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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References
- 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/.
MSU Bioethics 
There’s no ethical principle that divorces this question from that of homeopathy or any treatment which lacks adequate empirical evidence. The principle is the same. If people are willing to take on the risk omnipresent in the absence of evidence, there is no logical avenue through which healthcare providers can support a ban. Our role here is the same as always: to educate and help patients to choose the best choice in their own personal context. Changing the phase number is nothing more than a patch on a leaky pipe.
There are two basic differences between drug candidates in this situation and homeopathy – risk of harm and timing of the decision. First, homeopathy, while unlikely to help any disease context, is at least unlikely to hurt the patient to the same extent as one of these drug candidates. Second, these RTT laws specifically target patients at a particularly vulnerable time when they are facing death. I do agree that educating patients is a huge priority always.
Hmmm, you might be perceived as saying that:
1) If everybody cannot have a specific choice, then nobody should have that choice and that
2) If someone might make what we consider a bad choice, we should not let them make a choice.
You could also be perceived as buying into the fiction that drug companies should be able to charge whatever they want because of the “risks” that they take in developing new drugs. The high risk of drug discovery is now financed almost exclusively by the NIH and other tax supported agencies, and the drug companies are pretty much all making windfall profits.
I would advocate for patients who have not been adjudicated as incompetent to have the right to the information they need to make an informed decision. I would also advocate for drug companies being required to make trial medication available free under right to try programs.
Most of the cost of drug development is wrapped up in the FDA clinical trials which are run by the drug companies. As noted in my post, those trials are very high risk for the drug companies. While drug pricing is in need of reform, I’m not sure where the money for the trial medication would come from – especially since the cost of manufacturing small batches for the trials tend to be quite high.
This is indeed a complex issue, and I do agree that there are a number of potentially harmful effects. I understand the proposed solution of possibly making the Right To Try laws applicable to drugs entering in Phase 3 versus Phase 1, thereby making safer choices available to patients looking to go this route. I do think changes need to take place where patients have exclusions to healthcare coverage based upon their decision to take experimental drugs. Under the Right To Try legislation being passed in many states, there should be a clause which guarantees insurance coverage while taking experimental drugs. Just as insurance companies continue coverage for those on supplements not regulated by the FDA, so should they cover those taking experimental drugs.
I do have a problem with the outright exclusion of allowing patients the right to make the decision to try a drug that is not yet approved by the FDA. From a social worker’s perspective, it infringes on their right to self-determination. If a patient is fully informed of all of the potential risks as well as the benefits, they should have the right to do as they wish. Just as a patient has a right to refuse treatment they do not agree with, they also should have the right to seek alternative treatments when they feel they have exhausted all traditional options to no avail.
The insurance exclusion in many statutes seems murky. Some say that insurance coverage isn’t changed by the RTT law (though many don’t cover experimental drugs in the first place) while some say providers don’t have to cover medical issues consequent to taking a drug candidate. In the latter case, it is unclear if that means no coverage or just no coverage for side effects of the non-regulated drug.
Right to Try Laws present a very complex set of problems. I think it is important to note that desperate people do desperate things, and there is no shortage of individuals and companies that will take advantage of that desperation in order to make a profit. I think it is also important to note that these drugs are still in the trial stages and as the article states more than likely will provide false hope and could shorten an already limited life span. Also, as a social worker I believe that Right to Try Laws only benefit the wealthy. The poor will not have the resources to secure these drugs which are often not covered by insurance companies. So the question becomes if these laws spread how will we as a nation close the gap so that all patients that are willing to try these experimental drugs are able to do so at a rate that is affordable
Payment for these drug candidates is a huge issue if we want equitable access – these are often expensive. While the Affordable Care Act has attempted to provide insurance across the nation, insurance doesn’t usually cover experimental drugs. Government programs for access would further the misapprehension that these are approved therapies. Drug companies would prefer to enroll patients in actual clinical trials so that they can determine if the therapy can be approved.
I think that RTT laws present some very complex problems. As a social worker, I think that patients should have a right to self-determine. They should be given all the pros and cons, so that they can make an informed decision about whether an experimental medication might be a viable treatment plan for them. I think that in desperate times, people may make desperate decisions. They may not weigh the financial cost, as high as the opportunity of a “cure”. Some may think that no matter what the cost it is worth the shot. This could cause the patient and their family undue stress and the patient may die anyway.
