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‘Right to try’ law gives terminal patients access to drugs not approved by FDA

June 21, 2014 at 12:07 PM EDT
In May, Colorado became the first state to pass a so-called 'right to try' law, allowing terminal patients access to experimental drugs without FDA approval -- and Missouri is about to follow suit. NewsHour Weekend examines the issue by speaking with the Missouri bill's sponsor and his daughter, who is suffering from cancer.
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STEPHEN FEE: In early 2013, Kristina Brogan was pregnant with her fifth child when she began experiencing excruciating pain — and her obstetrician didn’t know why.

KRISTINA BROGAN: Finally my mom went with me to an appointment and said you got to find out why she’s hurting so bad. So they did a level two ultrasound at St. Luke’s and admitted me immediately.

STEPHEN FEE: Why? What did they find?

KRISTINA BROGAN: They found the tumor. And they’d found it had gone up into my liver.

STEPHEN FEE: Kristina, at the age of 39, was diagnosed with stage four colorectal cancer.

STEPHEN FEE: What was that like?

KRISTINA BROGAN: Scary.

STEPHEN FEE: What was running through your head?

KRISTINA BROGAN: I can’t say I had a – a normal thought in my head. I was just scared. Scared for my baby. Scared for me. Scared for my family.

STEPHEN FEE: Her doctors needed to take aggressive measures to fight the disease and chose to perform a C-section, just 28 weeks into Kristina’s pregnancy. Today, her son Evan is a happy and healthy one-year old. Kristina however is battling a disease with dispiriting odds even with regular chemotherapy treatment.

STEPHEN FEE: What did they tell you your future looked like?

KRISTINA BROGAN: They didn’t. And I don’t want them to. I will do what the doctors say. Do my best to get better.

STEPHEN FEE: While her four other boys and husband Jeff live in Gallatin, Missouri, Kristina has moved about 30 minutes away to the town of Cameron, where her mother, sister, and stepfather can help care for her. Her stepfather Jim Neely is a practicing physician at the local hospital.

JIM NEELY: I think as you age you tend to recognize the sanctity of life. But you also through the process of living you, you have been able to – deal with a lot of heartache in your activities.

STEPHEN FEE: Neely has decided to make public this very private, family matter. See, in addition to being a doctor, he just finished his first term as a Republican lawmaker in the Missouri House of Representatives. And this year, he introduced a bill that would allow terminally ill people to take unapproved, experimental medications outside of clinical trials — and without the federal government’s go-ahead. It’s called ‘the right to try.’

JIM NEELY, (R) MISSOURI STATE REPRESENTATIVE: Let’s try, let’s let a person be in charge of their life, and I’m not saying that this is the answer for everybody. But it’s an answer out there that if we can move forward and we’re gonna gain access to care. We want – we want options in life. And that’s what this bill’s trying to accomplish.”

STEPHEN FEE: Patients and their doctors can already petition a drug company to try an investigational medicine, even outside a trial. But they need approval from the federal Food and Drug Administration. ‘Right to try’ bills are state laws that would bypass that federal approval step — and similar measures are gaining ground nationwide.

Here in Missouri the ‘right to try’ law passed with unanimous support in the state legislature, as it did in Colorado and Louisiana. And this fall Arizona voters will have their own version of right to try on the ballot.

This May, Colorado’s Democratic governor signed the nation’s first ‘right to try’ bill. Like Missouri’s law — passed by a
Republican legislature — it cuts the feds out of the picture.

GOV. JOHN HICKENLOOPER, (D) COLORADO: Patients should be able to try a treatment even though it hasn’t been approved if it’s an attempt to save their life.

STEPHEN FEE: Lawmakers in that state began calling the measure the Dallas Buyers Club bill — after the 2013 Oscar-winning film about AIDS patients seeking unapproved medications, and one man’s efforts to sidestep federal rules.

MATTHEW MCCONAUGHEY, DALLAS BUYERS CLUB: They’re not illegal. They’re merely unapproved.

