Just a few weeks ago President Donald Trump gave his State of the Union address in which he mentioned the story of a rare disease survivor, Megan Crowley.  This child was diagnosed at fifteen months old with Pompe Disease, a rare and serious illness.  Doctors claimed she was not expected to live past five years; however, her father fought and found a drug company who could develop a cure.  Megan lived and her story is used to give people hope. It also gave Donald Trump a reason he believes the Food and Drug Administration (FDA) should be abolished. According to Donald Trump, “Our slow and burdensome approval process at the FDA keeps too many advances from reaching those in need.” Although this one victim of disease was able to overcome the odds without the FDA, this is not the same result for everyone. 

In 2015, scientists at Duke Hospital engineered a polio virus that was shown to target Recurrent Glioblastoma, an aggressive tumor of the brain (Patel). CNN did a “60 Minutes” segment on the treatment of a sixty-year-old woman named Nancy Justice who was a patient awaiting the experimental drug. The drug was proven to have worked for patient Stephanie Lipscomb which gave hope to the Justice family. At the start, the polio virus broke down the tumor and it seemed as if Nancy would survive; however, seven weeks after the treatment Nancy was rushed to the hospital after the tumor had taken over half her brain (Pelley).  The inflammation was a side effect from the dosage of the drug which ultimately ended Nancy’s life. As we can see not everyone has a happy ending.

The job of the FDA is to provide regulations for drug use and to set standards that are deemed safe to society. When patients are allowed to use treatments that have not gone through proper testing, factors such as dosage amount can lead to problems in the treatment. In Nancy Justice’s case an improper dosage resulted in death. If it was not for the FDA, thousands of other patients seeking experimental drugs could potentially die as well. The argument surrounding this debate is whether or not terminally-ill patients should have to go through the FDA to gain access to experimental drugs. Some people such as Donald Trump believe the FDA is not necessary because of its inefficiency, while others believe the FDA is vital to the safety of the American people. Since there are some issues with the way the FDA has allowed patients to access experimental drugs, the FDA’s system to gain experimental drugs, called Expanded Access, should be altered rather than replaced. 

The FDA constructed a program called Expanded Access that allows patients who have terminal illnesses to gain access to investigational drugs that have not yet received formal product approval (Darrow et al). These treatments are only available to those who have exhausted all other means of gaining therapeutic treatments such as applying for clinical trials or trying a drug that is already approved (Darrow et al). One of the main problems with this program is the time it takes to gain approval for access to experimental drugs. According to Dr. Razelle Kurzrock at the University of California, San Diego, “It is common for doctors to spend hours on the phone with the FDA trying to get a verbal commitment before even submitting an application” (Bellina). In addition to time issues there are practical, legal, and ethical issues with Expanded Access.

The practical issues with Expanded Access are the pharmaceutical manufacturers’ cooperation with the FDA, patients, and physicians.  This includes the manufacturers’ drug costs and physician’s knowledge of certain drugs in terms of the process to obtain them. Manufacturers play a big role in accessing drugs since they are the ones who make them. They control costs, availability, and ultimately who gets early access. Manufacturing companies also have the potential to charge direct costs to their patients or insurers (Darrow et al). Direct costs are the full prices that are attributed to the production of a drug. This can sometimes be the manufacturing and shipping costs as well as monitoring and reporting costs in the case of a wide-spread trial with many people involved. According to the FDA, drug companies may request that the direct costs of a drug be charged to the patient due to the complexity of manufacturing, scarcity of a natural resource, or the large quantity of the drug needed (4). Since most insurance companies will not pay for investigational drugs, the price of the drug remains very high for patients to pay out of pocket (FDA 2).

Availability of the drug is also controlled by manufacturers. Manufacturers make a certain amount of a drug and choose whether the drug goes to clinical trials or Expanded Access. Since clinical trials lead to drug approval, many companies will put clinical trials first when it comes to who will receive the drugs first (Darrow et al). The risk of the derailment of a trial due to Expanded Access is another incentive for manufacturers to not provide drugs for Expanded Access patients (Darrow et al). Although manufacturers play a big role in Expanded Access so do the physicians requesting treatment for their patients. Often times, many physicians are unaware of certain drugs and how to obtain them (Darrow et al). Regulations require physicians to determine the risks of the drug and the benefits of using it. Since manufacturers choose what information is provided about investigational drugs, it is often difficult for physicians to make an informed decision regarding whether or not patients should be allowed access to certain experimental drugs due to a lack of information about the drugs (Darrow et al). 

