Should the terminally ill have the RIGHT-TO-TRY non-FDA approved therapies?

“Five years ago, the phrase “right to try” wasn’t yet an inkling in the minds of its staunchest advocates. Today, the pithy shorthand for the campaign to get dying patients access to experimental treatments has been slapped on bumper stickers, emblazoned on T-shirts, and uttered by some of the most powerful figures in Washington…
But the story of the five-year fight over “right to try” actually starts at a small lunch meeting of Republican health care experts in Phoenix. It then winds through more than three dozen state legislatures and into the stately meeting rooms of Capitol Hill, where pharmaceutical company lobbyists, Food and Drug Administration officials, and libertarian leaders are still pushing to shape or even upend the ultimate package.
In September 2012, a group of executives from the Cancer Treatment Centers of America reached out to the Goldwater Institute, a small, libertarian think tank named for the former senator and presidential candidate.
Goldwater — which has in past years accepted support from major conservative organizations like the Charles Koch Foundation and Donors Capital Fund — had taken on a wide-ranging libertarian agenda that included activism on campus free speech and school choice. The for-profit hospital chain CTCA also has its own ties to the Koch family.
CTCA and, ultimately, the Goldwater experts believed the FDA’s existing expanded access program — through which the agency approves some 99 percent of requests from dying patients who can’t get into clinical trials but want to try experimental treatments — was too cumbersome.
It was time to do something about it, and a consultant with CTCA, Chuck Warren, coined the term “right to try,” recalled Starlee Coleman, a Goldwater senior adviser.
A little over a year after their meeting with CTCA, Goldwater’s model legislation was signed into law in Colorado, a largely Democratic state with a Democratic governor. Thirty-nine other state legislatures eventually followed, many without a single dissenting vote. (A)

“House lawmakers have unveiled their version of a bill that lets terminally ill patients try experimental treatments.
The bill was released early Saturday morning and is the House companion of a Senate version that passed last year. The White House has also made pushing legislation for right-to-try a major priority, with President Trump even mentioning it during his State of the Union address in January….
The bill would let patients gain access to a drug that has gone through the first of three phases of clinical trials. The patient must be terminally ill and have no other options to qualify to get the drug.
Manufacturers wouldn’t be required to supply the drug to terminally ill patients, but the bill hopes to entice drugmakers to participate in the program.
Drugmakers are sometimes hesitant to provide a drug outside of a clinical trial to a terminally ill patient because if the patient dies, then it could affect the approval of the product by the Food and Drug Administration. Experts have said that patients who would use right-to-try would likely be sicker than patients in a clinical trial.
The bill seeks to provide certainty to manufacturers on how the FDA will use patient outcomes when evaluating whether to approve the product. (B)

“In a major blow to the effort to pass a federal “right-to-try” law, House Republicans failed to muster the votes to pass a key compromise measure Tuesday.
The legislation was rejected by a vote of 259-140. The bill needed two-thirds majority to pass under suspension of rules.
The vote was an embarrassing defeat not only for House Republicans but for President Trump, who had called on Congress to quickly pass right-to-try legislation, and Vice President Mike Pence, a longtime supporter of the effort. It was also a rare misstep from Speaker Paul Ryan, who like most congressional leaders almost never schedules votes on legislation without some certainty that the given measure has the support to pass.
The controversial “right-to-try” bill would offer a pathway for patients with a terminal disease to get around Food and Drug Administration rules to request an experimental treatment from a drug maker before the agency has approved it. Supporters of the legislation, including the libertarian Goldwater Institute that first conceived of the pathway, say it would offer a necessary respite from what they say are overly cumbersome FDA rules.
Critics, however, including Minority Leader Nancy Pelosi of California, pointed out that the FDA already has a program in place to offer access to terminal patients, and the agency grants some 99 percent of all the requests it receives, often within a short time frame. They also argued that without more FDA oversight of the use of experimental therapies, desperate patients could be vulnerable to bad actors or unsafe treatments.” (C)

