Iowa is considering becoming the 34th state to allow terminally ill patients to obtain experimental drugs without getting federal approval, a policy commonly called “right to try” (RTT).
The current process for gaining approval for new medications is long and arduous. Patients seeking to use experimental drugs can either wait for the treatments to receive Food and Drug Administration (FDA) approval or go through a lengthy and difficult FDA exemption process, which can take several months. The exemption process requires physicians to complete paperwork that takes almost 100 hours to complete. Even after this process, the decision lies with FDA.
Prior to passage of the 1962 Kefauver-Harris Amendment, which added new requirements for proof of efficacy and safety requirements for approval of new drugs, the average time from the filing of a new drug application to approval was seven months. It now takes an average of 12 years for a drug to travel from the research lab to the patient, according to the California Biomedical Research Association. This is due in large part to delays by FDA. In order to speed up the approval process and improve access to potentially life-saving drugs for extremely sick people, several states have introduced new laws that would allow terminally ill patients to obtain experimental drugs without getting federal approval.
A proposal in Iowa would bypass FDA’s expanded-access program and allow patients to request experimental drugs from manufacturers without first obtaining FDA approval. The proposal would provide legal protection for health care providers and drug manufacturers. Providers would be protected from license revocation or sanction based on the recommendation of investigational treatments. Manufacturers would be protected from culpability as well, as patients would take responsibility for using a drug that has not been fully vetted. It also blocks the state from interfering with such experimental treatments.
The Goldwater Institute designed a right-to-try model to address several criticisms of federal policy on experimental drug use. Goldwater’s model pertains only to medications that passed basic safety testing (Phase I), in order to observe reasonable public safety standards for untested drugs. Under the model, access to experimental drugs is limited to use by terminal patients who have exhausted other available treatments. “Americans shouldn’t have to ask the government for permission to try to save their own lives,” said Darcy Olsen, president of the Goldwater Institute, to the Tenth Amendment Center. “They should be able to work with their doctors directly to decide what potentially life-saving treatments they are willing to try. This is exactly what Right To Try does.”
Additionally, the experimental medications would be made available only if the manufacturer chooses to make them available, and the request would require a doctor to diagnose a terminal disease and declare the drug would provide the patient with his or her best chance of survival. Patients would also have to provide informed consent, limiting legal exposure for the drug’s manufacturer.
One challenge state right-to-try laws will have to overcome is barriers put in place by current federal drug-approval laws and regulations. FDA was given jurisdiction over the drug-approval process by Congress and current regulations put into place by FDA makes using experimental drugs very difficult.
The Tenth Amendment Center argues right-to-try laws such as the one currently proposed in Iowa should serve as a model for states to use to combat federal laws that overstep constitutional limits. “Although these laws only address one small aspect of FDA regulation, they provide a clear model that demonstrates how to nullify federal statutes that violate the Constitution. The strategy narrows the influence of nullification to limited aspects of the law itself, which has proven to be very effective.”
The following articles discuss right to try in general and the various RTT proposals now being considered.
Iowa Right-to-Try Act Would Reject Some FDA Restrictions on Terminal Patients
This blog post from the Tenth Amendment Center outlines Iowa’s effort to introduce right-to-try legislation and how it could both help patients and create a model for limiting federal power. “Currently, 33 states have Right to Try laws on the books. Although these laws only address one small aspect of FDA regulation, they provide a clear model that demonstrates how to nullify federal statutes that violate the Constitution. The strategy narrows the influence of nullification to limited aspects of the law itself, which has proven to be very effective.”
Dead on Arrival: Federal ‘Compassionate Use’ Leaves Little Hope for Dying Patients
In this report from the Goldwater Institute, Mark Flatten discusses right-to-try laws and argues they could serve as a possible solution to overcoming the U.S. Food and Drug Administration’s arduous restrictions on partially tested drugs, thereby helping terminally ill patients.
The Real Defense of Right to Try
David S. D’Amato writes in Health Care News about the ongoing and contentious right-to-try debate and argues in favor of reform. “Sick people should not have to supplicate to government bureaucrats to assume certain risks in the desperate attempt to save themselves; such intimately personal health decisions are of no concern to the meddlers in government agencies, to anyone but the patient, his doctors, and his family,” wrote D’Amato.
The Right to Try and the Future of the FDA in the Age of Personalized Medicine
Adam D. Thierer of the Mercatus Center argues right-to-try laws have developed and expanded in recent years in part because technological innovation decentralizes and democratizes medical decisions. “This expansion does not mean that all FDA regulation will fade away, but it will necessitate a move away from the agency’s command-and-control methods of the past and toward a new focus on patient empowerment through enhanced choice, improved risk education, and clearer consent mechanism,” wrote Thierer.
‘Right to Try’ Experimental Prescription Drugs State Laws and Legislation for 2014 & 2015
The National Conference of State Legislatures outlines the current state of right-to-try laws and new right-to-try proposals across the country.
Dead on Arrival: Federal ‘Compassionate Use’ Leaves Little Hope for Dying Patients
The Goldwater Institute’s report on right to try laws as a possible solution to overcoming the U.S. Food and Drug Administration’s restrictions upon partially tested drugs that could help terminally ill patients.
Federal ‘Right to Try’ Act Would Help States Bypass FDA to Treat Terminally Ill
Josh Thomas of Health Care News examines new legislation introduced by U.S. Sen. Ron Johnson (R-WI) that would prevent the federal government from superseding state right-to-try laws, making it easier for terminally ill patients in certain states to obtain experimental treatments and devices.
Everyone Deserves the Right to Try: Empowering the Terminally Ill to Take Control of Their Treatment
Christina Corieri outlines the Goldwater Institute’s 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. Goldwater’s initiative would allow terminal patients access to investigational drugs that have completed basic safety testing, thereby dramatically reducing paperwork, wait times, bureaucracy, and, most importantly, potentially saving lives.
More Choices, Better Health
More Choices, Better Health: Free to Choose Experimental Drugs shows the U.S. Food and Drug Administration’s lengthy drug-approval process causes needless pain and suffering. The booklet presents an alternate solution that gives informed patients and their physicians easier access to experimental drugs.
Is the FDA Safe and Effective?
Drawing on the existing body of academic research, Daniel B. Klein and Alexander Tabarrok evaluate the costs and benefits of FDA policy. Their website also presents 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 FDA, and a reference section with many links.
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, The Heartland Institute’s website, and PolicyBot, Heartland’s free online research database.
If you have any questions about this issue or The Heartland Institute’s website, contact John Nothdurft, The Heartland Institute’s government relations director, at [email protected] or 312/377-4000.