It’s hard to process what the doctor is saying: You have a disease that will rapidly paralyze you until it eventually suffocates you to death. But you are one of the lucky ones: You qualify for a clinical trial of a promising experimental drug. There is a 30% to 50% chance of receiving a placebo instead of the experimental therapy. A placebo is necessary, you learn, because each person with your disease is so different, and there are no objective measures to predict the course of your disease. Still, you are grateful to qualify for the trial; most patients don’t.
Fortunately, the trial has a design that is friendly to patients, and so six months later, after the randomized portion is complete, all patients may receive the real drug as part of what’s called an “open label extension.” Without this, you may only get the placebo. And the access to the real drug may end once the trial is complete, even if it was helping you.
was one of the 36 participants who received the drug NurOwn in a Phase 2 clinical trial for amyotrophic lateral sclerosis. Mike regained some function, but he was able to get only one dose before the trial ended. Mike and I fought for continued access to treatment and to improve an archaic regulatory pathway. He died waiting for change in 2019.
Promising therapies are on the horizon for ALS, but most people with the disease won’t live to see them. Most patients die within two to five years of diagnosis. These patients need more access to promising therapies and a more flexible regulatory process that would accommodate the moderate advances needed to get closer to a cure.
In September 2019, the Food and Drug Administration put out new guidance on developing drugs for ALS, thought to be a step toward modernizing development. The agency says that during development “sponsors should collect safety data, including data from open-label studies or expanded access programs, from patients across the spectrum of disease stages and severities.” The agency reiterated its support for regulatory flexibility when applying standards to drugs for diseases with serious unmet medical needs.
But earlier this year, the FDA told
BrainStorm Cell Therapeutics
that the company didn’t have the data to support an approval application. Earlier this month, the company Amylyx announced that the FDA wants to see data from another placebo-controlled trial on its drug candidate before an approval application.
Patients still struggle to get access to investigational drugs.
Lisa Stockman Maurillo
is a 51-year-old wife and mother of three boys. She has a fast-progressing genetic form of ALS that affects about 500 people a year in the U.S. Lisa’s diagnosis came two weeks after enrollment closed for a Phase 3 clinical trial on a promising therapy. Her physician thought the drug, Tofersen, could help her. Lisa and many others like her don’t have time to wait for a lengthy FDA approval process to play out.
the company that makes Tofersen, denied access to Lisa. The company said that offering her the drug risked the results of the trial and is unethical because trial participants may still be on placebo. But an FDA analysis revealed only two instances of a trial being put on hold because of an adverse event from an expanded access use of a drug, out of some 11,000 requests over a decade. In both cases, the events were addressed promptly and the trial holds were lifted. This week Biogen said the company would update its policy to allow more patients with the rapidly progressing form of the disease to receive the drug as early as this summer. This is welcome news, though it may be too late for Lisa.
The FDA’s guidance is an empty promise if it isn’t accompanied by action, and patients need a more flexible regulatory process moving with urgency to help find treatments and cures for this deadly disease.
Ms. Cimbura is a Denver-area English as a second language teacher.
Copyright ©2020 Dow Jones & Company, Inc. All Rights Reserved. 87990cbe856818d5eddac44c7b1cdeb8
Appeared in the April 27, 2021, print edition.
All news and articles are copyrighted to the respective authors and/or News Broadcasters. VIXC.Com is an independent Online News Aggregator
Read more from original source here…