Ethical issues with Expanded Access involve the patients’ right to act autonomously and to choose what course of action to take when they have exhausted all means of gaining treatment (Schuklenk 88).  Expanded Access keeps patients from being able to make all decisions concerning their health. Instead of a doctor determining the risks and benefits of a drug, many opponents of Expanded Access wish for the patient to make that decisions themselves. 

Legal issues with Expanded Access came into play when state Right to Try laws were adopted. Many people saw problems with the Expanded Access system early on in its implementation and believed that they should have control over what treatments they choose to use. Thirty-two of the fifty states have adopted these laws that contradict the rules and regulations within the Expanded Access Program. Since no clear law has been set out, manufacturing companies are sitting back and waiting to see what the FDA will do. There is no incentive for manufacturing companies to go against the FDA. Even if they did it can cause legal conflicts on a federal level. Flaws in the system are clear which is why the “Right to Try” movement came to place in May of 2014.

The new legislation mentioned previously, called the Trickett Windler Right to Try Act, was written to replace Expanded Access and to provide a way for patients to bypass the FDA. How this act works is it requires only a doctor’s approval and a manufacturer to make a drug for a patient to use. No FDA approval is necessary. This act, however, was only passed on a state level. Advocates, such a Mathew Bellina, a terminally-ill ALS patient, believe that if this bill were to be passed on a federal level the FDA would not be able to interfere with the implementation of these state passed laws. On September 28, 2016 the Right to Try legislation failed in Congress due to a lack of liability and obligation of stakeholders involved. This bill did not hold anyone responsible if something were to go wrong in the treatment. The bill clearly states, “A producer, manufacturer, distributor, prescriber, dispenser, possessor, or user of such a treatment has no liability regarding the treatment” (United States Senate). When there is no responsibility put on any of the stakeholders, what happens if something were to go wrong with the treatment? Who would pay for it? 

It is clear that those in favor of removing the FDA from all phases of accessing experimental drugs and those in favor of FDA involvement wish for terminally-ill patients to gain access to experimental drugs. Both feel that those who are dying should have more readily accessible treatments than what they have at the moment. There are many flaws with the Expanded Access system and everyone believes there are different ways to fix it. Some wish to completely cut the FDA out of the picture and create a new program with a clean slate, while others wish to rectify the system in place. However, when we break down the steps to gaining access it becomes more complicated. There are several stakeholders involved, important regulatory groups and organizations, and ethical dilemmas when it comes to drug approval. Taking out the FDA would also mean losing any financial responsibility for companies and safety for patients. This is why I believe the Expanded Access Program should be altered rather than replaced by a new system. 

The article written by Tim K. Mackey and Virginia J. Schoenfeld describes the controversy surrounding Expanded Access and the different stakeholders involved within the Expanded Access Program. Among these stakeholders are the manufacturing companies. Although the FDA claims to approve almost all of its applicants, it is the approval from manufacturing companies prior to submitting an application to the FDA that renders patients helpless. Before the FDA can approve a patient’s application, the patient must receive permission from a manufacturing company to use their drugs. Protocols are created by manufacturers to set standard dosing, monitoring, and reporting for all patients within a trial.  According to Darrow and others, it takes about 120 hours of human effort to prepare a single protocol for a drug, and even if a manufacturer was willing to devote this amount of time there may not be enough capacity for both Expanded Access and clinical trials. This is where the Right to Try legislation goes wrong. Many activists of the Right to Try legislation immediately push blame on the FDA for denying access to patients when in reality it is the companies who own the drugs that can put a halt in the approval process (Davidson). According to Lisa Chedekel, an investigative journalist for Boston University Today, the main problem with the policy in place right now is that corporations are making humanitarian decisions while being influenced by cost, investors, and the potential impacts on future drug approvals. With so many stakeholders involved, how can we put the blame on just the FDA? 

The main issue people have with the FDA’s system is a result of their ignorance to Group Expanded Access Programs (Rabourn). The FDA has created two types of Expanded Access trials- Group level Access and Single-Patient Access. The most publicized form of Expanded Access is the Single-Patient Access where a doctor writes a customized treatment plan for each patient. This process was originally created for rare diseases or special cases in which the patients case is different from all other treatment options for a specific drug (Rabourn). Since many patients are making requests to drug companies, it causes an ethical dilemma when deciding which patients should get early access to the drugs (Rabourn). This dilemma is why many drug companies say no and why thousands of patients are left unhappy with the Expanded Access Program. If more patients were grouped together in a single treatment plan, then drug companies would not be faced with such a dilemma and the entire group can gain access. 

