“Right-to-try should not be right-to-die-poorer”

“The newly enacted right-to-try law allows drug makers to earn a profit by selling unproven therapies to desperate and dying patients. The FDA is powerless to stop it.
You’d think no drug maker would be dumb enough to actually try to make money this way, given prevailing public opinion that already views the drug industry as greedy, price-gouging profiteers.
But you’d be wrong. Enter BrainStorm Cell Therapeutics, which intends to start a “semi-commercial enterprise with modest profits” that would sell its experimental stem cell therapy to patients with amyotrophic lateral sclerosis, or ALS, according to a story from Bloomberg…
But the precedent that BrainStorm will set by turning what should be an altruistic act into a money-making venture is extremely dangerous and potentially exploitive. Unscrupulous drug companies will be incentivized to skip the traditional clinical trial and FDA review process altogether. Drugs no better than snake oil — but deemed safe — could be sold to desperate patients at a profit. The FDA would have no power to stop it from happening…
There’s little evidence that ALS patients, especially those with advanced disease, will benefit from NurOwn. In BrainStorm’s Phase 2 study, NurOwn was unable to slow the progression of ALS compared with a placebo. The company turned to a responder analysis to eke out a signal of efficacy, which it’s now trying to confirm in the Phase 3 study.
The idea that BrainStorm could make a profit from NurOwn before the treatment is proven effective and approved by the FDA is a bad look for the company and the entire pharmaceutical industry. It reeks of opportunism, even when couched in compassionate rhetoric.
Right-to-try should not be right-to-die-poorer, but that’s what the law will end up being for patients if profit motive takes hold.” (A)

The balance between investigational new drug access and protection of patients from therapies without established safety the FDA built during the past 20 years could be compromised by the new Right to Try law, a new study in JAMA Network Open suggests.
“To our knowledge, no studies have examined the timing and duration of drugs made available through expanded access programs to determine whether the program was serving its original purpose,” Jeremy Puthumana, MS, of the division of medical ethics at Yale School of Medicine, and colleagues wrote.
Researchers used ClinicalTrials.gov to locate investigational medicines made obtainable through expanded access and compassionate use programs prior to FDA approval and ascertained the start date of each program and several “key regulatory dates” — investigational new drug application activation, initial NDA submission and FDA approval — and timing and duration of expanded access availability in regard to NDA submission and FDA approval…
“For medicines that ultimately receive FDA approval, these findings suggest that the FDA and pharmaceutical industry have established a balance between investigational new drug access and protection of patients from therapies without established safety,” Puthumana and colleagues wrote.
“This balance may be compromised by policymakers seeking to speed access to investigational medicines through the Right to Try Act,” they added.
Despite more than 100 advocacy groups, including the American Lung Association and American Society of Clinical Oncology sending a letter to Congress strongly opposing the law, the bill passed both chambers. President Donald J. Trump signed Right to Try into law on May 30. An expert who spoke to Healio before the bill signing indicated the effect of the law on patients remained unclear.” (B)

“In essence,” says Klein, “patients and their doctors have had the ‘right to try’ investigational agents when all else fails and the company is willing to make the product available, for decades.”
For more than 30 years, people with serious diseases have had access to investigational drugs, vaccines, devices and biologics via the FDA’s expanded access pathway. Under the current program, which is also referred to as compassionate use, once a company agrees to provide an investigational product, the FDA and an institutional review board (IRB) must approve the product before it is administered to the patient. And the FDA retains the right to deny certain requests for drugs that it has not yet approved for safe use within the general population.
“Expanded access is really a ‘pathway,’ a regulatory process that permits companies to provide unapproved therapeutic agents to a patient if the company decides to do so. There are many reasons companies might say no. But the FDA allows more than 99 percent of requests to go forward. The small number of situations where the FDA doesn’t allow the expanded access use of the drug to proceed involves situations where there is a known risk that outweighs the potential benefit for the patient,” says Klein.
The expanded access program began in 1987 during the HIV/AIDS epidemic, when sick patients demanded that protocols be established for those not enrolled in a clinical trial who wanted to try potentially life-saving medications when nothing else was available.”..
Klein offers this scenario of how it works: “Suppose a patient came into the hospital late at night and had a stroke and the doctor thought a particular investigational drug was an appropriate option for this particular patient. The physician could contact the emergency call center, which would contact the medical officer on duty. They would review the case, ask a few pointed questions as necessary and provide an IND number by email or possibly over the phone. The IND allows the investigational product to be administered.” (C)

