Hopes rise for law to expand access to experimental drugs

Hopes rise for law to expand access to experimental drugs

(This story originally appeared in The Hill Extra.)


Jonathan Friedlaender had advanced metastatic melanoma, could barely eat, and nurses urged him to consider hospice care. 

More than two years later, he’s outlasted the doctor’s prognosis, in part, after taking an immunotherapy drug not yet on the market through the Food and Drug Administration’s expanded access program.

Friedlaender seems like the perfect advocate for Right To Try laws, which allow terminally ill patients to use unapproved drugs that have only passed the FDA’s basic safety test.

He isn't.

“I was a real miracle case. I was furious at the drug companies before that for not speeding up the authorization. So you would think I would be very much in favor of the Right to Try law," Friedlaender told The Hill Extra

Friedlaender’s concerns with the law echo those of other opponents. The legislation can’t compel drug companies to provide the drug to those who want to try it, and they’re oftentimes reluctant to do so, because of liability and cost issues. There’s also worry that it erodes the FDA’s power, and some note the agency already has a program that lets patients try investigational drugs.


But with Vice President Mike PenceMichael (Mike) Richard PenceKamala Harris shopping trip stirs Twitter campaign trail debate Pence met with silence after mentioning Trump in Munich speech Bill Kristol resurfaces video of Pence calling Obama executive action on immigration a 'profound mistake' MORE as a key ally in the White House, Right to Try advocates believe this is the year they'll finally see federal changes letting terminally ill patients try experimental drugs. More than 30 states have the law on their books, and Republicans in both chambers recently reintroduced federal legislation.

Congressional disclosure forms show an increased number of groups lobbying on the issue in 2016, with, for example, drug company AbbVie, the American Association for Justice, and Pharmaceutical Researchers and Manufacturers of America writing the issue on man­dat­ory fil­ings un­der the Lob­by­ing Dis­clos­ure Act. 

Reviving the debate. 

Families backing Right to Try met earlier this month with Pence, who has been an outspoken supporter, signing a law when governor of Indiana. Supporters say burdensome FDA regulations slow down drug approvals for terminal patients who don’t have time to wait. 

“It was a very gracious meeting,” Kelly Lange, who's on METAvivor's board of directors and a past president of the group which raises awareness about advanced breast cancer, told The Hill Extra. “They were very welcoming and supportive of the legislation, and I think with the administration’s approval and support, as well as the Republican majorities in both Houses of government, I think we have a good chance of getting this legislation passed this year.” 

Supporters argue that people who are near the end of their life should have a right to take riskier medicines. They’ve received a sympathetic ear in 33 states, and the meeting with the vice president marks a major moment for the movement. 

In addition, President Trump suggested he'd make changes to the drug rules for terminal patients in late January, saying not receiving drugs has “always disturbed” him.

Starlee Coleman, of the limited government group, the Goldwater Institute, said their organization has created the model for the legislation introduced in all 50 states. She expects future meetings with Pence, and said the relationship with the current administration is more promising than that with the Obama administration, which was not active on the issue.

“However this issue is resolved, whether it is through Congressional mandate to the FDA or whether or not it is the administration making the change itself at the FDA, we feel reasonably confident that there will be movement this calendar year,” Coleman told The Hill Extra. “And it could come from either direction, and maybe both.”

The Goldwater Institute argues the FDA already has the authority to make the necessary changes, which makes Trump's pick of an FDA commissioner a key choice, but they are also open to legislative changes at the federal level. 

“All the people that we have heard who have been put on the short list [for commissioner] would be favorable toward Right to Try, so we feel like any way he goes, we’re in good shape,” Coleman said. 

Sen. Ron JohnsonRonald (Ron) Harold JohnsonGOP senator voices concern about Trump order, hasn't decided whether he'll back it GOP braces for Trump's emergency declaration GOP wants to pit Ocasio-Cortez against Democrats in the Senate MORE (R-Wis.) has been a staunch advocate for Right to Try laws in the Senate. Last congressional session, he asked his colleagues to pass the bill by unanimous consent, but was shut down by then-Senate Majority Leader Harry ReidHarry Mason ReidConstitutional conservatives need to oppose the national emergency Klobuchar: 'I don't remember' conversation with Reid over alleged staff mistreatment Dems wary of killing off filibuster MORE (D-Nev.). 

