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The Cancer Letter – Right to Try edges closer to becoming federal law Libertarians rejoice, but can it solve compassionate use problems?

The Cancer Letter – Right to Try edges closer to becoming federal law Libertarians rejoice, but can it solve compassionate use problems?

Few Washington insiders doubt that a bill that allows terminally ill patients to circumvent FDA as they pursue Hail Mary pass therapies will become the law of the land.


Collectively known as the “Right to Try” law, the legislation has been passed by both chambers of Congress. The bills, which bear the names of four patients—Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina—are the culmination of years of intense lobbying by conservative groups and patient activists.


Supporters of the movement, which started to gain traction in 2013, say that the legislation will speed potentially lifesaving treatments to dying patients by removing bureaucratic FDA red tape.


Meanwhile, critics argue that the bills do not improve access, empower snake oil salesmen, and ultimately erode the patient protections that come with FDA’s expanded access program.


The House of Representatives voted 267-149 to pass its version of the legislation. If enacted, the bill would remove FDA’s mandate to determine whether individual terminally ill patients can receive experimental drugs as a last resort.


The legislation cleared the House March 21, one week after Republicans failed to secure support for the measure through an expedited process that required a two-thirds supermajority. On the second roll call, the bill passed largely along party lines, with the help of 35 Democrats. Two Republicans opposed the bill.


The Senate unanimously passed a similar measure in August 2017, and both chambers are expected to enter conference to iron out differences. The House version requires the sponsor or manufacturer of the investigational drug to immediately report adverse outcomes.


“They both have FDA as their focus, in terms of what they’re trying to achieve to get more access for terminally ill people. But that is just wrong,” said Arthur Caplan, the Drs. William F. and Virginia Connolly Mitty Professor and founding director of the Division of Medical Ethics at the New York University School of Medicine. “Control over access lies in the hands of companies and sponsors.


“The FDA is happy to sign off as long as company is willing to give,” Caplan said to The Cancer Letter. “They are not an obstacle or hindrance to patients. To take away that expertise, leaves patients more at risk. And it is cruel to promise access to the dying and their families that the legislation cannot deliver.”


A conversation with Caplan appears here.


The Congressional bills are a watershed moment for the Goldwater Institute, a libertarian think tank that has, in a matter of four years, introduced right-to-try laws in all 50 states. To date, 38 states have passed some version of the legislation.

“We are the organization that drafted the bill that the 38 state laws are based on. We are the organization that is working with the state-level sponsors of those bills,” Starlee Coleman, senior policy advisor for the Goldwater Institute, said to The Cancer Letter. “We are the ones who ultimately decided that federal companion legislation was going to have to be necessary to answer the use of outcomes question that companies have.


“But I don’t think that the reason that this issue has gotten as far as it has and has captured so many people’s hearts is because of policy. It’s because of people.”


According to Coleman, 97 percent of the Phoenix-based institute’s funding comes from individual contributors, with the remaining 3 percent coming from corporations. In a 2013 report, the Center for Media and Democracy and Arizona Working Families used public records to show that in 2008 to 2010 the institute received almost $1.6 million from the Donors Capital Fund, an entity connected to Charles and David Koch. Other major donors include the Charles G. Koch Charitable Foundation and conservative foundations across the country.


“We have received some funding [from the Kochs] in the past—which has been publicly reported on required IRS documents—but they are not and have never been major funders,” Coleman said. “I mean, this is like one of those conspiracy theories that pop up. Like, we’re conservative, and therefore we must be puppets of the Kochs. OK, thanks. Thanks for that.”


A conversation with Coleman appears here.


President Donald Trump and Vice President Mike Pence have been outspoken advocates for the Goldwater Institute’s efforts. “It is time for the Congress to give these wonderful Americans the ‘right to try,’” Trump said in his State of the Union speech Jan. 30.


“I want to also thank [FDA Commissioner] Scott Gottlieb. Scott is working on different things, but one of them is called ‘right-to-try’,” Trump said a March 21 speech in New Hampshire. “Do you know what ‘right-to-try’ is? These are the people who are terminally ill, and there are very good looking combinations of things or pills, medicines, potential cures. They’re terminal, they’re not going to be living much longer, and we don’t have the right to give them these experimental drugs or these early-stage drugs that show promise, for whatever reason, but they say because they don’t want to harm somebody, if you can believe it.


