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“Should the terminally ill have the RIGHT-TO-TRY non-FDA approved therapies?”
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“President Donald Trump signed the “Right to Try Act” Wednesday, a measure aimed at helping terminally ill patients access drug treatments that are yet to be fully approved by the Food and Drug Administration.
Trump, at a White House ceremony surrounded by patients and families who will be affected by the legislation, said his administration “worked hard on this” but said repeatedly he didn’t understand why it hadn’t been done before.
The bill will give terminally ill patients the right to seek drug treatments that remain in clinical trials and “have passed Phase 1 of the Food and Drug Administration’s approval process” but have not been fully approved by the FDA. Some opponents of the bill argue that the legislation won’t change much but could have a detrimental effect on how the FDA safeguards public health.
“With the passage of this bill, Americans will be able to seek cures,” Trump said adding they will finally be given “the right to try.” (A)
“Opponents of the law say Trump’s promises to help thousands or even hundreds of thousands are grossly unrealistic. It isn’t clear that any patients have successfully utilized the dozens of state laws that attempt to give patients a similar pathway for expanded treatments…
Supporters say the new law gives dying patients an important reprieve from overly cumbersome FDA rules, and from what they call a slow-moving bureaucracy that can delay access to time-sensitive treatments.
Opponents have cautioned that the legislation will undermine FDA’s authority and open vulnerable, desperate patients up to “snake oil salesmen.” They point out, too, that the FDA already has a process in place for approving such requests and grants some 99 percent of them, often within a short time frame…
In a statement, Gottlieb said the agency stands ready to implement the new law “in a way that seeks to protect [patients’] autonomy, their safety, and the safety of others following in their paths.” (B)
“Despite good intentions – and the legislation’s name – right to try legislation grants no rights. It would merely grant permission for a patient to try to get experimental medication from a pharmaceutical company.
Patients would be allowed to try experimental drugs, but nothing in the legislation would make it mandatory for pharmaceutical companies to provide these medications.
The reasons for a company to withhold a drug are many. Giving access to preapproval drugs can be costly, particularly given the limited supply, and almost no medical insurance will cover experimental treatments. Access to the drugs will likely only be feasible for wealthy Americans who can afford to pay for the treatment, as well as the consequences of any negative side effects out of pocket.
Many drug companies also worry that making the drug available without approval opens them up to lawsuits if the drugs prove ineffective or have unforeseen side effects. The current federal right to try proposal does provide some protections for companies in this regard, but until those protections have been tested in court, it is likely companies will remain cautious.
Providing the drug to patients outside of clinical trials can also be disruptive to the approval process because it can lead to data on negative clinical outcomes outside the highly controlled trial setting…” (C)
“Opponents also argue it gives “false hope” to patients, since drugmakers aren’t required to give unapproved medicines to patients who ask for them.
Supporters say, however, it will provide new treatment opportunities for terminally ill patients who have exhausted existing options.
“While a long time coming, today is a monumental win for patients desperately seeking the ‘right to try’ investigational treatments and therapies,” said Energy and Commerce Committee Chariman Greg Walden (R-Ore.) and health subcommittee chairman Michael Burgess (R-Texas).
“With ‘right to try’ being the law of the land, we are confident that the Trump Administration, and FDA Commissioner [Scott] Gottlieb, will take both congressional intent and the safety of patients into consideration when implementing this important law.” (D)
“It allows certain patients to ask drugmakers for medicines that have passed Phase 1 of the FDA approval process but haven’t been approved yet and are still undergoing testing. Patients must have exhausted other options and be unable to participate in a clinical trial. Drugmakers aren’t obligated to give patients the requested experimental medicines.
Critics say the legislation undermines the FDA’s authority to regulate drugs and could leave patients vulnerable to medicines that might not work or may even be harmful. The agency already runs an “expanded access” program where seriously ill patients can apply to gain access to experimental treatments.
Commissioner Scott Gottlieb has said the agency grants 99 percent of these requests. In a statement Wednesday, Gottlieb said the FDA is ready to implement the “right-to-try” legislation.
