While the “Right to Try Act,” which aims to give dying patients the right to try unapproved experimental drugs, is law in Florida as of today, its implementation isn't so clear.
In theory, the Right to Try law allows terminally ill patients access to drugs that have passed first-phase clinical trials and are going through later-stage trials as part of a new drug application to the Food and Drug Administration.
But in order for the law to work, drug companies have to be willing to give or sell the drugs to patients or their doctors without involving the FDA, the same agency the company depends on for the drug’s eventual approval.
That tension is creating problems, say officials from the libertarian Goldwater Institute, which has spearheaded the bill in Florida and other states. Drug companies see a lot of risk in providing a drug in the approval pipeline to a patient already in advanced stages of a disease.
“They’re worried about getting sideways with the FDA,” said Kurt Altman of the Phoenix-based institute, which began promoting the bill 18 months ago.
Florida was the 21st state to enact it with Gov. Rick Scott’s signature last month; two more states lack only their governors’ signature. The measure is pending in at least a dozen more.
But not a single patient has received access to an investigational drug through a state Right to Try law. Altman predicts a breakthrough by the end of the summer.
To ratchet up public pressure on the companies and the FDA to cooperate, the Goldwater Institute launched a website and a petition drive. It features personal stories of terminally-ill children and their parents pleading for one last try.
The FDA denies it is blocking access to the drugs and has taken no position on Right to Try.
“It is critical for the public to understand that FDA is not a barrier to accessing investigational drugs or medical devices,” press officer Sandy Walsh said in an email. The FDA “helps to ensure patients are adequately protected from receiving a potentially harmful or ineffective treatment.”
Walsh said keeping the FDA involved in the process also ensures the collection of data to guide FDA decisions. “The drug approval process represents the best way to assure development of and access to safe and effective new medicines for all patients,” Walsh wrote.
FDA has its own compassionate-use process, created in 2009. But the agency's own officials concede Expanded Access is so cumbersome to use that relatively few who could benefit apply. Right to Try advocates say bypassing the FDA program could save weeks or months, precious time for patients and their families.
When Right to Try came to the Florida House floor in April, several members spoke of the personal impact of the bill.
Debbie Mayfield, R-Vero Beach, said her husband Stan responded to treatment with a cancer drug when he was enrolled in a Phase 1 clinical trial at M.D. Anderson in Houston. But he could no longer get the drug after that trial ended. He died in 2007, three months shy of their oldest son’s graduation..
“Time is so important to cancer patients,” she said.
The House bill sponsor, Rep. Ray Pilon, said his 36-year-old son has colorectal cancer. While he’s doing well in treatment now, that could change. “So, this is personal to me,” said Pilon, R-Sarasota.
The act passed the House unanimously and got overwhelming bipartisan support in the Senate.
These moves by Florida and other states will force the FDA to make compassionate use easier and faster, Pilon said. “The passage of this bill…will finally make the federal government wake up and say, ‘There’s a better way to do this.’”
While the the FDA estimates its “Expanded Access” program application may take up to 100 hours to complete, Associate Commissioner Peter Lurie said the agency was proposing a shorter, simpler form expected to take just 45 minutes. The FDA also has streamlined its website, he said.
The new law does not require drug companies to make medications available. It just shields the company, as well as doctors and health facilities, from liability if it does. It also doesn’t require insurers to cover the treatment, meaning companies either have to give the drug for free or try to get money from a dying patient.
Walsh, from the FDA, said it is not unusual for the company to refuse, she said.
“Before a request for access to an investigational drug can be considered, a drug company must be willing to provide the drug,” she said. “The FDA cannot legally require a drug company to make a drug product available.”
The industry’s lobbying arm, the Pharmaceutical Research and Manufacturers of America (PhRMA), has taken no position on the state bills. State laws are well intentioned, but do little to improve the FDA’s process, said Sasha Haverfield, PhRMA vice president for scientific and regulatory affairs.
"We have serious concerns with any approach to make investigational medicines available that seeks to bypass the oversight of FDA and clinical trial process,” she said in an email. That “is not in the best interest of patients and public health.”
In December, PhRMA’s board adopted new principles on the safe and ethical conduct of clinical trials. Member companies said while the best route for access to investigational drugs is through clinical trials, they are committed to expanding access to the drugs for patients who don’t qualify for the trials or can’t participate.
There were several caveats: There is enough evidence to indicate the drug won’t do harm, even if it doesn’t do any good. There is enough of the drug left over after supplying patients in clinical trials.
And, the group said, access to the drug “should not delay, interfere or compromise the completion of clinical trials that are intended to support approval by national regulatory authorities.”