“I think we’re going to have good luck. The Democrats have been pushing back on it, but I think many of them are also coming around. It’s called ‘right-to-try.’”


FDA approves the vast majority of expanded access applications, but the availability of drugs—i.e. the sponsors’ reticence to provide them—is the principal obstacle here, Gottlieb said in an October 2017 testimony before the House Subcommittee on Health, Committee on Energy and Commerce.


“The most common obstacle to access to the investigational product is the willingness or ability of companies to provide it,” Gottlieb said. “Key protections are included for patients receiving experimental treatments through the expanded access program. These protections include specific labeling requirements, prohibitions on promoting or commercializing investigational drugs by sponsors and investigators, and limits on the costs charged to patients for investigational drugs.


“Adequate policies and processes must be in place to appropriately balance individual patients’ needs for access to investigational therapies while recognizing the importance of maintaining a rigorous clinical trial paradigm for testing investigational products to demonstrate safety and efficacy.”


In a joint statement to The Washington Post, four former FDA commissioners said, “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.”


The March 18 statement 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.


Under one dystopian scenario, mainstream pharma companies would remain reticent to provide access to drugs that are in the early-stages of development, while unorthodox practitioners would step in with questionable interventions.


The right-to-try debate intensified in 2013, after several patients launched high profile petitions and media campaigns about their efforts to obtain investigational drugs.


In late 2013, Andrea Sloan, a 45-year-old Austin attorney with stage IIIC ovarian cancer, became a national flashpoint after her public fight against the CEO of BioMarin Pharmaceutical Inc. drew over 230,000 petitioners, including 82 Texas lawmakers.


Sloan didn’t face obstacles from FDA. The agency had approved her for expanded access, but BioMarin refused to supply the PARP inhibitor she requested (The Cancer Letter, Dec. 3, 2013).


“We do not believe that patients should have to ask the federal government for permission to try a drug that could treat them,” Goldwater’s Coleman said. “There’s a philosophical objection that you have to ask the federal government for permission to save your own life.


“We have an issue with that on its own. We believe that patients along with their doctors should be able to make decisions about their own treatment without the federal government involved.”

Critics: Right to Try does not improve access

The right-to-try bills passed by Congress don’t guarantee access to experimental drugs, said Ellen Sigal, chair and founder of Friends of Cancer Research.


“It is sheer ideology with no basis in fact, and it’s very dangerous for patients,” Sigal said to The Cancer Letter. “It doesn’t compel companies to make their treatments available. The only thing this does is it says if they want to sell them, they can sell them.


“They don’t have to go through FDA, but it doesn’t guarantee or have any ability to say, ‘You must make it available,’ to companies. It basically says, ‘If a patient wants it and you want to give it, you can give it, you can sell it.’ The only requirements are that the sponsor or manufacturer needs to have filed an IND and have completed a phase I trial for the drug.”


FDA is not the problem, Sigal said.


“The big issue is, everyone blames the FDA. They say FDA is the problem, which of course is not true,” Sigal said. “They are of the belief that patients are dying because of delays by the FDA. They want the FDA process to be circumvented because they feel that’s the barrier. They’re saying that patients will get access to these drugs if FDA is out of the system. They’re blaming it on FDA, which of course is baseless— there’s no data to support that erroneous argument.”


Over two decades ago, Sigal’s sister, Gale, had died within two days of undergoing a bone marrow transplant to treat her breast cancer. At the time, the unproven therapy was known to kill as many as 20 percent of patients. Sigal’s testimony before the House Committee on Energy and Commerce is posted here.


For its part, FDA says that it authorizes about 99 percent of expanded access applications, and submissions are approved quickly, often in a matter of days—the result of a comprehensive streamlining process undertaken by the agency in 2015.

“Each year, FDA receives over 1,000 applications for the treatment of patients through expanded access,” agency officials said to The Cancer Letter. “The most recent numbers can be found here. We don’t have a breakdown for oncology specifically.


“In the case of emergencies, FDA will typically provide authorization over the phone in a matter of hours.


“We at FDA share the strong desire to help patients facing terminal illnesses access promising experimental treatments in their fight. We have been working closely with legislators to provide technical assistance as Congress works on this important issue to help ensure patients are also protected.”


The House legislation undermines patient safety considerations that are essential for developing new therapies without causing undue harm, said Chris Hansen, president of the American Cancer Society Cancer Action Network.


