Video of Mark Bedwell’s dramatic demonstration has been circulated widely on the internet and on television newscasts. Bedwell was making the case for FDA reforms championed by the “free to choose” and “right to try (RTT)” movements, which want to speed up the agency’s drug approval process which can take longer than many patients are expected to live. The panel applauded after Bedwell got up and did his lap around the conference tables.
“It worked,” a tearful Bedwell told the panel. “It was hard for me to walk, hard for me to talk. I’m very emotional about it. It works. I’ll run around the building with you.”
The experimental treatment Bedwell has been using is called NurOwn, by Brainstorm Therapeutics. NurOwn is a targeted cellular therapy designed to slow down the progression of ALS. It completed three Phase 2 FDA trials involving 70 patients and is now beginning Phase 3 trials with 200 patients. Bedwell participated in one of the completed trials.
Says FDA Excludes Too Many
Annie Swarts, an ALS activist, says the FDA’s drug approval process system for new drugs like NurOwn is outdated and excludes too many patients.
“The guidance documents are so archaic,” said Swarts. “They’re from the 1950s. They rule out a lot of people, right off the bat.”
Guidance documents are intended to encourage and guide prescription drug makers through trials. Swarts says the ALS community is being ill-served by the FDA.
“The FDA doesn’t have a point person for ALS,” said Swarts. “We’ve been failed on a lot of levels.”
‘Right to Try’ Falls Short
ALS patients have been trying to convince regulators that NurOwn should fall under RTT legislation President Trump signed into law in May 2018. Forty-one states had passed their own RTT laws, whereas S. 204 creates a uniform system for terminally ill patients who have exhausted treatment options and cannot get into clinical trials. The FDA has given such permission under its “expanded access” clause and other parallel programs, but many patients fall outside the eligibility requirements.
RTT drugs are generally in an earlier stage of development than the FDA’s access framework allows. RTT also grants liability protection to companies formulating the new drugs so access is not withheld out of fear of lawsuits. RTT drugs can be seen as a last resort, and many ALS patients and other terminally ill individuals feel they have no other options.
Christina Herrin, campaign manager for the Free to Choose Medicine initiative at The Heartland Institute, which publishes Health Care News, says there is good reason for their frustration.
“Twelve years is simply too long to get a drug approved,” said Herrin. “Instead of having the FDA pore over hypotheticals, we need to be helping these people.”
Government Obstacles to Drug Access
The FDA process is intended to ensure patient safety and efficacy for new drugs, which leads to long trial times and extensive periods of review. Experimental access is granted for drugs that have already passed safety trials.
The average cost for getting a new drug to market is $2.6 billion, according to a recent study by the Tufts Center for the Study of Drug Development. The cost may make some companies wary of the FDA process, as they are unsure their costs can be recuperated should early access disprove efficacy projections, or should the FDA refuse to approve the drug.
Bartley Madden, author of Free to Choose Medicineand a policy advisor to The Heartland Institute, writes how expanded access would work.
Free to Choose “sets up a competing system that would allow manufacturers to market new drugs after completion of the FDA’s Phase I testing and at least one round of Phase II testing, contingent upon certification of a FTCM Committee that the drug successfully completed that testing,” Madden proposes in his book. Patients could make drug treatment decisions based on their individual circumstances, and their experience would provide real-life data so drugs could get to market faster if they prove safe and effective.
Public Supports Faster Access
A poll by The Heartland Institute found 92.8 percent of active voters support implementing a faster FDA process for getting prescription drugs to market.
“This is a bipartisan issue,” said Herrin “Comprehensive reform is being demanded by the American people with 95 percent of Americans supporting patients and their doctors making the decisions regarding their health care, not the government.”
Swartz says a faster process is critical for those with terminal diseases such as ALS. “We need options, and if we keep waiting for that magic bullet, we’re never going to get any options,” said Swartz. “The concern about safety doesn’t always matter when you have a terminal disease. It will either work or it won’t.”
Getting drugs to patients faster has an added benefit, says Herron. “The goal of the Free to Choose Medicine Solution is to make drugs fail faster and succeed sooner. The current drug approval process is convoluted, lengthy and extremely expensive. In a country that prides itself on individual freedom, no American should be forced to die because government regulations can’t keep up with the medical advancements of the private sector.”
Andrew Whitney ([email protected]) writes from Lansing, Michigan.
Arkansas man with ALS runs in front of FDA staff, April 9, 2019
Tufts CSDD R&D Study, March 9, 2016
Free to Choose Medicine