Hi. I'm Art Caplan. I'm at the Division of Medical Ethics at the Grossman School of Medicine at NYU in New York City.
Amyotrophic lateral sclerosis (ALS) is a horrible disease. Some of us know it as Lou Gehrig disease, and it involves creeping paralysis, certain death, horrible disability, confinement to a wheelchair, and many people wind up only able to operate a computer screen to message. It is just a miserable disease. People have been trying to find ways to intervene, to fix it, to cure it, and to slow it.
Unfortunately, some patients, families, and some others believe that the biggest obstacle to getting the answer to ALS is the FDA and regulations. They believe that regulations are causing bureaucrats to slow down potentially beneficial interventions.
They believe that regulations and the FDA are indifferent to the suffering of patients, paying more attention to data analysis, wondering more about the certainty with which a particular claimed cure or intervention really can help, and not letting patients who are desperate make or take a risk-benefit choice, given their dire straits, that they are willing to try things and to gamble that something might help them. Even as some patients say: If it kills them or they die more quickly… Well, the disease is so bad that they would like to be able to make that choice.
Legislation has been introduced in the House of the Senate called the Promising Pathway Act, basically pushed forward by many ALS organizations, that would expedite a review by the FDA. It says the FDA should establish a rolling, real-time review, meaning taking a look every day, technically, at drugs that are intended to treat, prevent, or diagnose life-threatening diseases.
On this pathway, the FDA could grant a sort of limited approval, letting a drug that has some evidence of safety get out to the public and maybe has some promise that there's efficacy. The way that would be determined is not by a clinical trial, not by a study, but basically by real-world evidence, including claims from patients and family members that they feel better, they think they're doing better, and that they think the drug that a company is trying to get approval to sell is really doing something.
I think this is wrong, and it really is painful for me to say it. I think the difficulty with fighting and finding help for those with ALS or preventing it is not the FDA, it's the science. We just don't understand this disease very well. The science just isn't in a position yet to say we're going to be able to offer some benefits. Various drugs have been brought forward and presented to the FDA. The experts have taken a look at them, and they say they're just not convincing in terms of conveying any real benefit.
Desperation, of course, makes people want to try anything, but it doesn't mean that the science that's out there supports it nor does it mean that entrepreneurs and investors who want to sell things to ALS patients, who want to get the government to approve reimbursement for their drugs, are really telling us honestly what they are capable of doing. Sometimes, even they work with patient groups to try to get them to agitate Congress, the FDA, or both to provide approval so they can then get reimbursed.
This is a tough area. There are many little companies that, if they don't get their drug approved for some use, are not going to make it. The company won't survive because it doesn't have much money. There are other situations, where patients are saying, don't protect us from harm or a lack of efficacy. There are still others saying, look, if you approve some of these drugs and the price tags are hundreds of thousands of dollars per patient or millions, you're going to wind up spending a fortune on something that may not work at all.
I don't think the time has come to pass the Promising Pathway Act to try to have temporary approvals, less evidence, and allow more sketchy claims to be put forward as a basis for a temporary approval of a drug.
I think we still have to be tough and say that we need to get drugs into controlled clinical trials, then use claims from patients, real-world evidence, to supplement what the data are in the trials. There's no replacing clinical trials of drugs with patient stories and narratives built up by the company. That's just not the way to get the public's or the patients' interests served at a time when the science is still tough and not there for cracking ALS.
It's sad to say it, but I wouldn't put my money into efforts to reduce the bureaucracy. I'd put my money into more research, more rapid research, and bigger research projects.
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