“Those concerns include removing the FDA from consulting on drug dosing, scheduling and administration among other important safety measures that the agency is uniquely qualified to understand,” Hansen said in a statement. “Patients will not likely see increased access to treatments under this bill, but would instead face increased risks from weakened oversight and safety measures.


“We urge the Senate to reject this legislation and instead continue to engage with the patient community in constructive discussion on ways to meaningfully improve patients’ ability to access new treatments while preserving critical patient protections.”


FDA is often useful and constructive—a fact that mainstream, responsible companies know very well, said NYU’s Caplan.


“FDA has information access and expertise that companies and local doctors don’t, that can help companies design a trial or safely administer of an experimental drug,” Caplan said. “To take them out of the loop is flat-out stupid.”


On March 12, 78 health care organizations, professional societies, and patient advocacy groups opposed to the right-to-try bills signed a letter addressed to Reps. Paul Ryan (R-WI) and Nancy Pelosi (D-CA).


“The undersigned organizations collectively represent millions of patients with serious and life-threatening diseases,” the groups wrote in the joint statement. “FDA’s expanded access program, though imperfect, facilitates access to investigational therapies for over a thousand patients facing serious and life-threatening conditions each year.


“It is oftentimes the pharmaceutical company that denies access to its investigational therapy outside of its clinical trials for any number of reasons. The alternative pathway in the latest version of the legislation is still less safe for our patients than the current expanded access process.”

Proponents: FDA program is a barrier to access

Over a dozen cancer organizations signed the letter to Ryan and Pelosi—including the American Society of Clinical Oncology, the National Comprehensive Cancer Network, Susan G. Komen, and the Leukemia and Lymphoma Society.

“I’m sorry if I seem exasperated,” Goldwater’s Coleman said to The Cancer Letter. “It’s just, we’ve been talking about this issue for four years pretty extensively and it’s like, your audience are the smartest people in this entire debate, right? And the fact that they’re not connecting the most basic dots is so frustrating.”


FDA’s expanded access program is a barrier because not enough people are being helped, and 1,000 patients a year is a dismally low number, Coleman said.


“It’s not fair that there is a government program that only 1,000 individuals can take advantage of a year, because they happened to be seeing doctors who know how to navigate bureaucracy,” Coleman said. “Another barrier is this requirement that remains in the expanded access program to have an IRB sign off on a patient application after the FDA has done so. And the reason that that’s a barrier is because there’s absolutely no timeframe at all required for an IRB to review and approve an application. There’s no deadline at all.


“When you are terminal, and you’ve exhausted all of your options and you can’t get into a trial, you don’t have time for that. I mean you need an immediate decision. We just feel like all of these hurdles that terminal patients and their physicians are expected to jump through when they are truly in a desperate situation is just immoral, frankly. I mean, it’s wrong.


“We hear all the time from oncologists, including Razelle Kurzrock, an oncologist who used to run the early-stage trials for MD Anderson in Houston and now she is running trials at the University of California San Diego. She has talked to us extensively about the fact that the expanded access program is not workable for the vast majority of patients.”


Kurzrock, chief of the Division of Hematology and Oncology and director of the Center for Personalized Cancer Therapy and Clinical Trials Office at UCSD Moores Cancer Center, said her opinions on right-to-try laws are her own and independent from UCSD.


“In many ways, I think that the right of terminally ill patients to make decisions for themselves under Right to Try, without regulatory oversight, has already been adjudicated via the Right to Die,” Kurzrock said to The Cancer Letter. “Right to Die now exists in some areas of the country—including California, where I reside—and this permits physicians, on patient request and in appropriate circumstances, to prescribe, without FDA oversight, medications that are 100 percent unsafe and are 100 percent ineffective against the patient’s underlying illness.


“In other words, physicians can prescribe medications that will kill the patient. Therefore, if terminal patients can request medications that will kill them, and physicians can prescribe those medications without FDA oversight, it follows that terminally ill patients should be able to request medications that are less than 100 percent unsafe and that are less than 100 percent ineffective against their illness, that is experimental drugs, and physicians should be able to prescribe those medications. I will add the caveat that it is also my personal opinion that it would be best if Right to Try be a federal law so that all patients gain this right.


“Finally, my support for Right to Try does not diminish my belief in the importance of the FDA, which has been doing a really incredible job in the last few years with enabling breakthrough cancer drugs to come to market. It’s just that I feel that FDA should have oversight of the commercial licensing of drugs, but not over the decisions of individual terminally ill patients to try an experimental drug.”