“Anyone with a smidgen of knowledge about healthcare understood that the right-to-try legislation signed by President Trump on Wednesday was a scam, perpetrated by the Koch brothers and their henchmen.
Masquerading as a “compassionate” measure aimed at providing victims of terminal diseases with a last bit of hope that an experimental treatment might save them, it really was aimed at undermining the authority of the Food and Drug Administration to make sure our drugs are safe and effective.
Among those who bought into this pretense out of sheer ignorance was Trump, who claimed the measure would save “hundreds of thousands” lives, which was just fantasy.
This law intends to diminish the FDA’s power over people’s lives, not increase it.
Now, the measure’s chief sponsor has pulled open the curtain, so that even laypeople can understand how horrible this law is. He’s Sen. Ron Johnson, R-Wisc. In a letter Thursday to FDA Commissioner Scott Gottlieb, who was critical of the law, Johnson wrote:
“This law intends to diminish the FDA’s power over people’s lives, not increase it.”
Apparently under the misbegotten impression that he was doing the public a favor, Johnson proceeded to underscore some of the more egregious provisions of his own bill. Among them is a prohibition against the FDA’s using clinical results from these last-chance treatments as the drugs continue through its regulatory process.
Put simply, if a drug is found not to work or even to be harmful in right-to-try cases — findings the FDA normally considers of paramount importance in judging a drug’s fitness for approval — the FDA can’t use those findings in the approval process.”
FDA Commissioner Gottlieb drew Johnson’s ire by suggesting, just prior to the bill’s passage, that his agency would have to promulgate guidance and regulations to balance the measure’s broadening of access to experimental drugs with “appropriate patient protections.”
Johnson made clear that he has no use for any additional patient protections. His bill, he wrote, “is not meant to grant FDA more power or enable the FDA to write new guidance, rules, or regulations.” (D)

“So what’s the catch? Did something just happen that everyone loves, and will save hundreds of thousands of lives?
If that were the case, of course, the bill would’ve passed long ago. In fact, many ethicists and doctors and patient advocates quite emphatically oppose it, as do former FDA commissioners. The American Society of Clinical Oncology is among nearly 40 health organizations that have publicly dragged the bill, saying it “could do more harm than good to seriously ill patients.” In a February letter to Congress, the groups reminded legislators that the current regulatory system for medical products was created as a result of serious harm and exploitation that occurred early in the 20th century: “Birth defects resulting from Thalidomide are an example of what happens when drugs are given to humans without proper safety review and approval.”
The legislation is a product of the conservative advocacy organization the Goldwater Institute, and backed by the Koch Brothers’ Americans for Prosperity. The name is cynical but effective. As Trump said on Wednesday, “‘Right to Try.’ It’s such a great name. Some bills, they don’t have a good name. Okay? They really don’t. But this is such a great name, from the first day I heard it. It’s so perfect.”
The name is certainly catchier than the existing name for the program that already does almost exactly the same thing—allowing people with serious diseases to obtain experimental medicines. It is known as expanded access, or more commonly, “compassionate use.”
The difference is that the current program operates through the Food and Drug Administration, which retains the ability to deny some requests for drugs it has not yet approved for use in the general population. The FDA reports that it already authorizes more than 99 percent of requests—so the upcoming change could be minimal. But the potential to exploit this lack of oversight is a risk. In the rare cases when access to unapproved drugs is denied, it can be because of serious concerns about risks on behalf of the pharmaceutical company, or because a physician has overlooked an obviously better alternative.
Under “right to try” this safeguard will be gone, and drugs can be usable after just phase one of clinical trials. As David Gorski, a cancer surgeon and professor at Wayne State University, wrote on Twitter this week, “Claiming phase I testing is enough to show that a drug is ‘safe’ enough for #RightToTry … is utterly insane, as anyone who’s ever had anything to do with clinical trials knows.” …
The goal should not be a right to try, but a right to have safe, effective, affordable drugs that do not drive people to medical bankruptcy. When we remain so far from that, there is no celebration in offering people untested substances and crossing our fingers and then claiming that we have improved the health-care system, much less that we have saved even one life.” (E)

(A) Here come the right-to-try profiteers. The FDA is powerless to stop them, Adam Feuerstein, https://www.statnews.com/2018/06/20/right-to-try-opportunism/?utm_source=STAT+Newsletters&utm_campaign=daf8644f20-DC_Diagnosis&utm_medium=email&utm_term=0_8cab1d7961-daf8644f20-149527969
(B) New study suggests ‘Right to Try’ may undo efforts on patient safety, by Janel Miller, https://www.healio.com/family-medicine/practice-management/news/online/%7B106cd5cf-fa34-4c42-93dc-441ac64d9461%7D/new-study-suggests-right-to-try-may-undo-efforts-on-patient-safety
(C) FDA’s Expanded Access Pre-dates Right to Try by Decades, https://health.howstuffworks.com/medicine/healthcare/fdas-pathway-to-experimental-drugs-pre-dates-right-to-try-by-decades.htm
(D) GOP senator reveals the truth: Right-to-try bill was a scam tailored to harm public health, by Michael Hiltzik, http://www.latimes.com/business/hiltzik/la-fi-hiltzik-right-to-try-20180604-story.html
(E) The Disingenuousness of ‘Right to Try’, by James Hamblin, https://www.theatlantic.com/health/archive/2018/06/right-to-try/561770/

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