This post is a part of our Bioethics in the News series. For more information, click here.
By Jennifer Carter-Johnson, PhD, JD
Right to Try Laws Generally
Picture a mother with breast cancer hoping to see her daughters get married. Weep with parents as their nine year old son dies in their arms. Rally behind the newlywed couple fighting to experience just one anniversary. These are the people targeted by Right To Try (RTT) laws recently sweeping more than twenty states across the nation.
Based on model legislation put forth by the Goldwater Institute, RTT laws seek to allow terminally ill patients who have exhausted all approved drug therapies to access drugs that have not been approved for sale by the Food and Drug Administration (FDA). Proponents argue that the RTT laws empower terminally ill patients to attempt to try to save their own lives by allowing those patients access to drug candidates that have passed at least Phase 1 of FDA clinical trials.
FDA Clinical Trials Regulation
To understand why these laws are so troubling, it is important to put them in the context of the FDA regulation process. No chemical substance is completely safe – even drinking too much water can kill you. Cancer drugs are on the other end of the safety spectrum from water. Cancer drugs generally work because they kill cancer cells at a faster rate than they kill normal cells. The job of the FDA is to evaluate new drug candidates and approve one for sale when the proposed benefits of a drug outweigh its harms.
In order to make that determination, the FDA requires numerous studies culminating in a series of clinical trials using human patients. These clinical trials are divided into three basic phases with increasing numbers of patients enrolled in each. In a Phase 1 clinical trial, as few as twenty healthy patients are enrolled for a few months to determine a relatively safe dosage of the drug for use during further trials. Approximately seventy percent of drug candidates graduate from Phase 1 to Phase 2. It is not until Phase 2 that drug candidates begin to be assessed for effectiveness and side effects on a few hundred patients. Only about one third of the Phase 2 drug candidates show enough balance between effectiveness and side effects to move into Phase 3. Phase 3 trials involve large scale patient enrollment over several years to assess the true benefits and harms of a drug candidate. Of the drug candidates that enter Phase 3, only about twenty five percent are approved for marketing.
The drug approval pipeline is designed to screen large numbers of drugs to find the few that will be helpful in a given disease context. Of one hundred potential drug candidates, seventy will likely enter Phase 2; twenty three will pass into Phase 3 and about six will be approved for sale.
Impact of Right to Try on Patients and Society
Because most drug candidates enter Phase 2, a proposed cancer drug that kills normal cells too fast or causes side effects that kill a patient more quickly than the cancer will likely progress through at least Phase 2 trials before being dropped from the pipeline. That drug candidate would be available for use under the RTT Laws. In fact, a patient is far more likely to receive a drug candidate that will either do nothing to help the condition and/or have significant adverse side effects beyond the symptoms caused by the disease. Thus, patients who ask for drug candidates that have merely completed a Phase 1 trial very likely are banking on a false hope and may even have their lifespan decreased or their quality of life diminished.
Other problems plague the patients invoking RTT laws. RTT laws allow companies to charge for the drugs, and small scale batches of drugs created for clinical trials are often highly expensive. RTT laws do not require insurance to cover those expenses, and many explicitly allow drug companies to go after the patient’s estate to recover costs. Thus, RTT laws are likely to primarily benefit those with resources to pay many thousands of dollars out of pocket. The poor will have no more access to drugs than before the law, and those with savings, life insurance policies, or retirement accounts will be tempted to drain those accounts for a false hope – likely leaving loved ones bereft of any financial buffer that had originally been planned.
Additionally, if a patient using an experimental drug has adverse side effects, he will further deplete his savings paying for care. Most RTT laws exempt insurance companies from paying for medical problems that arise while on an experimental drug. Public hospitals who cannot turn away those without insurance coverage may lead to a drain on the taxpayer. Because these laws protect both the drug company and the prescribing doctor from liability, the monetary and physical costs of any severe side effects will be borne by the patient alone.
Finally, there is the danger of the birth of a new snake oil market. Before the FDA regulated drugs for safety and efficacy, salesmen took advantage of customers to sell all sorts of tonics. We see similar problems in the practically unregulated supplement market today. The RTT laws hold the potential to underlie a new industry of drug candidates that have gone through Phase 1 to gain a sheen of FDA credence but that hold little chance of final approval.
More Reasonable Approach?
The access to drug candidates that have completed no more than a Phase 1 clinical trial is the worst form of false hope that threatens to cost those most vulnerable everything. In a nod to autonomy and the power of hope, perhaps a more reasonable approach would be to allow patients the right to try drug candidates that have entered into Phase 3 clinical trials. While many of the problems with cost, insurance and liability would remain, perhaps the most toxic and least effective drug candidates may have been screened from the pool of drug candidates.
Jennifer Carter-Johnson, PhD, JD, is an Associate Professor of Law in the College of Law at Michigan State University. Dr. Carter-Johnson is a member of the Michigan State Bar and the Washington State Bar. She is registered to practice before the U.S. Patent and Trademark Office.
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- Alexander Gaffney, ‘Right to Try’ Legislation Tracker, REGULATORY AFFAIRS PROFESSIONAL SOCIETY, available at http://www.raps.org/Regulatory-Focus/News/Right-to-Try/.
- Tracy Seipel, ‘Right To Try’ Bill: Brown Rejects Proposal to Let Terminal Patients Use Unapproved Drugs and Devices, SAN JOSE MERCURY NEWS, October 12, 2015, available at http://www.mercurynews.com/health/ci_28954390/right-try-bill-brown-vetoes-proposal-let-terminal?source=infinite-up.
- Elisha Sauers, Simonaire Drafts ‘Right-To-Try’ Bill for Dying Patients to Access Unapproved Drugs in Maryland, CAPITAL GAZETTE, October 20, 2015, available at http://www.capitalgazette.com/news/government/ph-ac-cn-righttotry-simonaire-1020-20151020-story.html.
- Goldwater Institute, The Right to Try Resources: Model Legislation, available at http://goldwaterinstitute.org/en/work/topics/healthcare/right-to-try/right-try/.
- U.S. Food and Drug Administration, The Drug Development Process, Step 3: Clinical Trials, available at http://www.fda.gov/ForPatients/Approvals/Drugs/ucm405622.htm.
- Coco Ballantyne, Strange but True: Drinking Too Much Water Can Kill, SCIENTIFIC AMERICAN, available at http://www.scientificamerican.com/article/strange-but-true-drinking-too-much-water-can-kill/.