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Right to hope: New law lets patients request untried drugs, but many skeptical

July 11, 2018
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Vice President Mike Pence chats with young Hoosier Jordan McLinn, a namesake of federal right-to-try legislation, earlier this year at the White House. McLinn, who has Duchenne muscular dystrophy, previously championed passage of Indiana’s Right to Try Law in 2015. (Photo courtesy of The White House)

The national right-to-try legislation that was signed into law in May carries the names of four terminally ill individuals — including 8-year-old Hoosier Jordan McLinn — who wanted the ability to access experimental medication that may help them.

They and many other individuals fighting debilitating, life-threatening diseases are a sympathetic group who right-to-try advocates say should be given the hope of a cure. However, opponents say the law offers little more than hope and could inflict more harm.

The Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017 was passed after the right-to-try effort had caught fire in statehouses across the country. At least 40 states have passed right-to-try laws, according to the National Conference of State Legislatures. Indiana’s own version breezed through the Legislature in 2015 and ended with McLinn standing beside then-Gov. Mike Pence during the bill signing ceremony.

Former Indiana Rep. Wes Culver, R-Goshen, authored the Indiana law and characterized it as giving patients hope. That same sentiment was echoed by Sen. Ron Johnson, R-Wisconsin, when he urged his colleagues in the U.S. Senate to approve the federal right-to-try law.

“(This) is something that I think is going to give so many thousands — maybe tens of thousands, maybe millions — of Americans hope when they face a similar type of disease, where there is no hope, where there are no further options, other than potentially an experimental drug that has been proven safe, according to the FDA,” Johnson said.

Mohapatra Mohapatra

Seema Mohapatra, incoming health law professor at Indiana University Robert H. McKinney School of Law, is skeptical about the impact the new law will have. She pointed out the legislation does not guarantee patients will have access because drugmakers can still turn down requests for their experimental medicines.

In addition, Mohapatra and others highlighted the U.S. Food and Drug Administration’s expanded access program that helps patients obtain medications that are still undergoing clinical trials. An analysis of the FDA data shows between 2010 and 2015, the agency authorized 99 percent of patient applications for access to drugs still in clinical trials.

Mohapatra said the FDA’s program seems to be working well at providing a means to get the treatment while also helping to ensure patient safety and efficacy. In particular, the program enlists a review board to make certain that the patients have utilized all available options and are as well informed as possible about the potential risks of the experimental drug.

“Terminally ill patients are in a vulnerable position and the compassionate use program protects these patients,” she said.

When to give access

Backing the right-to-try movement is the Goldwater Institute, a libertarian think tank, which sees the legislation as protecting the “fundamental right” of people to try to save their own lives.

Speaking against the Trickett Wendler bill on the House floor, Rep. Frank Pallone, Jr., D-New Jersey, summarized the prevailing objections many have to the law. He described it, and a similar bill that was being considered at the time, as an “egregious attempt” to undermine the FDA’s drug approval process. Also, he pointed out that more than 100 patient organizations, including the National Organization for Rare Disorders, and four former FDA commissioners under the Obama and Bush administrations, opposed the legislation.

“There is no evidence that either bill would meaningfully improve access for patients, but both would remove FDA from the process and create a dangerous precedent that would erode protections for vulnerable patients,” Pallone said.

Fleury Fleury

The American Cancer Society Cancer Action Network was among the nonprofits and professional associations that argued against the right-to-try law. Mark Fleury, principal of policy development and emerging science at the network, said his organization fully supports the ability to access unapproved therapies, but it does have concerns about the conditions under which access is given.

Primarily, he said, the worry is that drugs being funneled through right-to-try would lose the benefit of the FDA expertise.

Under the FDA’s expanded access program, experimental drugs that have navigated the three phases of a clinical trial can be provided to patients. Phase I of a clinical trial, which can involve 20 to 100 individuals, just seeks to determine how the drug interacts in the body and whether it is safe. Phase II can encompass several hundred patients and tests to see if the drug is effective at treating the disease, while phase III can bring in a couple of thousand patients to study if the drug is beneficial.

The right-to-try law allows drugs that have only been through phase I to be offered.

Focus Right-to-Try factboxAny bad reactions that occur during a clinical trial must be reported to the FDA immediately. Conversely, right-to-try loosens that requirement to annual reporting. Moreover, the drug manufacturers and medical professionals are protected from liability if things go wrong.

“Overall the question is, could unsavory actors take advantage of patients under right-to-try? Yes, that is our concern,” Fleury said. “Will most pharmaceutical companies behave in an ethical manner and take care in what they give us? Yes. But right-to-try opens the door for the abuse of the system and patients being taken advantage of.”

Wait and see

Ultimately, right-to-try patients will not have control over whether they get the experimental drugs. That will rest largely with the pharmaceutical companies, just as it currently does with the FDA expanded access program.

According to Fleury, of the compassionate use requests that are turned down, most are denied because the drug manufacturer does not want to provide the product. The reasons can include the patient’s disease does not match the condition the medicine is targeting, or the manufacturing process is not far enough along to make the additional doses needed to fill the access applications.

In 2007, the U.S. Court of Appeals for the District of Columbia Circuit examined access to drugs in Abigail Alliance for Better Access to Developmental Drugs v. von Eschenbach, 495 F.3d 695 (D.C. Cir. 2007). Although the court did not address right-to-try specifically, it did find there is no fundamental right to experimental therapies.

The 8-2 majority sympathized that terminally ill patients need curative treatments, but noted the experimental drugs could end up hastening their deaths. “We therefore hold that the FDA’s policy of limiting access to investigational drugs is rationally related to the legitimate state interest of protecting patients, including the terminally ill, from potentially unsafe drugs with unknown therapeutic effects.”

Cost might be another factor curbing access. Drug companies are free to charge patients for the investigational drugs and physicians as well as hospitals will also likely charge to administer the medication. Even patients with health care coverage may have trouble paying because most insurance policies do not provide for experimental therapies.

For now, Fleury said his organization is watching to see how right to try is implemented and what patients experience.

Sen. Joe Donnelly, D-Indiana, was the leading co-sponsor of the new right-to-try law. He was frank with his Senate colleagues that the Act is not a cure-all, but he emphasized what he believes the law will do.

“This gives folks a shot,” Donnelly said. “It doesn’t provide any guarantees, but it allows folks to be able to take their care into their own hands, to make judgments, and to decide: I want to take a shot at this.”•

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