Right to Try

Right To Try Laws Are a Fine Start. Comprehensive Reform of the FDA's Drug Trials Would Be Better.

The drug regulator's clinical trials process for approving drugs needs a complete overhaul.

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In his State of the Union address this week, President Trump urged Congress to pass right-to-try legislation that would allow patients suffering from terminal illnesses to access new drugs and other treatments that have undergone preliminary Food and Drug Administration (FDA) safety trials, but have not yet been shown to be efficacious in clinical trials. It's a good start. But it's no substitute for a top-down overhaul of the agency's entire process for testing and approving new drugs.

Traditionally, patients only get access to new drug treatments once they are approved by FDA after passing through three phases of clinical trials. The goal of phase 1 trials is to test new compounds for patient tolerabilty and safety. Typically 20 to 100 patients participate in Phase 1 trials. Phase 2 trials aim to determine proper dosage levels for new treatments while also checking for further evidence that the new compound is safe and possibly efficacious. Several hundred patients may be enrolled in Phase 2 trials. Phase 3 trials focus on efficacy and monitor for adverse reactions and may involve thousands of participants. In Phase 3 trials, the efficacy of new medications is often benchmarked against current treatments.

One recent report estimates that only 1 in 10 new drugs that enter clinical trials are eventually approved by the FDA. It is noteworthy that only 31 percent of drugs that currently enter Phase 2 studies go on to Phase 3 trials. In other words, more than two-thirds of biopharmaceuticals that pass through Phase 1 safety trials end up being deemed insufficiently efficacious as treatments for the diseases at which they are targeted.

So far, 38 states have passed right-to-try legislation. Since drugs and medical treatments are regulated at the federal level, state laws so far have had little apparent effect on enabling patients gain access to experimental treatments. Consequently, right-to-try proponents want Congress to pass legislation that would allow patients with terminal illnesses to seek access to experimental drugs that have passed through Phase 1 safety trials.

Many drugmakers have been reluctant to offer treatments not yet approved by the FDA on a right-to-try basis for fear being sued by patients or their heirs should bad outcomes occur. In addition, they worry that any adverse events among right-to-try patients would delay eventual FDA approval. Consequently, the proposed federal legislation provides that the makers of experimental drugs could not be held liable by patients for any untoward outcomes and that FDA regulators would be barred from taking into account the results of right-to-try treatments when reviewing such drugs for approval.

Opponents of right-to-try legislation fear that it would enable unscrupulous practitioners to sell the moral equivalent of snake oil to desperate people. In addition, opponents point out that the FDA already has a compassionate use program that allows patients and their physicians expanded access to investigational drugs.

New FDA administrator Scott Gottlieb recently announced changes that aim to speed up the process of providing access to drugs and devices for patients with serious conditions (generally prior to product approval), when there is no therapeutic alternative.

In addition, the agency provided guidance to drugmakers clarifying that suspected adverse reactions from experimental treatments administered on a right-to-try basis must be reported "only if there is evidence to suggest a causal relationship between the drug and the adverse event." The FDA does also note that greenlighting access to experimental treatments does not require drugmakers to provide them to patients.

Right-to-try is at best a band-aid. Comprehensive reform of the clinical trial process is a better and cheaper way to speed new medications to the bedsides of patients. As I have earlier argued:

The FDA should be modernized so that new treatments become available to patients once they have made it through the Phase II safety testing. Patients who choose the new treatments would essentially be enrolled in Phase III efficacy trials. This would drastically cut the time and the expense it takes to get new medicines to people.

I am not alone in urging this reform of the drug approval process. In a February 14, 2012 Wall Street Journal op-ed, former FDA Commissioner Andrew von Eschenbach argued that "after proof of concept and safety testing, the [new therapeutic] product could be approved for marketing with every eligible patient entered in a registry so the company and the FDA can establish efficacy through post-market studies." Elsewhere von Eschenbach pointed out this FDA reform would mean that new drugs could…

…come to market after promising early-stage research in targeted patients, with appropriate post-marketing studies required. Payers and patients would be the ultimate judge about the quality of the product, and companies could learn from the experience to develop superior products if needed.

Companies would still be liable for unforeseen side effects, but patients and doctors would be warned—through the drug's labeling—that the product had been approved based on promising but provisional research.

Gradually replacing or reducing dependence on Phase 3 trials with smaller, faster adaptive trials and post-market surveillance would have a positive impact on medical innovation and the U.S. economy….

Ultimately, the molecular bases of health and illness will be unraveled later in this century, at which point the hit-or-miss process of using clinical trials to evaluate medicines will be superseded by precisely targeted individualized treatments.