Reid argued the proposal shouldn't be jammed through without more study.

The reintroduced legislation, S. 204, has 44 cosponsors, including two Democrats and independent Sen. Angus KingAngus Stanley KingDrama hits Senate Intel panel’s Russia inquiry Warner, Burr split on committee findings on collusion Overnight Defense: Top general wasn't consulted on Syria withdrawal | Senate passes bill breaking with Trump on Syria | What to watch for in State of the Union | US, South Korea reach deal on troop costs MORE (I-Maine). Johnson plans to try to attach the measure to other pieces of legislation moving this year. 

“To me, it’s just a basic issue of freedom,” Johnson told The Hill Extra. “These treatments, these drugs, will have passed the safety phase, and why allow a bureaucrat to make a decision for somebody who’s dying.”

Then-Sen. Sam Brownback (R-Kan.) first introduced a version of Right to Try in 2005. At the time, a group — The Abigail Alliance for Better Access to Developmental Drugs — had already begun advocating for easier availability of experimental drugs when there was no option available.

Frank Burroughs, the alliance’s president, daughter Abigail died in 2001 trying to get access to a drug not yet on the market. And while Burroughs acknowledges state and federal laws don’t force companies to provide patients with the drugs, he said they serve a larger purpose. 

“The biggest thing Right to Try laws do on a state level, is they encourage companies to provide the drugs, but the biggest thing really is that it brings this issue to the public,” he told The Hill Extra. “And that’s what legislation from the Hill does, I think, even more so is it brings attention to the problem, and it puts pressure on the FDA.”

The opposition. 

One complication with the laws: drugmakers are not always on board and if they are, that doesn't mean insurers will pay for experimental drugs. Pharmaceutical companies need patients to take part in clinical trials, especially for rare diseases. PhRMA, the drugmaker lobby, has not taken a position on the bill and said in a statement that "any legislation should protect the integrity of clinical trials and the FDA oversight of expanded access to maintain the best interests of patients."

“While well-intentioned," Merck, a pharmaceutical company, wrote in an emailed statement, "current ‘Right-to-Try’ legislation is not in the best interest of patients and is unlikely to help us bring forward innovative, safe and effective medicines to all patients as quickly as possible."

“In addition, we remain supportive of the FDA having an oversight role in the process around expanded access to investigational medicines.”

Johnson’s bill aims to address some of these concerns, such as saying manufacturers and others won't be on the hook for liability issues, but the ultimate decision is up to the drug company. However, a federal bill means companies are allowed to dole out access to these drugs once they’ve passed an initial, small-scale clinical trial, usually conducted in healthy patients.

"The argument is always, 'Well they are dying anyway,' but there are fates worse than death. If you have a prognosis of two years, you want the two years, you don't want to suffer more than you would,” Lisa Kearns, a senior research associate at New York University School of Medicine's division of medical ethics told The Hill Extra. “Many people have pointed out these drugs are not benign, and very little is known about them." 

Others say the effort is misguided.

“To me, it seems that the true purpose of this bill is to undermine the regulatory authority of the FDA in regards to unapproved medications,” Elizabeth Richardson, a managing associate at Duke’s Margolis Center for Health Policy. “I don’t think they will actually make it that much easier for patients to access drugs.”

Several sources pointed to the FDA’s expanded access program, which allows physicians to apply for a seriously ill patient to receive an investigational drug. The FDA has approved more than 99 percent of these requests between 2010 and 2015, according to September congressional testimony from Peter Lurie, FDA’s associate commissioner for public health strategy and analysis. 

The FDA recently updated the form physicians are required to fill out, which some said used to be overly burdensome. Some Right to Try advocates estimated the paperwork took more than 100 hours. This summer, the FDA released its new form that Lurie said takes 45 minutes to fill out.

Rather than pushing for Right To Try laws, some — such as the National Organization for Rare Disorders — see advocating for improvements to the expanded access program as the way to go instead. Paul Melmeyer, NORD’s associate director of public policy, said the group has been in conversations with the Hill on how to do just that.

Yet, for opponents, the optics of the issue aren’t easy.

“Nobody wants to seem like the bad guy who is withholding lifesaving medications from needy patients,” Richardson said.

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