked, would it stabilize my position or might it improve it? If I am in dreadful pain would I want a drug that just prolongs pain for some additional time. 2.

If I took the drug and it shortened my life I would be very glad to be out of pain or whatever other discomfort that there may be that might just drag on for an indefinite period of time. On balance, if I was terminally ill, in pain or other severe discomfort I would welcome the chance a new drug might improve my position.

If not, well at least it would shorten the amount of pain or discomfort I might otherwise have to look forward to. As for cost, I think the drug companies would welcome test cases to assist in their eventual approval by the FDA, so should write off the cost as part of that drug development program.

Is this a method to shortcut & save money on research for the already rich pharmaceutical industry?

Will this become a “wealthy only need apply” program? What effect will participation have on payment of life insurance?

I may be wrong but it seems like you are criticizing something that, regardless of its many drawbacks, may help some people. True, under this bill very few may be aided by its provisions, and, if they do use its provisions they should do so with their eyes wide open. So, yes, it is what it is. Yet it may help a few. –I don’t understand your critical tone here.

The drug companies must love this legislation. It allows them to make a claim a drug will help a terminal ill patient without any studies proving the claim. The drug company simply declares it will work. They also can charge a patient for further research instead of paying a patient to be in a research trial. And they cannot be sued for any problems occurring with its use.

Wow. Is this the way of the future for the leader of the world in drug research ? Who is more desperate for this legislation- patients or drug companies?

These meds have probably been used already in more PROGRESSIVE countries, which means they’ve already been well-tested. By the time FDA finishes diddling around, too many more people have died. If the patient wants it, they should have access without having to cross any borders.

I would be interested in a follow-up / companion article to this that discusses the compassionate use program.

Was told I have terminal cancer 6 years ago. Given 3 to 5 years to live. Can be treated but not cured. I was able to get into a clinical trial and still in remission. When told you are done, you will try anything. People are going out of the country.

Our Congress is no longer by the people or for the people. It is controlled by big money, insurance, drug, and legal entities. Isport your perspective.

What worries me is the possibility that somehow this is going to add to the high costs of approved prescription drugs. We’re already getting hit hard for R&D. How might this impact that?

If a patient is willing to act as a guinea pig, the drug company must provide the drug at no cost to him.

In this program drugs that have some evidence of efficacy and have some toxicity data, even though official regulatory clearance has not yet been obtained, would be the one’s logically chosen by physicians. Payments will have to be made by patients, their families, special appeals or charities …

One would think that the drug manufactures would be on board to have the patient try new drugs, this would work both ways. The patient end result and the drug company could have the drug tested by the patient without fear of being sued if the drug may help the patient or the drug may not.

Latrile anyone? Or any number of OTC diet pills that have come and gone in large numbers over the years. Ultimately they were at best ineffective. Remember Phen-fen, the use of a combination of 2 prescription diet pills for weight loss? Worked good for weight loss, not good for the negative effects it caused in many people.

Even well-tested drugs have been removed from the market when side effects popped up when used in the general population that weren’t manifested in trials. Noble in purpose, I don’t think this law is going to benefit a substantial amount of people. Too many pitfalls, and way more liable to be detrimental rather than beneficial. Often what looks good on paper turns out to be bad idea in practice.

I am not sure what is the best answer for this problem. My brother was accepted for a cancer trial. It did not cost him anything. The cancer went away for about two years. It did come back but he lived that time cancer free.

Not everyone qualifies for these trials. People have been going to other countries for treatments of illnesses that doctors will not use in this country. Desperation changes everything.

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