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  1. And when people hear Trump say that drug prices are too high, he’s probably thinking price controls, which would obviously be a huge mistake. Someone needs to get in his ear that the way to lower drug prices is to lower the costs of getting them approved.

    1. That too is my fear with Trump. He has a good eye for seeing a problem but sometimes his solutions are just as bad or worse then the problem.

      1. Isn’t that true of most people?

    2. Metformin. A drug that’s the most commonly prescribed drug for diabetes, which has been used in the US for more than 20 years, is safer than ibuprofen or aspirin, is used by 50 million Americans. It’s even cited as useful in preventing diabetes in pre-diabetics. The FDA still requires a doctor’s prescription. What happens when a person has to take two hours out of their day to attend a doctor’s appointment, pay $100 for the visit and waste 15 minutes of the doctor’s time writing a prescription? It costs taxpayers $5,000,000,000, eats up 100 MILLION man-hours of their time and wastes 6,250 doctor YEARS every year.

      Think about that. The government’s nonsense requirement that you get a prescription for Metformin means over 6,000 doctors are tied up all year just doing the stupid government-required paperwork for a drug that’s safer than aspirin.

      1. FDA’s excuse for this is language Congress put in the FFDCA re judgment of safety & efficacy of drugs & med’l devices: that metformin is for a serious disease, therefore people concerned about it should be seeing a doc. The experts say some diseases are not suited to self-care.

    3. CPAP equipment. Many Americans have sleep disorders that are easily treated with CPAP machines. The consequences of NOT treating sleep apnea can range from disorientation and fatigue to heart disease and death. The remedy is simple – a system that blows low-pressure air into your nose to keep the airways inflated so they don’t close down and starve your brain of oxygen while you’re asleep. The amount of pressure is about the same as if you went snorkeling at a depth of a foot or two, took a breath and held it to the surface. MAXIMUM! A person puts more pressure on their lungs when they blow up a child’s party balloon.

      In short – IT IS NOT DANGEROUS IN ANY WAY.

      But the FDA requires prescriptions for CPAP equipment, which drives its cost up by hundreds of dollars and makes the life-saving technology unavailable to many people.

      That’s right, people, the FDA is KILLING people by denying them simple, safe technology that could prevent serious health issues.

      Because of the FDA, people with sleep disorders (an estimated 22 million people), must have “sleep assessments” (which typically cost more than a thousand dollars) and spend hundreds or thousands of dollars on equipment which, if not government-controlled, would be available at Wal-Mart without a prescription for less than $100.

      1. Blame Congress, not FDA. FDA’s just enforcing the legisl’n, which says that a condition w serious consequences like sleep apnea, or who think they have such a condition, should have people getting professional advice, not caring for themselves by getting an OTC product for it.

  2. Effectively the blogger’s proposal turns phase 3 into 4, & phase 2 into 3. I’m afraid something like that’s not going to fly, because if that notion were widely enough acceptable it wouldn’t be necessary, because much deeper reform/abolition would be equally acceptable.

    I can think of a bunch of lesser but still significant reforms. One would be to adopt a statute that says that for purposes of Titles 20 & 21 of the USC, a product’s use could not be inferred as “intended” w/o a positive statement to that effect by its purveyor. That’d allow for a whole underground of alternative rxs as people discover possible uses of products, & get the sellers off the hook for selling them as categories regulated by FDA, EPA, or to some extent (analogs) DEA. But it’d preserve the fx of the regulators in preventing claims for products that are insufficiently supported.

    A little one would be to adopt a statement that “soap” includes any article the customer would think of as such; it’d deregulate some articles currently deemed or assumed to be “cosmetics”, such as shampoo.

  3. Better yet – get rid of the FDA completely. Get rid of the requirement for prescriptions. Let people take any drug they want whenever they want for any reason – or no reason at all.

    When you get right down to it, the FDA increases the price of drugs, limits the application, fails to detect serious side effects and generally just costs money and kills people.

    1. How you gonna get Congress to go w something like that? How you gonna get the voters to? Of all the fields of regul’n, food & drugs has the strongest support. You could deregulate almost everything else 1st. People might even go for free banking before they’ll go for free drugs & med’l devices.

  4. What a sad commentary on a society that we need a law to give individuals the “right” to try potentially life-saving medications. Sadder still is that it’s not the least bit surprising.

  5. I made a comment relative to the earlier story on licensing that applies here as well. Asking why government restricts our behavior leads to the concept of limiting government to assisting us in making decisions wisely but excluding government from controlling what those decisions should be. I hope you will read that comment as well, It is summarized in the slogan “INFORM, NOT ENFORCE” .

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