Trump's controversial new health care idea (The Agenda)

Trump’s controversial new health care idea

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It sounds like a humane and overdue change to federal drug regulations: Allow desperately sick patients to ask for experimental new medicines if they’re willing to take the risk. And give drugmakers a break on liability if they provide them to patients.

The idea, known as "right-to-try," got a huge boost Tuesday night when President Donald Trump mentioned it in his State of the Union address. “People who are terminally ill should not have to go from country to country to seek a cure—I want to give them a chance right here at home. It’s time for the Congress to give these wonderful, incredible Americans the ‘right-to-try.’” he said, surprising the health care industry and publicly citing for the first time an idea that Vice President Mike Pence had been pushing for a year.

A right-to-try bill is now making its way through Congress; it passed the Senate in August and could get a House vote in the next few weeks. Few political battles are easier to win than those waged on behalf of the dying, and state-level right-to-try laws have already cut a fast path through American legislatures, passing in 38 states in the past three years.

To supporters, the idea is a welcome last chance for suffering patients, a shot at trying risky medical innovations that just might save their lives. Behind the law, however, lies something beyond the patient-in-need stories that their advocates like to trot out. At both the state and federal level, right-to-try laws are part of a sustained campaign to roll back the power of the Food and Drug Administration more broadly, loosening more than half-a-decade’s worth of drug regulation, largely driven by one Arizona free-market think tank.

The authors of the legislation Trump touted in his speech aren’t patient advocates, or doctors, but the Phoenix-based Goldwater Institute. Dedicated to challenging what it sees as government overreach, Goldwater has spent years trying to roll back Obamacare and push school voucher programs. To the institute, right-to-try is a part of the same larger fight, an overdue shift of decision-making from regulators to patients—not just morally right, but inevitable. "A mom with a dying kid is not going to let her child die when she knows someone in her community is in a trial and getting the drug and is alive," said Starlee Coleman, the institute’s chief policy adviser.

The entire right-to-try movement comes wreathed in questions—including whether it makes any difference for patients at all. The FDA has already loosened its rules to allow dying patients to try experimental drugs, and two years’ worth of right-to-try laws passed at the state level so far have been almost totally ineffective at getting new drugs to patients. Nationally, in the 38 states to have passed right-to-try, Goldwater can point to only a single doctor who has treated patients with a drug because of the law.

Despite those failures, both advocates and opponents think a national law could change the landscape. Advocates believe it will force drugmakers’ hands, creating the publicity and momentum necessary to get drugs to more dying patients. And a law at the national level could eliminate the risks that drugmakers now face for violating interstate commerce laws if they provide drugs for federally unapproved uses. Opponents say it would establish a dangerous precedent—a national beachhead for a new set of ideas that could, someday, weaken a federal drug-safety system responsible for decades of American medical and business success.

Daniel Carpenter, a Harvard professor who runs a research project on drug regulation and has written a book on the FDA’s history, said he sees the right-to-try advocates as trying to establish a potentially dangerous “legal metaphor”—that access to drugs should be just as much of a right as free speech or reproductive freedom.

Goldwater isn’t just waiting for the right-to-try law to work its way through Congress. With right-to-try already the law in a majority of states, it has identified a new target: It wants to loosen up the FDA’s marketing rules so drug companies can promote their products even for unapproved uses. The first such law at the state level was signed in Arizona in March and Goldwater has since helped get bills introduced in Colorado and Missouri. This month, Goldwater rolled out a national strategy to get these bills passed in dozens of state legislatures and either force a court challenge to the FDA’s current policy on First Amendment grounds or using the state effort to agitate for a congressional solution.

With allies in the White House, and a strong grass-roots movement among patients, Goldwater believes its first federal victory against FDA is all but inevitable. The internet and social media have given patients and families more access to information about promising drugs. And they want them.

“Ultimately the way we feel about all of this is it’s a matter of time. The genie is sort of out of the bottle,” said the Goldwater Institute’s Coleman. “Patients have too much information now. They know when drugs in trials are working because they are connecting now more than they ever have been before. Maybe people who don’t want this will be able to stem the tide for a little while, but there will be something that comes later."

But it may not be quite that simple. Though most industries chafe at regulation, pharma’s relationship to the FDA is more co-dependent: American drug firms are global powerhouses in part because of the agency’s strict gatekeeping, which allows them to charge a high premium for successful drugs. Disrupting that system would threaten drug companies’ business as well as, potentially, the health of patients—putting Goldwater in the unenviable position of fighting both the industry and liberal patient-safety advocates.

