Access to Trial Meds Needs Market Incentive, Conservatives Say

Access to Trial Meds Needs Market Incentive, Conservatives Say

By Jeannie Baumann and Alex Ruoff | July 16, 2019 05:41AM ET | Bloomberg Law

Lawmakers and conservative advocates who fought to make it easier for Americans to use experimental medicines are floating a new twist: permitting drugmakers to sell unapproved drugs at a profit.

The “free to choose” idea is in early stages of conversation, largely among conservatives. It would shortcut clinical trials, which are the costliest of the decade-long drug development process and can run into the billions.

The free-to-choose push seeks to build upon the Right to Try Act, which President Donald Trump signed into law last year. It allows patients with life-threatening diseases to petition a drug company for an experimental product if they can’t get into a clinical trial, and they can do so without having to go through the FDA.

“We are supportive of Right to Try,” Christina Herrin, director of Free to Choose Medicine for the Heartland Institute, said in an interview. “But we think it’s missing a market incentive.”

The Heartland Institute, the Chicago-based think tank behind conservative messaging that downplays the impacts of climate change, is also a major force behind the free-to-choose proposal. The group says the change could clear up a bureaucratic bottleneck that stifles access to innovative treatments and limits patients’ health-care options. Opponents say it would expose patients to potentially dangerous medicines and that options for gaining access to experimental drugs already exist.

The free-to-choose idea would set up a parallel system to the FDA’s current drug approval process. Pharmaceutical companies could market drugs that have passed smaller clinical trials that test for toxicity and efficacy but not the larger studies that determine new drug approvals. It would also expand the eligible conditions to include debilitating diseases such as ALS and Alzheimer’s.

“Getting the right treatment to the right patient and at the right time is the goal,” said Naomi Lopez Bauman, director of health-care policy at the Goldwater Institute in Phoenix. “This is an important issue and the Free to Choose Medicine proposal deserves serious consideration.”

It’s also gotten the attention of members of the ultra-conservative Freedom Caucus in the U.S. House. A dozen Republicans, including several Freedom Caucus members, last month asked acting FDA Commissioner Ned Sharpless to expand the Right to Try track to diseases beyond HIV/AIDS.

Rep. Thomas Massie (R-Ky.), one of the letter’s signatories, said FDA officials have the discretion to go further when it comes to experimental medicines but aren’t using it. He wants to know why.

“In the instances where they’ve done it, it’s been a very positive thing, so let’s do more of it,” Massie told Bloomberg Law. “We’re asking them to use the discretion they have. And if they don’t, we can pass legislation—I’m all for that.”

A spokesperson for the FDA said the agency will review the lawmakers’ letter and respond directly to them. It may be a tough sell because FDA officials have told Congress in other contexts that the agency isn’t holding up the drug development process. It has incorporated faster reviews and broken records for the number of drugs approved.

Making a Profit

Under current policies, manufacturers can recoup costs they’ve incurred for providing experimental medicines, but they aren’t allowed to profit from the transactions.

Free to choose would allow marketing of early-stage drugs. It also would create a database to track genetic data, relevant biomarkers, and clinical treatment results from the use of free-to-choose drugs. The data would allow drug sponsors, researchers, physicians, and patients to judge what treatment options might be best for them, the Heartland Institute says.

“The current system is overly paternalistic and it violates patient’s rights of self-medication,” Jessica Flanigan, a medical ethicist at the University of Richmond, told Bloomberg Law. Free to choose “is a step in the right direction towards respecting patient’s rights of self medication.”

The FDA already has a program called “parallel track,” which it created at the height of the AIDS epidemic to allow access to experimental HIV/AIDS drugs. Since that time, the FDA has established other programs to allow the use of an unapproved medicine, including expanded access and emergency uses.

Raising Alarms

Gregg Gonsalves, a longtime HIV/AIDS activist who cofounded a nonprofit that pushed for earlier and broader access to experimental therapies in the 1990s, raised alarms about the freedom-to-choose proposals.

Gonsalves, a Yale University epidemiologist, cautioned against weakening the FDA’s oversight of investigational drugs. He said such a move could have tragic consequences. “In the case of pharmaceutical drugs, this also means dead patients too,” he said.

Tying a marketing go-ahead to Phase III clinical evidence is the agency’s “most important tool in getting industry to do the studies we need to know if drugs work,” he said.

Investigational medicines typically must pass three clinical trial phases to demonstrate they are safe and effective enough to market.

Phase I uses healthy volunteers to determine dosage and toxicity. Phase II studies are typically smaller ones that assess some level of effectiveness. Phase III studies are bigger trials to gather enough information about safety and effectiveness to warrant an FDA approval that also gives drugmakers the authority to market the medicine. Phase III trials also provide data that is used for labels physicians rely on.

Making Informed Decisions

Without Phase III testing, doctors and patients are missing important, high-quality data that they need to make an informed decision about treatment, University of Pennsylvania bioethicist Holly Fernandez Lynch told Bloomberg Law.

Nearly 70 percent of medicines in development fail in Phase III, which means patients could end up taking unnecessary risks, especially for conditions that aren’t life threatening and for which other treatment options exist, she said.

Flanigan acknowledged that objections to free to choose may arise “on the grounds that it would allow people to access drugs that have not fully completed efficacy testing. But people can already access drugs that have not been tested for efficacy when they use drugs off-label.”

Allowing biotech companies to charge for experimental drugs under free to choose would allow smaller drug companies to develop new drugs, the Heartland Institute said.

To contact the reporters on this story: Jeannie Baumann in Washington at jbaumann@bloomberglaw.com; Alex Ruoff in Washington at aruoff@bgov.com

To contact the editors responsible for this story: Fawn Johnson at fjohnson@bloomberglaw.com; Brent Bierman at bbierman@bloomberglaw.com