STEPHEN FEE: Adding to the Colorado bill’s momentum, the story of 41 year old Denver dad Nick Auden. Though he would later succumb to stage four melanoma, Auden’s family made an emotional online appeal for an experimental drug. Auden was on a trial for the promising pharmaceutical but was disqualified after a complication.

NICK AUDEN: If there’s a chance, we’re taking it. If there’s a possibility, then we’re gonna be part of that possibility. What’s more, a conservative think tank in Arizona — the Goldwater Institute — has lent its support to the ‘right to try’ movement, even helping lawmakers like Jim Neely draft legislation.

GOLDWATER VIDEO NARRATOR: …shifting the decision to use safe, new medications out of the hands of bureaucrats…

STEPHEN FEE: While the FDA doesn’t take a position on ‘right to try’ laws, the agency says it already has at least three pathways to allow patients access to experimentals. There are clinical trials, of course — those are run by drug companies and overseen by federal regulators. But if a patient doesn’t qualify, the FDA allows individuals and even groups of patients to apply for access to experimental meds outside of a trial. Since 2009, the FDA says it has greenlit 99 percent of those individual and group applications.

RICHARD KLEIN, FDA PATIENT LIAISON PROGRAM: The agency has a pathway. It seems to work quite well, and I’m not sure what the state right to try bills really add to that — and in fact I think might take away some of the safety advantages people have by going through the FDA process where you’ve got institutional review boards, you’ve got somebody checking the informed consent, make sure the patients are fully aware of what they’re getting into, and what’s the balance that we know so far about this particular product.”

STEPHEN FEE: Bioethicist Arthur Caplan believes ‘right to try’ laws are well-intentioned…especially with the time it takes to get new drugs to market.

ARTHUR CAPLAN, NYU LANGONE MEDICAL CENTER: That process can take years. And for many people who are terminally ill they don’t have years. So the laws basically say, you know what? We’re tired of all these bureaucratic obstacles; we’re going to pass a law in our state that says, if you’re terminally ill, once it’s passed phase one — that tiny safety testing on a small number of people — you can get anything you want.

STEPHEN FEE: But Caplan says some federal regulation is still
necessary to ensure patients are aware of risks.

ARTHUR CAPLAN, NYU LANGONE MEDICAL CENTER: Because many new drugs can make you die faster. And I’ve personally given access through sitting on research ethics committees and working with the FDA to many new drugs. A number of them killed the person who got them more quickly than they would’ve died from their disease. They just had horrible side effects. You can also die more miserably from access to a new drug.

STEPHEN FEE: So if the FDA has such a high approval rate when it comes to experimental access, why have so many taken to the web to plead their cases? Well sometimes, the drug companies say no. And ‘right to try’ bills wouldn’t change that. The Pharmaceutical Research and Manufacturers of America – an industry group — told us their members have to evaluate the potential ‘harm or pain’ their investigational meds might cause. And they consider all applications on a ‘case-by-case basis.’ Back in Missouri, lawmaker Jim Neely concedes the federal government
isn’t the only hurdle to accessing investigational drugs.

JIM NEELY, (R) MISSOURI STATE REPRESENTATIVE: But this is a step in the right direction. We need the FDA in our world, but we need access to care, and I firmly believe that we’re going to see a boatload of new medicines that are out there in the investigational world, and we need to gain access to those at a faster pace.

STEPHEN FEE: And for Kristina, his step-daughter struggling with a dire prognosis, government regulators have no place in her medical care.

KRISTINA BROGAN: They’re not sitting there counseling me on these medications and what I should do, what I shouldn’t do. How to take care of myself to keep myself healthy. And my doctor is, and if my doctor thinks this medication would help me in some way, then that’s up to my doctor. That shouldn’t be up to somebody that has no involvement in my care.

STEPHEN FEE: Between you and your doctor.

KRISTINA BROGAN: Right.

STEPHEN FEE: Kristina isn’t taking any investigational drugs, and at this point her doctors are not seeking access to one of the 40 clinical trials for her type of cancer. But at a later stage, if she opts to try an investigational drug as a last resort outside of a trial, she wants that choice to be hers alone.