Utah is one of several states expected to consider legislation allowing terminally ill patients to obtain experimental drugs without getting federal approval, known as “Right to Try.” The current process to gain approval for new medications is long and arduous, with patients seeking to use experimental drugs either waiting years for the treatments to receive FDA approval or facing a lengthy and difficult FDA exemption process, which may or may not be approved.
The Goldwater Institute designed its Right to Try model to address several criticisms of federal policy on experimental drug use. First, the model pertains only to medications that pass basic safety testing (Phase I) in order to observe reasonable public safety standards for untested drugs. Second, access to these drugs is limited to use by terminal patients who have exhausted other available treatments. Finally, the experimental medications are made available only if the manufacturer chooses to make them available. The request 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 drug’s manufacturer.
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 an investigational new drug application (IND) to approval was 7 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, due in large part to delays by the FDA, the U.S. government agency responsible for drug approval. The FDA allows only one in 1,000, or .1 percent, of the drugs beginning preclinical testing to advance to human testing. Only 20 percent of the drugs reaching the preclinical phase complete testing and are approved for human use.
Colorado was the first state to implement a Right to Try law in May 2014. The Colorado law allows patients to work with pharmaceutical companies to access experimental drugs without forcing any party to participate. Insurance companies are not required to pay for the experimental drugs, and drug makers are not required to provide them. The bill also provides protections for health care providers, while also requiring them to notify patients the treatments may incur costs.
Congress is considering a similar bill, the Compassionate Freedom of Choice Act of 2014. According to Congress.gov, the bill would amend the Federal Food, Drug, and Cosmetic Act to declare “nothing in it or in the Public Health Service Act shall prevent or restrict, and the Food and Drug Administration (FDA) shall not implement or enforce any law to prevent or restrict, the manufacture, importation, distribution, or sale of investigational drugs or devices for terminally ill patients.”
One challenge state Right to Try laws will have to overcome is the current federal drug approval laws; the FDA has jurisdiction over the drug approval process through Congress’ authority to regulate interstate commerce. Although states cannot preempt this process directly, they can send a strong message to Congress and the FDA they want and need more choice and access to new experimental drugs.
Right to Try laws would give patients the ability, with the advice of their doctors, to choose what treatments work for them. These laws take appropriate steps to ensure the drugs are reasonably safe and both drug manufacturers and patients take necessary steps to manage legal risk. Utah legislators should follow Colorado’s lead and seriously consider Right to Try legislation.
The following articles discuss the Right to Try, how it would work, and the various proposals now being considered.
Everyone Deserves the Right to Try: Empowering the Terminally Ill to Take Control of Their Treatment
http://heartland.org/policy-documents/everyone-deserves-right-try-empowering-terminally-ill-take-control-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 Right to Try 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
http://heartland.org/policy-documents/more-choices-better-health?artId=21194
More Choices, Better Health: Free to Choose Experimental Drugs shows the U.S. Food & Drug Administration’s lengthy drug approval process causes needless pain and suffering. The booklet presents an alternate solution giving informed patients and their physicians easier access to experimental drugs.
Making Drugs Safe and Available without the FDA
http://heartland.org/policy-documents/making-drugs-safe-and-available-without-fda
Noel D. Campbell of the National Center for Policy Analysis argues Americans should be free to choose when selecting drugs and health care treatments. Americans should be able to relieve pain without fear of our safety; “[t]hus 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,” he wrote.
A Clinical Trial for the FDA’s Clinical Trial Process
http://heartland.org/policy-documents/clinical-trial-fdas-clinical-trial-process
Bartley Madden argues FDA control over drugs and medical 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?
http://www.fdareview.org/
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 the FDA, and a reference section with many links.
The ‘Dallas Buyers Club’ Bill
http://www.thedailybeast.com/articles/2014/03/04/the-dallas-buyers-club-bill.html
Writing in The Daily Beast, Eleanor Clift discusses Right to Try efforts, explaining how they first emerged and how several states are now considering legislation to allow terminal patients greater access to experimental drugs.
“Right to Try”: States Move to Expand Access to Experimental Drugs
http://www.nbcnews.com/health/health-news/right-try-states-move-expand-access-experimental-drugs-n108316
NBC News covers the Colorado Right to Try bill and its passage through the state’s 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 years-long federal approval process for experimental drugs in the pipeline.”
Every Patient Deserves a “Right to Try”
http://townhall.com/tipsheet/townhallmagazine/2014/04/10/every-patient-deserves-a-right-to-try-n1822217
The federal government is allowing terminally ill patients to suffer while the FDA drags out the approval process. Some states are fighting back. In the April issue of Townhall Magazine, where this article originally appeared, HotAir’s Mary Katharine Ham discusses one mother’s battle with the FDA to save her terminally ill sons.
The Right to Try
http://freebeacon.com/issues/the-right-to-try/
Mary Lou Byrd of the Free Beacon discusses the Arizona Right to Try bill, which would allow the terminally ill to use experimental drugs not yet approved by the Food and Drug Administration. “Arizona lawmakers passed the bill out of committee and in the next three weeks both chambers of the legislature could vote on it. It would then need to be approved by voters in November,” she wrote.
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.
The Heartland Institute can send an expert to your state to testify or brief your caucus; host an event in your state; or send you further information on a topic. Please don’t hesitate to contact us if we can be of assistance! If you have any questions or comments, contact Nathan Makla, Heartland’s state government relations manager, at [email protected] or 312/377-4000.
Matthew Glans joined the staff of The Heartland Institute in November 2007 as legislative specialist for insurance and finance.
