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congressional hearing that will be held on Thursday to explore the controversial Right to Try laws is growing tense before the proceedings even get under way.

Among those expected to testify was Dr. Robert Califf, the US Food and Drug Administration commissioner, but scheduling conflicts apparently will prevent him from appearing as planned. But the chairman of the Senate Homeland Security and Governmental Affairs Committee, which will hold the hearing, is upset about the agency official who is being sent to testify, instead.

As far as Senator Ron Johnson (R-Wis.) is concerned, Dr. Peter Lurie, an associate commissioner for public health strategy and analysis, is not qualified to substitute for Califf. After Lurie met with committee staff, Johnson expressed concern Lurie was “unable to answer questions” about Right to Try laws and a host of related issues, according to a letter that Johnson wrote to Califf this past Monday.

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“I continue to believe that you, as the head of the agency, are the most appropriate official to answer questions on behalf of the FDA, especially where questions may touch upon issues outside of Dr. Lurie’s purview,” Johnson continued. “I request that you send a substitute in your place (who) is fully prepared to answer” questions about the laws, which are designed to speed patient access to experimental drugs.

An FDA spokeswoman wrote us to say that Lurie will testify.

The flare-up occurs amid ongoing debate over Right to Try laws, which allow patients to leapfrog a drug-development process that takes years before new drugs become available. The laws, which were passed in 31 states, reflect rising frustration with an FDA program called expanded access, in which people who are seriously ill can obtain a drug under development, even though they aren’t enrolled in a clinical trial.

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Under federal law, if terminally ill patients are not eligible to obtain an experimental drug through a clinical trial, they can apply to the FDA for the expanded access program, which is also known as compassionate use. The agency actually approves the vast majority of these requests, but critics continue to maintain that the federal program is arbitrary and cumbersome.

Whether the Right to Try movement can succeed, though, is uncertain. Legislation has also been introduced in the House and Senate — Johnson, in fact, introduced the Senate bill earlier this year. But there are questions about the extent to which the laws — which have been drafted by the Goldwater Institute, a Libertarian think tank, and introduced in another 15 states — can accomplish their goals.

As we have noted previously, whether patients are protected by Right to Try laws is uncertain since the FDA does not provide oversight of the laws. The agency does not require drug makers to make medicines available. Insurers are not required to pay for treatment, since most states failed to create funding to help patients cover medical expenses. And there are unanswered legal questions about whether state laws trump federal regulations.

The hearing is designed to deflate such concerns. Johnson, however, is angling to get the FDA to explain its procedures for overseeing the expanded access program, as well as address questions about ways it would implement Right to Try laws. For instance, Johnson wants to know if the FDA would enforce federal laws if a doctor or company made unapproved drugs available in a state where a law has passed.

The Right to Try laws, by the way, are part of a broader push for the FDA to make medicines available faster, although the various efforts are engendering significant controversy.

For instance, a bill that passed the House, known as the 21 Century Cures Act, would allow the FDA to approve added uses for a drug without relying on a randomized clinical trial — which some call a gold standard for determining whether a drug can benefit patients and the extent to which there are serious side effects. A comparable effort in the Senate is still being worked on.

There have also been more overt lobbying efforts by advocacy groups, sometimes coordinated with drug makers, to spur the FDA to issue approvals. Two recent examples: the Duchenne muscular dystrophy drug that was approved this week after agency bickering over clinical trial data and the Addyi female libido pill, which involved a publicity campaign that accused the FDA of bias against women.

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  • Maybe Califf didn’t make the hearing cuz he was too busy writing his 12 page memo urging the Sarepta paper to be retracted. This guy is off to a BAD start. FDA commmish usually neither seen nor heard from at least this early on. Something tells me he ain’t long for this job.

  • Our scribe has summed it all up in the final paragraph which has nothing to do with the stated subject. I would say it’s all about lobbying except in my heart I know it’s all about the money. Oh, and how could the FDA challenge a physicians ‘off-label use’ when there is no label? Now getting Big (or even small) pharma to give something away and possibly skew their data is something else again.

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