“Supporters of this legislation talk as if effective treatments are being withheld from patients,” said Dr. Robert M. Califf, who was the commissioner of the Food and Drug Administration under President Barack Obama. “The vast majority of experimental therapies are toxic or ineffective. The only way we find out is through controlled studies where we measure the effects.”
In a letter to House leaders, more than 75 patient advocacy groups, including the lobbying arm of the American Cancer Society, opposed the bill.
The American Medical Association said it “does not believe that the bill will substantially improve patient access to investigational therapies.” And the American Society of Clinical Oncology, representing cancer doctors, said the bill “could do more harm than good for patients with life-threatening illnesses” because it would remove the Food and Drug Administration from the evaluation of the risks and potential benefits of some treatments.
Drug companies can already provide experimental medicines to patients outside clinical trials under a program known as expanded access or compassionate use. The F.D.A. says it approves about 99 percent of compassionate use requests. In emergencies or other urgent cases, the agency says, it approves requests for the use of experimental medicines within hours or days of being contacted by a doctor.
Representative Jan Schakowsky, Democrat of Illinois, said the bill would have created “a dangerous back door around the F.D.A. approval process.”..
The drug industry was uncharacteristically quiet. The main lobbies for the industry — the Pharmaceutical Research and Manufacturers of America and the Biotechnology Innovation Organization — said they had not taken a formal position on the legislation.
The drive for right-to-try legislation was spearheaded by the Goldwater Institute, a public policy organization that advocates free markets and limited government.
Starlee Coleman, a policy adviser at the institute, said 38 states had adopted right-to-try laws, often with broad bipartisan support.” (D)

“The House will have to try again, before the bill can go back to the Senate (where it previously passed in August by unanimous consent) for further consideration. Before the new draft is revealed, here are several things about the Right To Try movement that need to be cleared up.
The “Right To Try” Is Not A New “Right”
A qualifying individual may petition to access an experimental therapy, but that does not mean they will receive it. Drug companies still have the authority to refuse any request. Thus, the right to try is really just federal-level permission to ask for an experimental therapy. The bill does, however, attempt to incentivize manufacturers by acknowledging that Right To Try patients will be sicker, and thus any negative outcomes from allowing terminally ill patients to try therapies will not be included in the FDA’s decision-making for future approvals.
Treatments Must Still Have Some Rigor. According to the new legislation, even an eligible investigational treatment must: (1) have successfully completed a phase 1 (initial, small scale) clinical trial; (2) remain under investigation in a clinical trial approved by the FDA; and (3) not be approved, licensed, or cleared for sale under the Federal Food, Drug, or Cosmetic Act or the Public Health Service Act. This is good news for those concerned about safety, but some argue the barriers are not enough.
Clinical Trial Efforts Have Failed Patients. The truth is that many individuals pursue the right to try because they have been unsuccessful in clinical trials. Further, traditional trials add not only years to the review process for therapies, but inevitably increase drug costs. Ongoing arguments suggest that if the clinical trial process in the U.S. were improved, the need for the Right To Try Act wouldn’t exist. However, others contend that the FDA trial process is necessarily rigorous and that any patient rights to skip clinical trials will undermine future trial enrollment. (E)

“Every day, every hour, counts for them. But the process by which the federal Food and Drug Administration approves medicines and treatments takes an average of 14 years, and during that time, patients are, with a tiny number of exceptions, legally barred from using medicines that could save their lives — medicines the FDA has deemed safe and that it is giving people in clinical trials…
“The Right to Try movement was born to change this cruel reality. Alongside patients, doctors and policymakers nationwide, the Goldwater Institute crafted a new approach, a state-law reform that allows ill Americans to seek investigational treatments when they’re out of other options.
Right to Try’s success has been spectacular. In just five years, Right to Try bills have been introduced in every state, and 38 have adopted it, including Arizona, where voters passed it overwhemingly in 2014. The Wisconsin Legislature just sent a bill to the governor for his signature…
The wealthy and privileged have always been able to get the medicines they need, even without FDA approval. They can travel to other countries where they can get the medicines the FDA keeps locked up here.
A fortunate few get FDA permission to enter clinical trials or receive the so-called “Expanded Access” exception. It allows some people, on a case-by-case basis and through a cumbersome process, to request permission from the FDA to access medications outside of a clinical trial.
But these options provide false hope for most people, because only a fraction of those who need these exemptions are even allowed to request them, and most people can’t afford to travel the world to get treatment.” (F)

