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Brainstorm Health: Right to Try, Alzheimer’s Blood Test, Vertex Cystic Fibrosis Trials

February 1, 2018, 7:14 PM UTC

Hello, readers! This is Sy.

President Donald Trump on Tuesday delivered his first State of the Union address. In a wide-ranging, 80-minute speech, the president touched on the economy, immigration, infrastructure, prescription drug prices, and myriad other issues (you can read the full State of the Union transcript here). But one piece of health care legislation the president urged Congress to pass (that was not a repeal of Obamacare) may be less familiar to the public than others—a “Right to Try” bill for terminally ill patients.

So what exactly is Right to Try? Simply put, it’s proposed legislation that would give Americans with terminal illnesses more latitude to take experimental treatments that have yet to be approved by the Food and Drug Administration (FDA). Patients, under the advice of their doctors, would be able to reach out to a drug manufacturer to request an investigational therapy without having to go through the FDA. A federal version of a Right to Try bill already passed the Senate and may, with Trump’s and Vice President Mike Pence’s full-throated support, ultimately clear the House of Representatives.

“We also believe that patients with terminal conditions should have access to experimental treatments that could potentially save their lives,” said Trump during the State of the Union. “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 is time for the Congress to give these wonderful Americans the right to try.”

Perhaps the most interesting aspect of Right to Try is that it’s created some strange bedfellows among politicians and patients alike. There are already 38 states with their own versions of such laws on the books, signed by scores of GOP and Democratic governors alike; the version that passed the Senate was a bipartisan proposal from Democratic Sen. Joe Donnelly of Indiana and Republican Ron Johnson of Wisconsin. But Rep. Frank Pallone, the ranking Democrat on the House Energy and Commerce Committee, argues that such a law “could expose seriously ill patients to greater harm instead of the greater access that they are looking for.”

There’s some disconnect among patient groups, too. For instance, organizations like Teen Cancer America and the Rare Disease United Foundation are strong supporters of Right to Try; on the flip side, the National Organization for Rare Disorders (NORD) “opposes the Right to Try Act very strongly,” the group’s director of federal policy, Paul Melmeyer, told Fortune in an interview. “We quickly found that it would actually be harmful to our patient base.” NORD and other Right to Try critics usually note that the FDA already has a compassionate and expanded access program for terminally ill patients and that “99.3% of the time, the FDA grants these requests,” and within an average of about four days. Those numbers check out—the FDA approved anywhere from 98.9% to 99.4% of expanded access requests between 2013 and 2017.

But proponents retort that the process is still cumbersome and, on a philosophical level, a patient with a terminal illness shouldn’t have to as the government’s permission if there’s the smallest chance that a medicine could help mollify his or her pain or even save his or her life. (Strikingly, Right to Try laws have also been called “Dallas Buyers Club” laws, a reference to the award-winning 2013 biographical film about Ron Woodroof, who worked around the system in order to secure unapproved HIV/AIDS medications for patients following his own AIDS diagnosis.)

The conservative-libertarian think tank Goldwater Institute is one of the leading forces championing Right to Try, and its director of health care policy, Naomi Lopez Bauman, argues that the federal version of the legislation does, in a sense, still preserve the FDA’s oversight role. “There’s a requirement that the drugs being requested have to pass a phase one clinical trial, which is a small safety trial, which means that you know there are other patients that are simultaneously taking the treatment,” Bauman told Fortune in an interview. Furthermore, such a request must be submitted by a physician, and some companies may feel uncomfortable issuing treatments to terminal patients under the current system over fear of liability in the case of adverse events—firms would receive immunity from retaliatory lawsuits under Right to Try.

The main concern for many critics is that largely removing the FDA from the expanded access equation may also lead to less effective use of experimental drugs; for instance, a 2016 FDA study found that, out of 150 randomly selected expanded access proposals, the agency recommended dosing, informed consent, or safety monitoring changes to the submitted proposals 11% of the time.

But the passion driving the Right to Try movement, and its new high-profile champion in the White House, may well convince lawmakers that the concerns are overstated, and that Right to Try would ultimately do more good than harm.

Read on for the day’s news.

Sy Mukherjee


We may be inching closer to an Alzheimer's blood test. A Japanese study published in the journal Nature suggests that a blood test could actually be used to check for Alzheimer's disease. How? By detecting biomarkers that suggest the buildup of amyloid beta protein, which leads to plaque buildups in the brains of Alzheimer's patients. Currently, amyloid detection requires more invasive and expensive procedures like lumbar punctures or PET scans. (Scientific American)


Vertex presses on with triple combination cystic fibrosis drugs. Rare disease drug maker Vertex Pharmaceuticals is betting that new triple-drug combinations for the lung disease cystic fibrosis, which the company will be pushing into late-stage trials, can help treat a far wider swath of patients with the illness. Vertex's current CF medications, Kalydeco and Orkambi, can treat about 45% of the disease population. (Reuters)


The flu is so bad it's forcing hospitals to take extreme measures. The Wall Street Journal reports on some of the extraordinary moves hospitals are being forced to take during this year's brutal flu season, including postponing elective surgeries that would take up beds, expanding emergency rooms into waiting rooms, and even setting up external mobile units. It goes to show that severe flu seasons aren't just bad from a public health standpoint, but also big financial hits on hospitals, which aren't reimbursed all that much for flu care compared to other services. (Wall Street Journal)


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Produced by Sy Mukherjee

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