One of the problems with the idea that patients receiving all the pros and cons to make an informed decision is that we don’t know any of that information at this point. The whole point of the FDA process is to develop that information for the patient. Many people choose not to take drugs that the FDA approves for their illness because the side effects outweigh a proven benefit for them personally – but at least they know the drug has been approved (or shown effective) for their illness in that case.
This article gave me a lot of insight about how the distribution of drugs can cause side effects that could kill a person before their illness does. I feel a person should only be prescribed a certain amount of drugs at a time so they won’t overdose, have many side effects, or make their illness spread to other places in their body. It is wrong for a patient to receive more drugs than they can handle. There should be a drug approval pipeline that only allows patients to get what they need but not large amounts. The way a person is feeling one day, good or bad can cause them to do anything with the drugs prescribed to them if its too much. I do believe if a person’s insurance can’t cover the drugs they are trying to receive then they should have to pay for them themselves. RTT laws protects the insurance company and the patient’s but it is always good for a person to have coverage. Companies should allow people medication free of charge for one month to see if its helping them or not. FDA should examine the drugs before handing them out to patients as well.
This article was good but I feel it could have given me more to work with. Statics or research could have improved this article. As far as the social work profession I think that the principle that ties into this is service. Social workers have to be willing to serve those in need especially their patients. I think with their help the distribution could be a little bit more simple and could cause better outcomes for the patients. Also, showing that you can bring in more resources shows that you want this patient to attain a better life.
I totally agree that more research needs to be done on this subject! I was limited to 1000 words in the blog post but I’m working on a longer full length piece that should address more concerns (I hope!).
The RRT laws are a step in the right direction. Drugs are tested on many people before they are approved and this is allowed. So why should it be any different for anyone else that wants to take the risk as those who enter into the drug testing protocol? I would have to question the motives behind anyone who would not want this to happen and ask what’s in it for them that they would seek to stop someone else to make their own decisions for their individual case. Just because something isn’t researched to the full level that the FDA decides is appropriate, doesn’t mean it isn’t a worthy drug. There are many alternative therapies that do not have the money nor the financial backing to have the same extent of gaining empirical evidence that these major drug companies do. This does not equate them to not being as effective. Rights are being violated to make decisions for one’s self, based on what others deem right for us.
Imagine the level of innovation that could occur in the drug market if the powers that be had to share in the equation. This would leave the political realm of the drug industry less monopolized.
We may actually have some competitive changes that treat the whole health of the person. I feel political agenda’s need to leave the drug market and more of a universal approach to all therapies be engaged and that people should be allowed to make decisions based on where they are at in treatment while being allowed to decide for themselves what is acceptable. Educating people before, during, and after treatment would be vital to achieving this properly.
I don’t think we have to imagine to see what would happen if the FDA stepped out of drug regulation process. Look to history before the FDA had the procedures. Tons of “innovation” was going on – much of it things like tonics that “cured” everything from baldness to cancer. Many of them cured by killing the person so there was no longer a problem. The default mindset really shouldn’t be in the absence of data the drugworks.
As for clinical trials – they are a necessary risk in order to gain evidence as to whether a drug works. The people enrolled should know that the drug tested has not been proven effective and that they are receiving it to gain data – not necessarily a cure. These RTT laws on the other hand are sold to people as access to drugs that have passed basic safety trials and just may not work – not true in all cases.
Additionally, these RTT negate much of the product safety laws that surround the drug industry. If you are seriously harmed by a drug (especially in clinical trials), there are ways to gain compensation and/or medical care, not so under RTT law usage.
Finally, as for my motives in questioning these laws, it is basic patient protection. Desperate people overlook real risks for a “hope” of a cure. These laws falsely enhance the perception that a given drug candidate works while at the same time they take all liability away from the drug company and the doctors. Alternative “therapies” already pose a similar problem for patients as far as determining effectiveness, and we should work on a solution to help patients to sort out which of those are effective. RTT laws do not impact that problem and may even make it worse if people get a mindset that all therapies are equally good even if they lack empirical evidence.