POLITICALLY SPEAKING, RIGHT-TO-TRY is almost a no-brainer. Saying no to dying patients is “sort of like kicking puppies,” said Richard Klein, who recently retired as FDA’s Patient Liaison Program Director after a 41-year career at the agency.

The movement was born in 2013, when a chain of for-profit hospitals called Cancer Treatment Centers of America requested a meeting with Goldwater. CTC’s business is built on selling patients high-priced infusion drugs, including personalized genomic therapies, along with complementary services like homeopathy and herbal and botanical products that lack evidence to support their use. The company raised concerns that the current FDA approval process is not set up to handle the future of medicine, where individuals may require custom-tailored drugs that haven’t gone through FDA approval.

The meeting prompted Goldwater to look at the data from the FDA’s existing program to provide experimental drugs to dying patients. Called "compassionate use," the program was first laid out in regulations in 1987 and allows patients to get access to medicine still in the testing phase, if their doctor gets FDA sign-off. Doctors must certify that the patient has no comparable alternative, and FDA must determine there is adequate evidence to support the use of the drug, and that the patient’s use won’t interfere with the clinical trials being conducted to get the product broader marketing approval.
Although 99 percent of compassionate-use requests are approved by FDA, Goldwater’s Coleman said they “were shocked” to find out only 1,000 patients use that program every year, despite more than half a million Americans dying annually from cancer alone. They concluded that FDA’s process was “inadequate,” Coleman said.

For Coleman, the compassionate-use process hits close to home. Her brother died of melanoma in 2009, after unsuccessful attempts to obtain access to drugs that were currently in the testing phase. “It’s not OK to keep promising treatments from patients who are willing to take that risk,” she said. But to close observers of drug development, Goldwater’s plan would carry real risks for
precisely the most vulnerable patients, those most desperate for something to help.

The modern scaffolding of drug safety dates back to 1962, when Congress for the first time added language to the Food, Drug and Cosmetic Act to require that manufacturers demonstrate not only the safety of their medicines but substantial evidence of effectiveness. That law was passed after the thalidomide crisis—when thousands of babies were born with birth defects because their mothers took that drug during pregnancy. Once the new rules went into effect, FDA went back and reviewed the 16,500 drugs already on the market, and found that 70 percent of the claimed uses were not supported by substantial evidence. Only 434 were found to be effective for all their claimed uses. A 2017, FDA paper found that about only one third of the drugs requested for expanded access go on to be approved by FDA.

Today, drugs go through multiple rounds of human testing, first mainly for safety, and then larger clinical trials to prove effectiveness. The reason experimental drugs aren’t legally on the market is that they haven’t completed that testing process, and haven’t yet proved their benefits are strong enough to outweigh any side effects. Until the results are submitted and the FDA approves, an experimental drug is available only to patients who are officially being tested as part of clinical trials, and a handful registered under compassionate-use protocols. Though the process can be onerous, it also protects patients from companies eager to push exciting-sounding but unproven therapies to desperate families.

Goldwater’s "right-to-try" idea would short-circuit that process, allowing patients with a life-threatening condition to ask for drugs that have gone through at least one phase of clinical trials, proving they were safe to use in humans. It wouldn’t require the drugmakers to say yes, but it would insulate firms and doctors that did from lawsuits—a significant issue, considering the serious health risks of unapproved medicines. There are still some limitations—patients must have exhausted approved treatment options and be unable to participate in a clinical trial of the requested drug, and the drug requested must be in active FDA-overseen clinical trials—but it amounts to a significant loosening of the current rules.

This short-circuiting is exactly what worries patient-safety advocates. Drugs for serious diseases such as cancer can often have profound side effects, life-threatening in themselves, which may outweigh the modest benefits the drugs offer. The Goldwater Institute largely dismisses safety concerns over right-to-try by pointing out that the newly available drugs will already be in some phase of clinical testing, meaning that they’ve been given the green light for human study by FDA. But detractors say a new law would totally change the safety equation: Under right-to-try, for-profit businesses could encourage doctors to use medicines being studied in legitimate trials for one purpose, for a host of other diseases not given the same government clearance.

And because use of a drug through right-to-try doesn’t come with the same rigorous oversight and reporting requirements that are mandated in clinical trials, it could take as long as a year for FDA to be aware of serious harms or even deaths. Meanwhile, right-to-try’s indemnification of manufacturers and doctors could effectively insulate them from bad behavior, while offering patients limited recourse in worst-case scenarios.

“People could die and there is no accountability. No one would know,” an FDA official said.

