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.
- 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
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