For years, terminally ill patients that can afford to travel to other countries have done so in order to obtain medications that were not yet approved by the FDA. The RTT laws would make drugs that have not yet gained FDA approval. While the RTT laws do not seem to make the drug more affordable, it would make them available for people that can afford the expense of uprooting their life and relocating to attain medication.
The issue of cost and who should pay for the medication to be available is frustrating. Many studies indicate how an individual’s socioeconomic status affects their health and well-being. If the RTT laws only offer the drugs to those that can afford them then it will not be possible to learn how the drug would affect individuals that are economically challenged. If we force insurance companies to cover these drugs than the cost of health care will increase for all of us.
It concerns me that only individuals who are terminally ill are going to be offered the opportunity to try the medication that is not yet approved. If only individuals that are at a final stage of an illness are able to attain the drug, then how do we know how effective the medication is at an earlier stage in the progression of the disease?
I waited a few days to do my blog, because I was really unsure about where I stood on this issue. We must make sure that are drugs are tested and safe. The article did not say how or if the individuals that would be able to obtain drugs under the RTT laws would be followed, so that the drug companies would be able to include them in research. I believe that individual’s should be allowed to be allowed to determine for themselves whether or not they want to try a medication that has not yet received FDA approval. However, even as I make this statement I realize that there are many questions that remain unanswered and perhaps ones that we have not even considered.
The patients who use drug candidates under RTT would not be included in clinical trial data. The trials are very specifically designed to determine effectiveness of the new drug candidate. For instance both the patients and the doctors are often unaware of whether they are getting the drug candidate or another approved treatment in order to prevent the placebo effect from giving the drug candidate a false positive results. Therefore, the RTT laws have the very real possibility of lowering clinical trial enrollment and the approval rates of the drugs that do work.
I have a friend that I have known since middle school. She was diagnosed with breast cancer very soon after she was married, at the age or 32. As if that wasn’t bad enough, it eventually moved into her breast bone, and continues to move through her body. She has been very public about her fight. Her campaign “Rock What You Got” and the foundation she has founded “Jills Wish” has been all over the media. She is probably one of the most influential people that I have ever met. She has never once complained about cancer, but has chosen to fight and keep living and spread her message to the world. But, I cannot help but wonder if drugs that were in stage 2 or stage 3 of clinical trials could have saved her from the cancer that is not ravaging her body. While I believe she will leave a huge mark on this world, I cannot help but wonder how much bigger it would be if; by the chance one of these new drugs were able to cure her, or at the very least have slowed the progression of her cancer.
I feel as though the medicine business is more about profit than saving peoples lives. Insurances companies are the same way. Thus allowing someone to have access to a drug that could potentially save a persons life, puts us at the mercy of both. If a person is already dying, they really do not have much to loose. Yes, they could loose their money, but maybe the answer is that these insurance and drug companies supply these stage 2 and stage 3 drugs for free until they are approved by the FDA. In doing so we have research and knowledge that we can use. Additionally people who have run out of options can at least help with the research and leave the world knowing that they have helped to either rule out a medication or rule it in as a potential treatment or cure. Yes there may also be some negative side effects, but if you are already dying and have exhausted all options more than likely you are already really sick, and dying. Yes there is more cost associated with worsening conditions, but you are going to get worse anyway. There is no predictor, of how each person will react to cancers progression. Therefor, it will be costly even if the disease progresses without the experimental drugs or without them.
I may be a bit biased given the situation with my friend, and given that we have so many cancers and other diseases that we have not cured, and research is slow; this could be a way of speeding up the process. It could be empowering for those willing to help try these medications to be able to at least leave the world knowing that they contributed to help curing or treating the disease that killed them so others will not have to die the same way. I would rather fight, and I think this is because I have seen my friend do the same thing everyday……Leave a good mark, don’t sit back and take no for an answer, or given in to a disease, you can accept you have it. But what you do determines if you die helping to cure it or die succumbing to not only your death but others who will die as a result behind you.
I am sorry to hear about your friend. I’ve recently watched some of my family members fight cancer, and it is so heart breaking. I agree that using your disease help try new drugs is noble – and the driving force for many enrolling in clinical trials. But know that patients that would use these drug candidates under RTT laws are not enrolled in clinical trials and produce no data that the drug companies can use to determine whether the product actually works. In fact RTT laws could divert people from clinical trials and slow research further.