'Right to Try' law would be no miracle cure

NASHVILLE — Marty Nord does not expect a miracle cure if Tennessee gives her the right to try experimental drugs for Lou Gehrig's disease.

Her disease has progressed to the point that she uses a wheelchair and relies on a device to help with breathing.

Nevertheless, she supports a Tennessee bill that would allow doctors to prescribe medicines not approved by the U.S. Food and Drug Administration — legislation being pushed by the Goldwater Institute, an organization that advocates for personal freedoms over federal regulations.

Eight states have now passed "Right to Try" laws. Bills are moving through several other states, including Tennessee.

Basically, the law would allow Tennessee doctors to prescribe any drug in FDA clinical trials that has already undergone phase 1 testing, which checks for safety in healthy volunteers. People with incurable diseases could get the drugs even though the agency hasn't determined their effectiveness.

"I have had ALS for five years," Nord said. "There are certain things that happen to you that can't be corrected. When you lose muscle mass in ALS, they are not looking for ways to get well. Fixing that muscle mass is something that is not going to be easy to do. Now, slowing it down and maybe eventually stopping it? Yes, that's a viable approach."

A state law also might allow her quicker access to specialized equipment to maintain and improve the quality of her life, she said.

Nord is a former university professor, a business communications specialist and a realist. She's educated enough to avoid false hopes.

Physician concerns

A patient's overoptimistic expectations is just one of the scenarios that a Vanderbilt University Medical Center physician who specializes in palliative care worries about with the new legislation.

Dr. Mohana Karlekar said side effects from experimental drugs in healthy volunteers could be magnified in sick people. She's also concerned about families demanding drugs that doctors might not feel comfortable prescribing.

"When I think about the issue, I have a hard time wrapping my brain around if I would fully support it or not support it," Karlekar said. "I can argue both sides pretty vigorously. I think having people have options is always a good thing, but I think in health care people very often don't know what those options mean."

She said she's encountered people with metastatic cancer who can't answer whether they are taking a drug treatment with the goal of curing the disease or extending life and easing symptoms.

"Before people decide it's great or it's terrible, they really need to think it through about all the nuances," Karlekar said. "What might be good for one may not be good for another."

Experimental drugs are already available for patients with incurable diseases if they live in an area where they can get into a clinical trial and meet its protocols. But only about 3% of people with medical conditions get into clinical trials, said Kurt Altman, national policy adviser for Goldwater Institute.

He also noted that drugs the FDA still has in clinical trials are already market approved in Europe — medicines that can be lifesaving.

Although clinicaltrials.gov is a resource for people searching for those trials, the information on the website isn't always reliable.

Researchers on a majority of those drug trials failed to report results back to the website in a timely manner as required, according to a review by a team from Duke University published this month in the New England Journal of Medicine. Without those results being posted, people do not know whether a trial shows enough promise to join.

Also with clinical trials, one group receives a placebo or the currently approved best treatment while another group receives the actual experimental drug during a set period of time. Researchers then compare patient results to determine whether the drug is effective.

Possible federal challenge

As "Right to Try" bills are approved by state legislatures and signed into law by governors, the stage is being set for a possible challenge from the federal government. Altman said he believes "Right to Try" laws will prevail because of the precedent set by a right to die case. In 2006, the U.S. Supreme Court ruled in favor of Oregon when the U.S. attorney general challenged a physician-assisted suicide law in that state.

But this legislation is about the "right to survive." That's how Rep. Jon Lundberg, R-Bristol, the sponsor of the House version of the bill, describes it. But he also stressed the legislation is no panacea for incurable diseases.

"The right to survive is that inherent element that most of us have that is the most important, fundamental piece of DNA that I think we have," Lundberg said to fellow legislators. "Government, though, has put up a blockade for those who want to try new things, new drugs, new biologics, new devices that have come out."

Pressure on decision makers

Although ALS has put Nord in a wheelchair, it has not confined her. Still in charge of the gardening at her home, she had a gardenia blooming in a sunny window on a winter day.

She said she has participated in two clinical trials, but the focus of those was on better understanding the progression of ALS, not testing a cure. Millions of dollars raised through the ALS Ice Bucket Challenge for research should spur more drug research, she said.

"The legislation can only go so far," she said. "If the FDA trumps it and says 'no, you can't do it,' then the legislation is wasted. The pressure needs to be where the decisions are actually made."

'Right to Try' law

The legislation would prohibit disciplinary actions against a doctor for prescribing an investigational drug to a patient. However, it would not require insurers to expand covered medicines or manufacturers to provide products to patients. Patients with progressive diseases for which there are no available treatments would be allowed access to experimental drugs and devices recommended by their doctors.