“The FDA is dedicated to achieving the goals that Congress set forth in this legislation, so that patients facing terminal conditions have an additional avenue to access promising investigational medicines,” he said.” (E)
““The Administration believes that treatment decisions for those facing terminal illnesses are best made by the patients with the support and guidance of their treating physicians,” the White House said in its statement of support Monday.
Opponents, meanwhile, say the legislation will allow “snake oil salesmen” to take advantage of desperate patients and could ultimately weaken or undermine the FDA’s authority to regulate drugs. They point out, too, that the FDA already has a system in place for granting terminally ill patients access to experimental therapies, often within a tight timeframe. Though opponents said the more refined version of the legislation was an improvement, they said it still failed to address many of their concerns.
“The legislation would roll back essential patient safeguards and could result in patients being harmed by unproven, and potentially unsafe, therapies,” a coalition of patient groups, including the American Cancer Society Cancer Action Network, Friends of Cancer Research, and the National Organization for Rare Disorders, wrote in a letter to lawmakers.. “Furthermore, the legislation would significantly restrict FDA’s ability to stop access to an experimental therapy and would remove expert consulting requirements on dosing and other important safety measures currently provided by FDA.”” (F)
“The president said the issue was “very personal” for him.
“As I proudly sign this bill, thousands of terminally ill Americans will have the help, the hope and the fighting chance — and I think it’s going to be better than chance — that they will be cured, that they will be helped, that they will be able to be with their families for a long time, or maybe just for a longer time,” Trump said. “But we’re able to give them the absolute best we have at this current moment, at this current second. We’re going to help a lot of people. It’s an honor to be signing this.” “ (G)
“A program known as compassionate use, or expanded access, has been in place since the 1970s. It allows patients with a serious disease or condition to obtain experimental medicines; the Food and Drug Administration says it authorizes 99 percent of the requests for expanded access that it receives.
The new national law — like similar laws in more than three dozen states — allows patients and doctors to ask drug companies directly for access to the experimental drugs, rather than wait for approval by the agency.
Yet these laws “do not ensure that manufacturers will provide the drug or that insurance companies will cover the cost,” according to a policy report from Rice University. Obtaining the medicines from manufacturers can be more cumbersome than going through the Food and Drug Administration’s existing program, the report found.
Colorado enacted the first right-to-try law in 2014. Since then, “there have been no documented cases of anyone receiving access, because of a right-to-try law, to an experimental product that would not have been available via the F.D.A.’s expanded access program,” a 2017 study by researchers from New York University concluded.
Alison Bateman-House, a professor of medical ethics and an author of the study, called it “extremely unlikely” that the national right-to-try law would directly save hundreds of thousands of lives, given that drug companies are using — and accustomed to — the existing process.” (H)
“Most experimental drugs are nowhere close to being effective, or carry side effects outweighing their benefits. But if they were, who could afford to try them?
“The law gives the right to pay, not the right to try,” says Arthur Caplan, head of medical ethics at New York University’s School of Medicine, who also studies compassionate access to medication. The law, he explains, doesn’t come with a budget, leaving patients paying out-of-pocket for unproven treatments. Insurance companies are unlikely to help out; they’re not even required to cover all the drugs that are approved by the FDA.
Medical costs are already the leading cause of bankruptcy in the US. Under “right to try,” a cancer patient could end up paying additional hundreds of thousands of dollars for experimental treatment, on top of their existing costs. False hope could tempt sick Americans to poor financial decisions, argues Caplan. “You are asking terminally ill people to spend their family’s savings on drugs that don’t work,” he says.” (I)
“Today, 40 states have passed similar laws. And on Wednesday, President Trump will sign a national Right to Try Act into law. In the states, Right to Try has not been a partisan issue. In most instances, it passed without a single dissenting vote…
But, sadly, this spirit of bipartisanship did not translate to Washington. Only 22 House Democrats voted for the final bill that Trump will sign. In August, a version introduced by Sen. Ron Johnson, R-Wis., passed the Senate by unanimous consent, but stalled in the House, which eventually passed a more limited bill from Rep. Greg Walden, R-Ore. Last week, Senate Republicans tried to accommodate Democratic objections by bringing up the Walden bill for a vote, but Senate Minority Leader Charles Schumer blocked it from coming to the floor. So the House passed the Johnson version, with Democrats nearly united in opposition.