For their part, drug companies generally don’t see this as part of the regulatory system they particularly want to loosen up. New drugs can be very expensive to make. And providing them to sick patients runs the risk of disrupting the delicate, high-stakes trial process. The appearance of an unexpected side effect in a patient outside a trial could damage a drug’s safety record, hurting the treatment’s ultimate chance of approval and even causing FDA to halt the clinical trial, companies argue. The drug industry also needs patients to be incentivized to participate in controlled clinical trials, which remain the only way to accurately determine if a drug works and for whom. If they can get any experimental medicine through a doctor, they have less incentive to enroll in the tests that prove whether the treatments really work.

The drug industry’s powerful lobbying group, PhRMA, has kept a low profile on the issue, worried it might look like it’s throwing obstacles in the way of dying Americans, but has quietly weighed in for the status quo, saying it wants FDA to remain a part of the compassionate-use process. Other companies have gotten individually involved: Merck, one of the nation’s largest drugmakers, came out against right-to-try before the Senate bill was approved, and health care giant Johnson & Johnson has said it will evaluate patients’ compassionate-use requests only if the patient goes through FDA’s program. Some nonprofit disease groups with industry funding have been more publicly pushing back against right-to-try; including by offering lawmakers alternative ideas they say will do a better job of getting more patients experimental drugs than Goldwater’s bill.

Some in the health care industry feel like they’ve seen this all before, and don’t expect a new law to change much for patients. The drug industry and FDA went through an even higher-profile public fight on this issue in the late 1980s and early ’90s, when AIDS activists targeted the government with protests for its sluggish approval process, convinced that FDA was dragging its heels on access to new treatments that could save their lives.

Pushed by activists, in 1992 Congress created an accelerated approval process that lets companies design streamlined clinical studies and FDA approve drugs for life-threatening diseases like AIDS before the treatments have demonstrated they will extend patients’ lives. Congress also developed a program in which drug companies pay FDA significant application fees, helping the agency fund itself in return for faster application reviews. After these reforms, and others that followed, FDA is now the fastest agency in the world in reviewing new drugs for market.

“If you really care about patients who have serious and life-threating disease, and you want to get drugs that work, do the clinical trial and do the clinical trial expeditiously and then the patient will know whether to take that drug or some other drugs,” said former FDA Commissioner David Kessler, who oversaw FDA during the pivotal reforms of the early 90s. “Otherwise we are living in the world of snake oil.”

THE GOLDWATER INSTITUTE said it doesn’t want to tear down FDA and return to the old free-for-all world of unproven medicines, but it does want to shine a light on the agency’s flaws, and how its rigorous review process slows down patient access and adds to the cost of drugs. Goldwater argues that if drugs had a lower barrier to approval, with fewer prolonged and expensive clinical trials, they would cost less. Goldwater believes right-to-try is the first step on a longer path to push FDA to loosen up on its oversight, ultimately ushering in these savings–although many critics contend drug companies are more likely to pocket those savings as profits.

And the institute has expanded its critique beyond FDA’s drug approval process to question whether FDA should even have authority to regulate some medical products at all. It points to a new generation of stem-cell therapies in which the cells are derived from a patient’s own body, rather than manufactured in a chemistry lab. FDA currently regulates many of these therapies like drugs, but Goldwater argues that because the treatments come from the patient’s own body, the government shouldn’t have the ability to control their use. Safety advocates, meanwhile, point out that treatments aren’t free from harm just because they originated with material from a patient’s own body. Plus, there’s very little evidence these treatments work–exactly why FDA’s oversight is necessary.

Last year, Goldwater also went after FDA’s ability to regulate compounding pharmacies, a type of local drug-manufacturing operation that got extra congressionally mandated oversight after a deadly meningitis outbreak was traced to one in 2012. Compounders can make customized versions of FDA-approved drugs for patients with a special need–say, a liquid version of a pill, or a capsule with an allergy-causing ingredient removed–but they aren’t supposed to make or reproduce versions of drugs not approved by FDA. Goldwater wants FDA to back off its stricter regulation of compounding pharmacy practices.

“I should have the right to determine whether I should be able to take something that helps me." said Colleen Benner, a patient quoted in Goldwater’s compounding report. “I understand that they [FDA] are trying to be protective. But tell me the risks and then let me make my own decision. I don’t need a nanny.”

While the Arizona libertarian organization has become the face of this movement, it isn’t alone. It has allies among some advocacy groups, including Freedom Works and the Abigail Alliance, a group driven by relatives of patients who died unable to get their compassionate-use request filled. It’s also supported by a group of thinkers in academia and business, who object to FDA for ideological or economic reasons.

