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WASHINGTON — In a wide-ranging State of the Union address Tuesday evening, President Trump endorsed Congress’s efforts to pass a bill allowing access to experimental treatments for patients with terminal conditions.

So-called “right-to-try” legislation was passed by the Senate last fall, but the effort has since stalled in the House. Vice President Mike Pence has endorsed a right-to-try bill. In February Trump gave his support to the legislation but he had not spoken publicly about the issue prior to Tuesday’s address.

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  • Having twenty plus years as an IRB board member I think I have some relevant points. You have a “right to try” currently. It’s called being a drug trial. Yes you may be placed in a placebo arm and not get the drug but that’s how medicine finds out if a drug works. Note our organization and all others organization I am awry of require the trial drugs to be free. That won’t happen with the try it group. It will be expensive! Also the Medicare doesn’t pay for unapproved drugs.

    A real issue with research is you need numbers to reach statistical significance. If people don’t join research via studies their success or failure on drug isn’t counted in finding if the drug works. This is an increasingly important issue as genetics stratifies potential candidates into increasingly small groups.

    One last of many more problems with a “right to try”, sometimes the drugs not only don’t work they kill people. If people are off protocol as terminal patients you won’t always see if drug kills in large numbers masked by the terminal nature of the unmonitored group, has side effects the manufacture can track and mitigate and finally works in a sub group of patients but not population at large.

    No one wants to be the last man to die in a war but simply put this a bad idea with good intentions.

  • We patients applaud the President for supporting this Bill. My wife was the first ” terminally ill ” patient to testify for RTT in Arizona and Colo. You can view ” Lorraines’ Story ” on the Goldwater Institute website. She was dying in 2011 from metastatic breast cancer and we sought access to an experimental drug, Kadcyla. We immediately ran into roadblocks from the FDA . Their hours long ” compassionate ” use paperwork discouraged pt.’s and their doctors from applying. The FDA of Pres. Obamas’ first term was ” flexing it’s regulatory muscles ” (WSJ) with Big Pharma and instead punishing dying pt.’s . They inexplicably denied early approval for this drug in 2010 , shocking our doctors at Farber and MGH. We had to travel at our own expense to get the drug – 16 trips to Wash. DC. It saved her life . Eventually it became one of the top 10 drugs approved by the FDA in 2013 – 3 years later, too late for the 45, 000 Americans who died from that type of bc over those years . Any pt. who dealt with the FDA back then knows how much reform is needed in access. The FDA should only step in when they have a question of safety – not bureaucratically delay every chance at life. After RTT passes our attention will shift to the other Major Player – Big Pharma – they give almost no compassionate use. Putting $ ahead of moral responsibility.

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