Another improvement that has been made to the system is a shorter form for physicians who have to fill out Single-Patient Expanded Access forms. This form is designed specifically for Single-Patient Access and provides clear instructions to the physicians on how to fill it out. The time is takes for a physician to fill out a Single-Patient form has dropped from 8 hours to 45 minutes (Darrow et al). This is a significant reduction on the physician’s work load which will hopefully make them more willing to fill out the form. The FDA has also uploaded a question and answer style document to their website informing patients of all the possible ways to gain access to experimental drugs and a document listing out the regulations on how a patient may be charged for the drugs (Califf). 

Educating physicians and giving them all the tools they need to quickly and sufficiently fill out applications for their patients is the primary way to improve the Expanded Access Program. One way this can be accomplished is through an Institutional Review Board (IRB). An IRB’s main goal is to make sure physicians and patients are informed on every step in the process of receiving an experimental drug (Holbein et al 531). One of the main differences between the Right to Try Act and Expanded Access is the participation of an IRB (Holbein et al 532). Since Right to Try was created to exclude the FDA and almost all regulatory groups, it leaves vulnerable individuals open to unproven and sometimes dangerous drugs (Holbein et al 531). That is why the IRB is so important. It acts as a safe-guard between physicians and patients when it comes to administering these unapproved drugs (Holbein et al 531). According to Blair Holbein and others, abolishing the federal oversight will not expedite the process of getting safe drugs to patients, rather it is imperative to give physicians and staff the information they need to meet the Expanded Access and IRB requirements. 

In addition to educating physicians, it is also important for the patient to be informed of what the potential risks and benefits are of a particular drug. An expert in the field of bioethics, Udo Schuklenk, wrote about autonomy in one of his books. His main concern with the ethical side of this debate is the ability for a patient to act autonomously when requesting experimental drugs (Schuklenk). If a patient understands the implications that comes with using experimental drugs they are legally held responsible. However, according to Schuklenk, “This can lead to situations in which social factors render patients incompetent” (88). Since patients come from a variety of different economic, social, and educational standings, they do not all have equal access to information on experimental drugs. With this in mind, Schuklenk believes that the best way to inform all patients of the same information would be to have freely accessible databases. This would allow for patients and doctors of all socioeconomic backgrounds to have access to the same information. Although there are many online databases in use today, it would be helpful to have a single database on a public website that would contain a list of information of all experimental drugs that are available for experimental use. 

As we can see there are several issues with getting rid of the FDA and the regulations they provide. When legislation was written to try to get rid of them, it failed in congress. The FDA is an important agency when it comes to the health and safety of the people, after all, it is their job. Although completely eradicating the FDA is not the best decisions, there are still ethical, legal, and practical issues with the system that need to be fixed. With recent legislation failing to over throw the FDA, it is time for patients to listen to new remedies for the Expanded Access Program. As we can see it is not so easy to rewrite a program that took years to make. That is why my solutions to the program are feasible. 

In order to rectify the system there needs to be cooperation from manufacturing companies. They need some sort of incentive to provide drugs for Expanded Access patients in addition to clinical trials. Whether it is a monetary incentive from the FDA or a simple agreement that no unexpected outcome from an Expanded Access trial can derail an ongoing drug approval process. It is understood that the main goal of a manufacturing company is to create a drug that can be FDA approved and allowed to treat people all over the country. Having this agreement will allow manufacturers to live with a peace of mind knowing that their approval process will be unharmed and patients who are terminally-ill will be able to get the treatment they need. 

Patient ignorance should also not be a factor anymore. Everyone should have access to the same information regarding experimental drugs and how to obtain it. Every patient should be aware of the risks involved with taking these medications and if the benefits are significant. A freely accessible database is a great resource that could provide people of all socioeconomic statuses the same information and can help avoid any legal issues later on. Patients should also be informed of Group Access versus Single-Patient Access. Since Single-Patient Access seems to be the only option being promoted, it would help to inform patients of Group Access programs. Many people are unaware of these trials and may not know that other people are in the same situation as them. 

Lastly, it is vital that physicians receive the information they need when filling out applications for their patients. Having more access to information on the drugs they are requesting is important for physicians to make informed decisions. Since manufacturers still have control over what information is available, it is important for IRB’s and other regulatory groups to be involved with the approval process. All physicians should be required to have contact with an IRB or regulatory group and the time it takes for IRB’s to approve the process should be hastened with the help of the freely accessible database. Our main goal is to quicken the approval time for patients in need, while also keeping them safe in the process and hopefully these solutions will solve that. 

 