Would a ‘Right to Try’ Law Save Patient Lives?
Health Letter, November 2014
By Elizabeth Barbehenn, Ph.D.
The idea sounds wonderful: A terminally ill patient who has exhausted all available treatment options would be allowed access to drugs and medical devices not yet approved by the Food and Drug Administration (FDA) — and thus not yet on the market. Although there is now a fairly quick way to accomplish this by going through the FDA, the Goldwater Institute, a libertarian think tank, is pushing for state laws that would seek to do just this without FDA oversight, bypassing FDA regulations intended to ensure that such uses of experimental products have appropriate safety monitoring.[1]
To facilitate the adoption of “Right to Try” laws, the institute has posted on its website a template for a state law that would grant patients the right to access unapproved drugs and medical devices.[2] Under these laws, patients could get drugs and devices as long as Phase 1 trials of the products — the earliest testing of medical products in humans — have been completed and clinical trials of the products are ongoing. A manufacturer would be allowed to provide the drug or device as long as a doctor states that the treatment might save a patient’s life, writes a prescription or recommendation for the drug or device, and obtains the patient’s informed consent in writing.
Three states — Colorado, Louisiana and Missouri — adopted these laws in 2014. The issue also is on the November ballot in Arizona.[3]
FDA drug approval history
In the late 1800s and early 1900s, medicines were sold in the U.S. without undergoing testing to show that they were safe and effective and without labels describing the ingredients. These products often contained morphine, opium, cocaine or high levels of alcohol without that fact being noted on the drug label.[4],[5] They were sold with colorful names and bogus claims of curing a universe of ailments.[6] To counter the safety problems that arose from this practice, Congress passed the Food and Drugs Act of 1906, which forbade the manufacture, sale and transportation across state lines of mislabeled drugs.[7] The 1906 law also established the FDA’s modern regulatory functions.
In 1938, Congress passed the Food, Drug, and Cosmetic Act, establishing for the first time standards for ensuring the safety of drugs before they were allowed to be marketed. This act followed the deaths of 107 people, many of them children, from an antibiotic drug, sulfanilamide, that had been sold as a solution in diethylene glycol, a poisonous compound.[8]
To avoid further disasters, the FDA eventually created a multistep process to test drugs for both safety and efficacy; this includes serial trials with increasing numbers of subjects and longer durations. The testing starts with small trials of usually healthy volunteers (typically 20 to 80 people). These trials, known as Phase 1 trials, represent the first time experimental drugs are given to people. The purpose of such trials is to evaluate a drug’s toxicity and not its effectiveness.
It is after this step that the Right to Try laws would take effect, allowing patients access to inadequately tested drugs and medical devices. Under the state laws, there would be no requirement for physicians to maintain detailed records of the effectiveness or adverse reactions related to use of the experimental products.[9] Patients who might otherwise enter the later clinical trials that are needed to establish safety and effectiveness of an experimental drug or device might opt to instead access the drug through these laws. This would make recruitment for those later trials more difficult and ultimately would slow down the development of important new drugs and devices for many patients.
Ultimately, such state laws would do little to change the regulatory landscape for use of experimental drugs because FDA laws and regulations would trump the state laws.[10]
Access to unapproved drugs
Behind the Goldwater Institute’s push is the idea that a terminally ill patient should be able to obtain an unapproved drug or device if that patient thinks it might be effective.[11] The institute postulates that quicker access to experimental drugs and devices would save thousands of lives in the U.S. However, the Goldwater Institute mistakenly assumes that any drug on which there have been Phase 1 trials has a good probability of being effective, even though only about one out of 10 drugs in Phase 1 trials ever demonstrates efficacy (and drugs that have completed phase 1 testing may have yet-to-be-detected serious toxicity).[12]
The institute also defends Right to Try laws by stating that “patients who have a terminal disease have a fundamental right to attempt to pursue the preservation of their own life by accessing available investigational drugs, biological products, and devices.”[13] The Goldwater Institute maintains this position in spite of the U.S. Supreme Court’s decision to leave in place an earlier U.S. Court of Appeals decision that “patients have no right ‘to a potentially toxic drug with no proven benefit.’”[14]
A number of groups with similar goals are assisting the Goldwater Institute in its efforts. One of these groups is the Abigail Alliance for Better Access to Developmental Drugs,[15] which, along with most of its allies, endorses a federal version of the state Right to Try laws dubbed the Compassionate Access Act. This federal act would have the same goals as those of the Goldwater Institute, that is, to allow another path by which patients could obtain drugs lacking FDA approval. The act states, “Seriously ill patients have a right to take actions to preserve their life by accessing available investigational drugs, biological products, and devices.”[16] The Abigail Alliance itself suggests that members of the public write their congressional representatives urging them to support such a law.
