If you were dying and had exhausted all conventional treatment options, wouldn’t you want immediate access to a drug that might prove to be a miracle cure?
That’s the promise of legislation that, if signed by Gov. Jay Nixon, would make Missouri the third state in the country – after Colorado and Louisiana – to enact a so-called “Right to Try” law, which aims to get investigational drugs into the hands of terminally ill patients as quickly as possible.
“If there is something out there, particularly if there are medications that are being used in India or Europe, why do we in the United States have to wait for the (Food and Drug Administration) to give the blessing?” said state Rep. Jim Neely, the Cameron Republican who sponsored the bill.
Neely is a longtime family physician with a daughter who is battling advanced colon cancer.
Working with the Phoenix-based Goldwater Institute, a free market-oriented think-tank seeking to enact similar laws in states across the country, he is also backed by patients and families who have lost relatives.
The FDA is neutral on the initiative, according to a spokeswoman, but the institute’s efforts are opposed by a leading drug industry interest group, the Pharmaceutical Research and Manufacturers of America (PhRMA).
Other skeptics include Russell Melchert, dean of the University of Missouri-Kansas City School of Pharmacy, and Melissa Gilmore, a Washington, D.C., attorney who leads the FDA practice at the Polsinelli law firm, which is based in Kansas City.
“In my opinion,” Gilmore said, “the legislation is basically pointless.”
In general, the legislation would allow patients and their physicians to work directly with drug companies to obtain investigational drugs outside the process established by the FDA.
A Nixon spokesman said the governor is reviewing the measure and has not indicated whether he will sign the bill.
Path to approval
It can take as long as 15 years and $1.8 billion to bring a drug to market, according to research cited by PhRMA.
In its 2013 industry profile, the group said the 44 new medicines blessed by the FDA in 2012 were the largest number of approvals in 15 years.
Once laboratory studies show promise, drug developers can get approval from the FDA to begin clinical trials, which use human subjects to determine if a compound is safe and effective.
The trials run through four phases, with Phase 1 involving a small group of people who evaluate a drug’s safety, dosage range and side effects.
Recognizing the dire straits faced by dying patients who cannot get into clinical trials, the FDA makes unapproved drugs available through its Expanded Access, or “compassionate use,” program.
The requests typically come from patients’ physicians, agency spokeswoman Stephanie Yao said in an e-mail. But a company can also request participation for an intermediate-sized group of two to 99 patients or under a treatment protocol for a larger group if the patients do not meet the criteria for a clinical trial.
An FDA official is authorized to give emergency approval over the telephone.
In petitions from individuals, the FDA cannot compel a drug company to provide the investigational drug.
In the last four reporting periods, covering October 2009 through September 2013, the FDA received an average of 391 emergency requests from patients and approved all but five.
Much of the disagreement about the Right to Try legislation stems from differing views about the effectiveness of the FDA’s expanded access program.
Right to Try proponents argue expanded access takes too long to help patients who are in desperate shape.
The process can take as long as six months and involve dozens of hours of paperwork for the physician, said Victor Riches, vice president of the Goldwater Institute.
“While the expanded access program is a great program for a lot of folks,” he said, “it is not always beneficial and usually is not applicable for these patients that have gone through every other effort to try and save their life, and this is really their last opportunity.”
Under Right to Try, Riches said, the patient could get the drug in days as opposed to months.
As with the expanded access program, Neely’s bill would not require a manufacturer to fulfill the request. The bill also stipulates that:
The manufacturer can require payment for the drug
Health insurers are not required to cover the service
The state licensing board cannot take action against a physician based solely on the physician’s recommendation that a patient try an investigational drug
Riches acknowledged the effectiveness of Right to Try depends on the willingness of drug manufacturers to participate. He says most are waiting to see how the FDA will react to these bills.
“There is no question that for these laws to be maximally effective, there needs to be a positive balance between doctors, the pharmaceutical companies, the FDA. Everyone needs to be working together to really make these things work,” Riches said.
Blame the companies?
Gilmore, the Polsinelli attorney, said the real problem is not the FDA or its administration of the expanded access program.
Rather, she said, it is drug companies’ refusal to make the investigational drugs available even after the FDA has given them the go-ahead.
Gilmore said that, as part of her pro bono work, she has gained about a dozen FDA approvals through the expanded access program. All have foundered, she said, because drug companies declined to participate.
Gilmore suspects they feared bad publicity or had concerns a negative outcome could affect final approval of the drug.
Officials with PhRMA declined to respond to Gilmore’s comments on the record.
Instead, they cited a June 14 post on their website by Sascha Haverfield, the organization’s vice president of scientific and regulatory affairs.
“Expanded access programs are part of many biopharmaceutical companies’ commitment to patients,” he wrote.
He added that, while the Right to Try bills may be well-intentioned, “they seek to bypass FDA oversight and the clinical trial process, which is not in the best interest of patients and public health, and is unlikely to achieve our shared goal of bringing innovative, safe, and effective medicines to patients as quickly as possible.”
Likewise, Melchert, the UMKC dean, said he does not see how Right to Try legislation would be of any benefit.
He says the FDA’s expanded access program is straightforward and information is available to help patients understand the process.
“It is all in place,” he says. “I really don’t see why we would need anything else.”
And even that process is not without risk, Melchert adds, given that a drug that has cleared a Phase I trial could lead to the immediate death of someone who still might have six to nine months to live.
Melchert said he would like to see the energy around the Right to Try effort channeled into the more important issue of directing additional money to research for new cures and to wellness campaigns to prevent disease.
Bill hits home
As word of Neely’s bill circulated in Jefferson City, the effort hit home with a couple of lobbyists.
One is Ross Nichols, who works for the Missouri Trucking Association. The other is Richard McCullough, who represents several clients in the capitol.
Nichols, a 41-year-old father of two boys, has a brain tumor that was diagnosed nearly three years ago. He has had two operations but a recent check-up revealed another growth.
He is changing treatments, but he said it would be comforting to know that, if Missouri enacts the Right to Try law, he and his family would have access to a last-resort treatment.
McCullough’s support for the measure comes from his son, Nick, who died in December 2011 at the age of 27. He had battled a head-and-neck cancer for three years before his death, but had been an unsuitable candidate for a couple of clinical trials, McCullough said.
Neither man knew of the FDA’s expanded access program but both are enthusiastic about Neely’s bill.
Nichols said he prefers solutions that involve less government. McCullough favors state-level solutions.
Referring to his son’s experience, McCullough said, “The name Right to Try is very appropriate because he never had a chance to try.”