Congressional bill facilitates state "Right to Try" laws

Congressional bill facilitates state "Right to Try" laws

A growing number of states are enacting "Right to Try" laws, which attempt to bypass the FDA's new drug approval process by allowing terminally ill patients access to drugs that have successfully completed only a Phase I Clinical Trial. A major stumbling block to access, however, is the fact that states can't trump the FDA's regulatory authority over unapproved drugs, according to the U.S. Constitution. A bill recently introduced in Congress, the "Right to Try Act of 2015" (HR 3012) seeks to remove this hurdle by allowing access to unapproved drugs in states having a Right to Try law.

State right to try laws are a superficially appealing idea. What's wrong, the argument goes, with allowing terminally ill patients access to "potentially life saving" drugs? As it turns out, there's plenty wrong. David Gorski, MD, and I have written several posts over on SBM about the pitfalls of Right to Try. (Other posts by David can be found here, here, and here.) As we've pointed out, among other things:

  • The idea that there is a plethora of life-saving drugs just waiting to get to market is unfounded. The vast majority of drugs that pass Phase I trials do not make it to market, having been knocked out of consideration in the required Phase II and Phase III trials. Phase I trials try to set the appropriate dose; they do not test safety and efficacy. Even drugs that show some evidence of efficacy at this early stage may wind up failing to get approval because of problems discovered later in the clinical trial process.
  • Unapproved drugs are not without risk. Instead of "life saving," they can prove deadly. Patients desperately pursuing drugs under Right to Try laws may forego palliative care and time that could be spent more meaningfully with family and friends.
  • Drug manufacturers are unlikely to give the drugs to patients even when they comply with all statutory requirements, either for liability reasons or a desire to stay out of hot water with the FDA for bypassing the federally required drug approval process. (Manufacturers have no obligation under the laws to provide the requested drugs.) Drug manufacturers are also wary of compromising the clinical trial process. Terminally ill patients may opt for access to an unapproved drug rather than participating in clinical trials.
  • Patient protection is compromised. In some versions, the government is prohibited from taking action to protect patients from harm and physicians are shielded from medical board sanction for approving access in cases that would otherwise warrant discipline. It is easy to envision unscrupulous practitioners setting up "access approval mills." And there is no requirement that the patient's reaction to the drug be reported. We will learn nothing that might advance our knowledge about the drug's potential, or lack thereof.
  • The need for these state laws is questionable. The FDA almost never denies access to an experimental drug under its own Expanded Access program.

HB 3012 removes the FDA as an impediment to what would otherwise be unauthorized access to an unapproved drug. It provides that the federal government "shall not take any action to prohibit the production, manufacture, distribution, prescribing, dispensing, possession, or use of an experimental drug, biological product, or device" as long as it is authorized by state law and the drug, product or device has successfully completed a Phase I investigation and it remains under investigation by the FDA.

It is unsurprising that two of the bills three sponsors are Arizona congressmen. Arizona is home to the Goldwater Institute, a libertarian "think tank" whose only interest in health care policy appears to be pushing these laws thorough state legislatures as a thumb in the FDA's regulatory eye and lambasting the Affordable Care Act. The Robert Wood Johnson Foundation this ain't. The Institute's misleading and factually questionable position paper, along with its Right to Try model legislation, give proponents a slickly produced PR weapon to hand out to legislators. It will fly its lobbyists in to testify before legislative committees as well.

HB 3012 not only upends the FDA's new drug application process, it is also at odds with more thoughtful legislation now before Congress to expedite the FDA's Expanded Access program, which is admittedly slow and cumbersome. (See Sec. 2082 & 2083) The FDA issued draft guidance back in 2013 to expedite the process but never finalized it. This proposed legislation is part of the comprehensive "21st Century Cures Act," a bipartisan effort that passed in the House and has a reasonable chance of passing in the Senate. By any fair estimation, the Act will render state Right to Try laws and HB 3012 unnecessary, while retaining FDA control over the drug approval process. Of course, it won't alleviate the Goldwater Institute's ideological problem with the FDA -- indeed with practically all regulatory authority. Good for patients, and too bad for them.

Points of Interest 08/01/2015
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