FDA allows access to unapproved drugs and devices [17]
Actually, the FDA has in place a relatively straightforward method for physicians and patients to obtain access to unapproved drugs. In a non-emergency, a doctor can complete a simple form to do so. However, in an emergency, physicians can forgo the form and both apply and receive authorization by telephone, with documentation provided later.[18] In both cases, oversight by the FDA and by local institutional review boards provides important safeguards for protecting patients accessing experimental products. Similar mechanisms exist for access to experimental medical devices.
In a thoughtful commentary published online in JAMA Internal Medicine on September 29, two law professors at Stanford University nicely summarized the fundamental problem with these Right to Try laws:[19]
The problem is that all these efforts are unlikely to actually help the patients with life-threatening diseases. Indeed, these laws may be harmful if they draw attention and resources away from efforts to develop effective treatments, engender confusion about the FDA pathway for compassionate use of medications, or create false hopes for terminally ill patients.
Still, the Goldwater Institute continues to push its dangerous proposal, which will doubtless cause more harm by weakening existing consumer protections against untested, possibly harmful drugs.
References
[1] Corieri C. Everyone deserves the right to try. Goldwater Institute. http://goldwaterinstitute.org/article/everyone-deserves-right-try-empowering-terminally-ill-take-control-their-treatment. Accessed October 18, 2014.
[2] Goldwater Institute. Proposed Statutory Language. February 11, 2014. https://www.citizen.org/sites/default/files/proposed_statutory_language.pdf. Accessed October 18, 2014.
[3] Byrd ML. Third state passes right to try legislation. http://freebeacon.com/issues/third-state-passes-right-to-try-legislation/. Accessed October 18, 2014.
[4] Hagley Museum and Library. History of Patent Medicine. http://www.hagley.org/online_exhibits/patentmed/history/history.html. Accessed October 18, 2014.
[5] Alcohol in patent medicines. http://www.ncbi.nlm.nih.gov/pmc/articles/PMC1649586/pdf/calstatejmed00236-0012b.pdf. Accessed October 18, 2014.
[6] Hagley Museum and Library. History of Patent Medicine. http://www.hagley.org/online_exhibits/patentmed/history/history.html. Accessed October 18, 2014.
[7] Food and Drug Administration. Federal Food and Drugs Act of 1906. Updated May 20, 2009. http://www.fda.gov/regulatoryinformation/legislation/ucm148690.htm. Accessed October 18, 2014.
[8] Food and Drug Administration. A History of the FDA and Drug Regulation in the United States. http://www.fda.gov/downloads/Drugs/ResourcesForYou/Consumers/BuyingUsingMedicineSafely/UnderstandingOver-the-CounterMedicines/ucm093550.pdf. Accessed October 18, 2014.
[9] Zettler PJ, Greely HT. The strange allure of state “right-to-try laws. JAMA Internal Medicine. Published online September 29, 2014.
[11] Corieri C. Everyone deserves the right to try. Goldwater Institute. http://goldwaterinstitute.org/article/everyone-deserves-right-try-empowering-terminally-ill-take-control-their-treatment. Accessed October 18, 2014.
[12] Servick K. ‘Right to Try’ laws bypass FDA for last-ditch treatments. Science. June 20, 2014;344:1329.
[13] Goldwater Institute. Proposed Statutory Language. February 11, 2014. https://www.citizen.org/sites/default/files/proposed_statutory_language.pdf. Accessed October 18, 2014.
[14] Brower V. Food and Drug Administration responds to pressure for expanded drug access. JNCI. 2014;106(6): dju171 doi: 10.1093/jnci/dju171.
[15] The Abigail Alliance. Our Allies. http://www.abigail-alliance.org/allies.php. Accessed October 18, 2014.
[16] The Compassionate Use Act of 2010. https://www.govtrack.us/congress/bills/111/hr4732/text. Accessed October 18, 2014.
[17] Food and Drug Administration. Devices. Emergency use. http://www.fda.gov/medicaldevices/deviceregulationandguidance/howtomarketyourdevice/investigationaldeviceexemptionide/ucm051345.htm#compassionateuse. Accessed October 18, 2014.
[18] Food and Drug Administration. Physician request for an individual patient IND under expanded access for non-emergency or emergency use. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/ucm107434.htm. Accessed October 18, 2014.
[19] Zettler PJ, Greely HT. The strange allure of state “right-to-try” laws. JAMA Internal Medicine. Published online September 29, 2014.