“Four former commissioners of the Food and Drug Administration are expressing opposition to congressional “right to try” legislation, just as Republican House leaders prepare to bring a bill up for another vote a week after it failed to pass….
“There is no evidence that either bill would meaningfully improve access for patients, but both would remove the FDA from the process and create a dangerous precedent that would erode protections for vulnerable patients,” they say…
The statement by the former FDA heads was signed by Robert Califf and Margaret Hamburg, who were commissioners during the Obama administration, and Mark McClellan and Andrew von Eschenbach, who served under George W. Bush. Some have previously expressed reservations individually about the bills. The joint statement is a show of bipartisan unity to try to slow the legislation’s progress on Capitol Hill.” (G)

“Thirty eight States have enacted right-to-try (RTT) laws, the intent is to increase the availability of experimental medicines to individuals battling life-threatening conditions A federal version has support at the highest levels of the Trump administration including the president himself. Yet the Goldwater Institute, which created and has strongly championed these laws, cannot provide clear examples of patients who have gained access to potentially life-saving treatments through state RTT laws that they otherwise wouldn’t have received under the FDA’s current Expanded Access Program (EAP). Furthermore, federal RTT law will not increase access either…
All this being said, one cannot ignore the desire to increase the access of critically and terminally ill individuals to experimental medicines. The question then becomes how to accomplish this in a way that meets the needs and requirements of regulators, companies and the current and future individuals in need?
One way to meld the intent of RTT laws with the existing EA process would be to create a more explicit regulatory pathway. This allows EA safety and efficacy data to be incorporated into the label of a new medicine once it is formally approved for its primary indication via “traditional,” highly controlled clinical trials.
In this way, companies could more fully balance the risks and benefits of an EAP to the overall development process of the experimental medicine. Companies would be incented to undertake these broader EA programs because they could determine how the EA program would potentially lead to the ability to treat a larger future patient population.
This would require legislative and regulatory changes to existing guidelines for intermediate- or large-scale EAPs, but would still be within the framework and intent of the 21st Century Cures Act.
Rather than circumventing the FDA oversight process, companies would have to reach agreement with the FDA as to the parameters under which “real-world evidence” (evidence regarding the potential benefits and risks of a product) collected from patients enrolled in expanded access trials could be used to support additional label claims for a new medicine. These programs would be overseen as if they were formal clinical trials designed to provide full input into the drug development process.” (H)

“Right-to-try legislation would create a pathway to remove FDA oversight of experimental, unproven therapies. These are the treatments that most need rigorous regulatory oversight tempered by willingness to allow pre-approval access for patients who need them most: those who have run out of treatment options. Excluding the FDA from the development and delivery of these therapies will make it harder to gauge their safety and effectiveness.
Advocates like us have spent decades fighting to accelerate the pace of scientific discovery and the development of new treatments, with patient safety and effectiveness as the North Star. We have each seen firsthand the perils of disease and the anguish of loved ones who have run out of options to fight deadly diseases like cancer or AIDS. But we cannot allow this pain, and our unwavering commitment to end suffering, blind our judgment about legislation that would create a climate where the most vulnerable patients are taken advantage of, their safety endangered, and drug development driven off track.
During the peak of the AIDS crisis, with 50,000 Americans dying from the disease each year, brave and determined activists pushed the FDA to increase regulatory flexibility — creating a variety of mechanisms that brought patients faster access to lifesaving therapies, therapies that are now saving millions of lives around the world and preventing new HIV infections. Cancer advocates have also long worked side-by-side with the FDA and Congress and continue to successfully enact innovative policy changes and expedited approval mechanisms such as Accelerated Approval and the Breakthrough Therapy designation. These changes have made a significant and lasting difference for patients.
If the right-to-try legislation can’t speed expanded access by circumventing FDA review, then what would it do? One result is certain: It would create uncertainty. In the 38 states that have passed similar bills, there is no evidence so far showing if these policies have increased patient access to experimental therapies. The absence of data here is both alarming and telling.” (I)