Some would-be FDA deregulators are in the Trump sphere of influence directly, or via their ties to investor-entrepreneur Peter Thiel, himself a sometimes adviser to the president. A few of the harshest critics of the system were even on the short list for FDA commissioner before Trump settled on a more conventional appointee. Some critics espouse even more radical ideas than right-to-try, including lowering FDA’s approval standards and letting insurance companies act as the gatekeepers for drug effectiveness. A few have gone further, pushing ideas like having FDA evaluate only safety, and get out of the business of requiring proof of effectiveness entirely—or putting drugs on the market with preliminary data, and then collecting more information through a government-sponsored Web portal.

One of these more radical FDA critics currently has a perch within the White House: Tomas Philipson, an economist and health care consultant whom Trump named last summer to the Council of Economic Advisers. Philipson has argued for getting rid of phase III clinical trials, the large final round of human testing that now forms the primary basis for FDA approval decisions.

Alex Tabarrok, a free-market economist at George Mason University who focuses on the regulation of pharmaceuticals, said the current concern about safety needs to be flipped. Instead of worrying about what he calls rare but highly visible cases when an unsafe drug reaches the market, the concern should be about what happens when a drug doesn’t reach patients who need it. Those patients, those who died while waiting for a new drug or who would have benefited from a rejected one, end up in an “invisible graveyard”—a death toll, he argues, that could well be much larger than the number of patients harmed by approved drugs.

“With training, you can
see the invisible graveyard, [and] understand a little bit better that there are costs to FDA-required clinical trials as well as benefits. So, if right-to-try lets the public see a little better some of the costs, then maybe we will have a more balanced system,” Tabarrok said.

Critics worry that all these reforms to get drugs to consumers faster are dangerous. Typical patients just aren’t equipped to make their own sound medical assessments based on complex science and statistics, even if groups like Goldwater insist they have a right to do just that. Even doctors sometimes struggle to sort out pharma companies’ claims from the real benefits of their drugs.

“If you put a drug on the market, who’s really following it? You have social media where you can have people planting whatever information they want. What data would you know is real and which would you know is not?” asked Klein, the former FDA official. “There are people who would take advantage of that. We saw that with AIDS. How many strange approaches to treating AIDS popped up? And people were spending a lot of money on very unproven things, and dying.”

Behind his concern is a bigger and more difficult truth about the drug industry, which is that developing a working drug is hard, and rare—and that the promise of a cure is often illusory. The vast majority of experimental drugs ultimately fail: FDA estimates that for every 100 drugs that enter the first phase of human testing, only five to seven will eventually get approval. The other 95 percent either didn’t work, or had risks that outweighed their benefits, and any patients who took them on an “experimental” basis would have suffered pointlessly.

This was a lesson that a previous generation of pro-access activists learned back in the 1990s, during the AIDS crisis. “I remember the whole idea was that there were like hundreds of drugs at the FDA for AIDS, and if we just scream loud enough or pushed them enough, that dam would break. And there would be this torrent of new therapeutics for people who had HIV,” recalled Gregg Gonsalves, a leader of the HIV/AIDS movement at the time.

Now a public health professor at Yale, Gonsalves said over time, the AIDS activists came to understand that those drugs just weren’t there. Yes, there were potential therapies in the pipeline, but most of them wouldn’t work—and patients weren’t going to benefit from decimating the federal agency that weeds out the quality medicines from the duds.

Gonsalves sees patient groups today operating with a similar set of misguided assumptions—desperate to find a cure, and, as he puts it, “egged on” by groups like Goldwater that use their medical need to help advance a more ideological agenda.

That group now includes the president, who two days after his State of the Union address, again pressed Republican lawmakers to pass the bill at the GOP retreat in West Virginia this week. Patients travel “all over the world to find a cure and we have great experimental drugs, but it will be years before it will come onto the market,” Trump said Thursday. “So it’s called right-to-try and I hope you folks can approve it and I hope you agree with it, but I think it’s so important.”

Though the legislation stalled in the House this summer because of lackluster interest from the two Republican lawmakers with the most oversight of FDA, Trump and Pence’s interest is forcing them to spring into action. Rep. Andy Biggs (R-Ariz.), the co-sponsor of the House legislation, said he was promised a floor vote in early 2018.

If that vote succeeds, many believe patients could be in for a letdown. Though Goldwater may have won its ideological battle with regulators, it’s not clear how big a difference the law will make for people in medical need. Among the skeptics is Trump’s own FDA chief, Scott Gottlieb. “There is a perception that certain products that aren’t being offered under FDA expanded access … will be offered under right-to-try," he testified at a House Energy and Commerce hearing on the topic in early October. "I don’t see that."

via The Agenda

February 2, 2018 at 03:22AM