In other words, Democrats in Washington managed to take an issue that unified thousands of legislators from both parties in 40 states, and turned it into a divisive, party-line vote. Thanks to Trump, Americans facing terminal diagnoses will now have a new chance at life. How tragic — and pathetic — that Democrats refused to join him in making that happen.” (J)
“From now on, these patients — in fact, practically anyone who has been diagnosed with any life-threatening disease or condition, whether or not he or she is near death — will be able to choose to trust doctors and drug companies alone to look out for their well-being…
Under the new law, without the FDA’s expert guidance, companies will have even more reason to turn down requests for experimental medicines. Those inclined to provide the drugs, if they’re wise, will continue to do so only through the FDA’s existing channel — as Janssen Pharmaceuticals has decided to do. The company says that the FDA may be “uniquely aware” of available safety data.
FDA Commissioner Scott Gottlieb has pledged to carry out the new law in a way that promotes access and protects patients. But he has no authority to do anything except issue guidance and receive annual summaries from companies providing investigational drugs.
A word of advice to doctors and patients who choose to sidestep the FDA: Proceed with care, and don’t count on a miracle.” (K)
(A) Trump signs ‘Right to Try Act’ aimed at helping terminally ill patients seek drug treatments, by Allie Malloy, https://www.cnn.com/2018/05/30/politics/right-to-try-donald-trump/index.html
(B) Trump signs right-to-try legislation, making controversial measure law of the land, by ERIN MERSHON, https://www.statnews.com/2018/05/30/trump-signs-right-to-try/
(C) What is “right to try” and will it help terminally ill patients? by Morten Wendelbo, Timothy Callaghan, https://www.cbsnews.com/news/right-to-try-bill-trump-signing-will-it-help-terminally-ill-patients-today-2018-05-30/
(D) Trump signs ‘right to try’ drug bill, by Jessie Hellmann, http://thehill.com/policy/healthcare/389908-trump-signs-right-to-try-bill-for-terminally-ill-patients
(E) President Trump signs ‘right-to-try’ bill for experimental drugs, by Angelica LaVito,| https://www.cnbc.com/2018/05/30/trump-signs-right-to-try-legislation-on-experimental-medicines.html
(F) Congress passes ‘right-to-try’ measure, sending hard-fought bill to Trump’s desk, https://www.statnews.com/2018/05/22/house-vote-right-to-try/?utm_source=STAT+Newsletters&utm_campaign=cdfd050a18-MR_COPY_01&utm_medium=email&utm_term=0_8cab1d7961-cdfd050a18-149527969
(G) Trump signs ‘Right to Try,’ says it will save ‘tremendous number of lives’, by Brooke Singman, http://www.foxnews.com/politics/2018/05/30/trump-signs-right-to-try-says-it-will-save-tremendous-number-lives.html
(H) Trump Oversells New ‘Right to Try’ Law, by Linda Qiu, https://www.nytimes.com/2018/05/30/us/politics/fact-check-trump-right-to-try-law-.html
(I) Who pays for the “right to try” experimental medicine?, by Annalisa Merelli, https://qz.com/1292947/under-the-right-to-try-act-who-pays-probably-not-insurance/
(J) Thiessen: Terminally ill thank GOP for ‘Right to Try’, by MARC A. THIESSEN, https://www.mercurynews.com/2018/05/30/thiessen-terminally-ill-can-thank-republicans-for-right-to-try-investigational-drugs/
(K) A ‘Right to Try’ That Americans Didn’t Need, https://www.bloomberg.com/view/articles/2018-05-31/a-right-to-try-that-americans-didn-t-need