“The right-to-try bill, which suffered a surprise 259-140 defeat in the House of Representatives last week, is scheduled for another vote this week. The House Committee on Rules was scheduled to meet late in the day Monday to change the procedure so that it could be passed with just a simple majority, rather than a two-thirds majority, and this time approval seems likely.” (J)

“The House, spurred on by President Trump, passed a bill on Wednesday that would give patients with terminal illnesses a right to try unproven experimental treatments.
The measure, which was approved by a vote of 267 to 149, appears to have a good chance of becoming law. The Senate approved a similar proposal last year…
Mr. Trump endorsed the right to try in his State of the Union address in January and again on Monday, on a trip to Manchester, N.H., where he described potential beneficiaries of the bill.
“A patient is terminal,” Mr. Trump said. “There’s good progress made with a certain drug. We’re going to make it possible for that patient to get that drug. And maybe it’s going to work. It’s hope.
Thirty-five House Democrats voted for the bill on Wednesday night, along with 232 Republicans…
The House and Senate bills would establish a new pathway providing access to unapproved medicines for certain patients who had exhausted other treatment options. To qualify under the House bill, a patient would have to have some kind of terminal illness: a condition that is likely to cause death “within a matter of months” or “irreversible morbidity that is likely to lead to severely premature death.”
Nothing in the bill would require pharmaceutical companies to provide experimental drugs to patients who requested them. Drug manufacturers sometimes turn down requests because they have only a limited supply or they are concerned about legal and medical risks.
To address such concerns, the legislation would shield drugmakers, doctors and hospitals from some of the legal risks of providing unapproved drugs to patients. Doctors and hospitals would generally be protected unless they engaged in gross negligence or willful, reckless or criminal misconduct.” (K)

“Republican supporters of the bill said the “right-to-try” law would allow patients to have more control over their care. “I’m a physician and scientist with over 40 years’ experience treating patients, some of whom had the dreaded diagnosis of cancer,” said Rep. Phil Roe, MD, R-Tenn. “Six months ago, I was operated on for cancer, and I — to this day — am a cancer survivor. If needed, I would like to have the right to try.”” (L)

(A) How the ‘right-to-try’ movement muscled its way into Washington, by ERIN MERSHON,
(B) House panel unveils right-to-try bill, by Robert King,
(C) ‘Right-to-try’ bill rejected by House, in major blow to GOP efforts, by ERIN MERSHON,
(D) House Rejects Bill to Give Patients a ‘Right to Try’ Experimental Drugs by By ROBERT PEAR,
(E) 7 Things To Know About Experimental Drugs And The ‘Right To Try’ Act, by Nicole Fisher,
(F) Your Turn: How Right to Try laws could one day help save your life, Christina Sandefur,
(G) Former FDA commissioners say right-to-try bills could endanger ‘vulnerable patients’, by Laurie McGinley,
(H) Here’s how to structure successful right-to-try laws, by BY KENNETH I. MOCH, ANDREW MCFADYEN AND ARTHUR CAPLAN,
(I) ‘Right-to-try’ law threatens patient safety and rational drug development, By MARK HARRINGTON and ELLEN V. SIGAL,
(J) Republicans Get Ready to Vote on “Right-to-Try” Bill Again, by Allison Inserro,
(K) House Passes Bill That Would Give Patients Access to Experimental Drugs, by ROBERT PEAR,
(L) House passes ‘right-to-try’ bill on 2nd vote: 10 things to know, by Megan Knowles,

Revised 3/22/18

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