The current process for pharmaceutical firms seeking Food and Drug Administration (FDA) approval for new medications is long and arduous. And patients seeking to use experimental drugs before FDA approval must go through an exemption process that can take several months, requiring physicians to file paperwork that takes almost 100 hours to complete. Even after this lengthy process, the decision to grant an exemption for the patient’s use of the experimental drug lies solely with the FDA.
To speed up this process and improve the access of terminally ill patients to potentially beneficial drugs, several states have introduced “Right to Try” laws that would allow patients to obtain experimental drugs without getting federal approval. Three states—Colorado, Louisiana, and Missouri—are on track to become the first to pass such laws. Arizona voters will consider a similar measure in November.
The Right to Try model was designed by the Goldwater Institute to address several concerns about patients’ use of experimental drugs. First, the model allows access only to medications that have passed basic safety testing (FDA Phase I). Second, access is limited to use by terminally ill patients who have exhausted other available treatments. Finally, a medication is made available only if the company manufacturing it chooses to do so. A patient’s request for access to an experimental drug would require a doctor to diagnose a terminal disease and declare the drug represents the patient’s best chance at survival with the patient providing informed consent, limiting the legal exposure of the manufacturer of the drug.
Critics of Right to Try claim providing experimental drugs to terminally ill patients creates a false hope. Supporters say any hope is better than the alternative of no hope, which is inevitable when no treatments are made available for terminal patients. Milton Friedman once noted the FDA drug approval process, done in the name of safety, has harmful consequences for both the health of the public and the economy: “The FDA has done enormous harm to the health of the American public by greatly increasing the costs of pharmaceutical research, thereby reducing the supply of new and effective drugs, and by delaying the approval of such drugs as survive the tortuous FDA process.”
According to the California Biomedical Research Association (CBRA), it takes an average of 12 years for a drug to travel from the research lab through the FDA to the patient. In addition, the FDA allows only five in 5,000, or .1 percent, of the drugs that begin preclinical testing ever to make it to human testing, and of those five, only one is ultimately approved for human use. Prior to passage of the 1962 Kefauver Harris Amendment, which added new requirements for proof of efficacy in addition to safety for approval of new drugs, the average time from the filing of an investigational new drug application to approval was seven months.
Right to Try laws allow patients, with the advice of their doctors, to choose what treatments to try. These laws take reasonable steps to ensure the drugs are reasonably safe and that drug manufacturers and patients work to manage legal risk. Legislators in other states should consider passing such laws.
The following documents provide additional information about Right to Try laws.
Everyone Deserves the Right to Try: Empowering the Terminally Ill to Take Control of Their Treatment
Christina Corieri of the Goldwater Institute outlines the Right to Try initiative. Corieri argues states should enact Right to Try measures to protect the fundamental right of people to try to save their own lives. Designed by the Goldwater Institute, the initiative would allow terminal patients access to experimental drugs that have completed basic safety testing. The measure would dramatically reduce paperwork, wait times, and bureaucracy. Most importantly, it may save lives.
Free to Choose Medicine
The core idea of Free to Choose Medicine is to give people the freedom to make informed decisions about whether to use not-yet-FDA-approved therapeutic drugs—new drugs that have successfully passed safety trials, generated preliminary efficacy data, and may offer us the opportunity to improve individuals’ health and save lives.
Making Drugs Safe and Available without the FDA
Noel D. Campbell of the National Center for Policy Analysis argues Americans should be free to choose what level of risk to accept when selecting drugs and health treatments. Individuals would be able to relieve pain without fear for their safety. “Thus the best reform is the most comprehensive reform. When the 105th Congress undertakes FDA reform, the reform should be profound. Congress should abolish the FDA and allow private organizations to certify drugs and medical devices,” Campbell writes.
A Clinical Trial for the FDA’s Clinical Trial Process
Bartley Madden argues FDA control over drugs and devices has large and often overlooked costs that almost certainly exceed the benefits: “We believe that the FDA regulation of the medical industry has suppressed and delayed new drugs and devices, and has increased costs, with a net result of more morbidity and mortality. A large body of academic research has investigated the FDA and with unusual consensus has reached the same conclusion.”
Is the FDA Safe and Effective?
Drawing on an existing body of academic research, Daniel B. Klein and Alexander Tabarrok evaluate the costs and benefits of FDA policy. They also present a detailed history of the FDA, a review of the major plans for FDA reform, a glossary of terms, a collection of quotes from economists who have studied the FDA, and a reference section with many webbed links.
The ‘Dallas Buyers Club’ Bill
Eleanor Clift of The Daily Beast discusses Right to Try efforts, how they first emerged, and states’ consideration of legislation to allow terminal patients greater access to experimental drugs.
‘Right to Try’: States Move to Expand Access to Experimental Drugs
NBC News reports on the Colorado Right to Try bill and how it passed through the Colorado legislature: “The ‘Right To Try’ law allows terminally ill patients to obtain experimental drugs without getting federal approval. It’s a proposal being advanced in several states by patient advocates who are frustrated by the yearslong federal approval process for experimental drugs in the pipeline.”
Every Patient Deserves a “Right to Try”
The federal government is forcing terminally ill patients to suffer while the FDA drags out the drug approval process. Some states are fighting back. Writing in Townhall Magazine, Mary Katharine Ham discusses one mother’s battle with the FDA to save her terminally ill sons.
The Right to Try
Mary Lou Byrd of the Free Beacon reports on the Arizona Right to Try bill, which would allow the terminally ill to use experimental drugs not yet approved for use by the Federal Drug Administration.
Nothing in this Research & Commentary is intended to influence the passage of legislation, and it does not necessarily represent the views of The Heartland Institute. For further information on this subject, visit Health Care News at http://news.heartland.org/health, The Heartland Institute’s website at http://heartland.org, and PolicyBot, Heartland’s free online research database at www.policybot.org.
If you have any questions about this issue or the Heartland Institute Web site, contact Heartland Institute Government Relations Director John Nothdurft at [email protected] or 312/377-4000.