Who Benefits From The “Right to Try” Experimental Medicines?

doctor-mom-and-sonThe Goldwater Institute has had great success getting states to pass “Right to Try” laws. Right to Try allows a desperately sick patient to take an experimental new medicine before the FDA has approved it.

Since I last wrote about this policy in November 2014, 31 states have passed Right to Try. Further, U.S. Senator Ron Johnson (R-WI) has tried to get a federal Right to Try law through the U.S. Senate.

However, there has been push-back. According to Allison Bateman-House of NYU Langone Medical Center, “there is no confirmed instance of anyone getting a drug through Right to Try.” Jonathan Friedlaender, a survivor of advanced metastatic melanoma, has written a compelling essay in Health Affairs, which concludes Johnson’s proposed federal law would not improve access to experimental medicines.

The problem has two parts:

First, research-based drug companies do not distribute drugs under Right to Try because the drugs are usually appropriate for small populations of patients. Every patient who receives an experimental dose outside a randomized-control trial (RCT) is a patient who cannot be enrolled in an RCT. RCTs are very expensive and it is very hard to recruit enough patients. The smaller the pool, the more loath the drug company is to shrink it by distributing drug early.

Second, even if a drug company uses the current “compassionate use” exemption (which is stricter than Right to Try, which is an attempt to go around its limits), it cannot charge more than the drug’s manufacturing cost. Manufacturing costs are a very small proportion of a drug’s total cost, which is mostly research & development. A company that dispenses drugs for free or charges only manufacturing costs enters a political minefield after the drug is approved by the FDA and it charges the market price.

Right to Try might not be having the expected impact. However, that is no reason to give up. The 21st Century Cures Act is a comprehensive FDA reform bill which was passed overwhelmingly by the House of Representatives and is currently in the U.S. Senate. It would allow drug makers to use more sophisticated statistical methods to get FDA approval. These methods would let them put their drugs on the market sooner and reduce the barriers to access imposed by the current need for RCTs.

Comments (5)

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  1. Devon Herrick says:

    I favor Right to Try, but I also agree that it’s probably little more than a symbolic gesture to nudge new legislation along. The notion that people allowed to try a drug not yet approved cannot have their data considered along with the clinical trial results is an example of why FDA reform is needed.

  2. Ron Greiner says:

    Hillary, the non-profits and the media won’t talk about Obamacare before the election but Bill Clinton didn’t get the message.

    At a campaign stop in Flint, Mich., Bill Clinton blamed Obamacare’s exchange system in particular for the chaos.
    “It doesn’t make any sense. The insurance model doesn’t work here,” he said. “So you’ve got this crazy system where all of a sudden 25 million more people have healthcare and then the people who are out there busting it, sometimes 60 hours a week, wind up with their premiums doubled and their coverage cut in half.”

    Bill Clinton did further assert, “It’s the craziest thing in the world.”

    Clinton even pointed out that small business owners and the middle class are the hardest hit by Obamacare.

    “The current system works fine if you are an employee at a non-profit, if you’re eligible for Medicaid, if you’re a lower-income working person; if you’re already on Medicare, or if you get enough subsidies on a modest income that you can afford your health care,” he added. “But the people that are getting killed in this deal are small business people and individuals who make just a little too much to get any of these subsidies.”

    Bill Clinton should talk about pet insurance or Rx price gouging and never mention Obamacare this close to the election. But these Clintons are not smart people.

    • Ron Greiner says:

      Kellyanne Conway, TRUMP’s campaign manager, sarcastically praised Clinton on “Morning Joe” for his comments that the Affordable Care Act is an effective law yet “needs to be fixed.”

      “We’ve got now President Bill Clinton is our best surrogate,” Conway said, smiling. “We’re thinking of having him in the spin room with us.”

      Even Bill knows Obamacare sucks and will vote against Hillary.