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.
A bill recently passed in the U.S. Senate could dramatically streamline the FDA’s drug-approval process and expand access to new drugs to thousands of people nationwide. The legislation, known as “right to try,” allows patients to request experimental drugs from manufacturers without first obtaining FDA approval. The bill, titled the Trickett Wendler Right to Try Act of 2017, was named after Trickett Wendler, a Wisconsin woman who died in 2015 from amyotrophic lateral sclerosis.
The new proposal would restrict the federal government from blocking patients from getting access to medications that have undergone only preliminary testing in humans after patients have tried all other available treatments and are unable to participate in clinical trials.
Like the right-to-try bills that have already been passed in 37 states, the federal proposal would provide important legal protections for health care providers and drug manufacturers. Providers would be protected from license revocation or sanction based on the recommendation of investigational treatments, and manufacturers would be protected from culpability as well, because patients would take responsibility for using a drug that has not been fully vetted.
According to Politico, drug companies would not be required to give patients medicine under the proposed law, and the bill would remove a provision requiring companies and physicians to obtain FDA’s approval prior to treating patients with experimental medicines. The federal bill is also broader than many state laws, covering some serious but not imminently deadly conditions, such as muscular dystrophy.
Passing a right-to-try bill at the national level addresses one key weakness in state laws: State right-to-try legislation has always faced barriers put in place by current federal drug-approval laws and regulations. The FDA was given jurisdiction over the drug-approval process by Congress, and current FDA regulations make using experimental drugs very difficult. A national right-to-try law would set a new baseline for states, allowing for experimental drugs to be requested and used by patients. States would then be permitted to expand upon existing law as they see fit.
Right to try may already be saving lives. One example promoted by the Goldwater Institute is the positive results found by Dr. Ebrahim Delpassand in Texas, where he has helped nearly 100 patients using the Texas Right to Try law by providing experimental treatment for advanced stage neuroendocrine cancer. Many of Dr. Delpassand’s patients have had their lives extended by a year or more after having been told they had only months to live.
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.
The Goldwater Institute designed its’ 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.”
Right-to-try laws allow patients, with the advice of their doctor, to pursue the medical treatments that would provide them with the best chance of improving and/or extending life while minimizing unnecessary health and legal risks. Congress should follow the lead of the states and implement right-to-try legislation.
The following documents provide additional information about right-to-try laws.
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.