Since early 2014, at least forty states have introduced “Right to Try” laws, and twenty-two of those states have passed “Right to Try” laws. The goal of these laws is provide patients with terminal illnesses access to investigational drugs, biological products, and devices which have not yet received full FDA approval. This article will discuss these “Right to Try” laws, the laudable goals behind such legislation, the federal regulatory context in which these laws exist, and some legal and practical obstacles to full recognition of the goals of this legislation.
FDA Regulation of Investigational Drugs
To understand the “Right to Try” laws, it is necessary to understand some of the basics of the federal regulatory process for bringing an investigational drug to market. The sale of drugs in the United States is regulated by the U.S. Food and Drug Administration (FDA). Before new drugs or biologics may be sold in the U.S., they must be evaluated by FDA. This evaluation process ensures that testing demonstrates the drug’s health benefits outweigh its known risks before the drug is approved for sale. This testing also serves to provide information to physicians and patients necessary to make educated decisions about the use of a drug.
Access to Investigational Drugs Through Clinical Trials
The most straight-forward way to access investigational drugs under FDA regulations is through a clinical research trial. Because clinical research trials involve human testing with investigational drugs, which by their nature have unknown efficacy and risks, clinical research trials are heavily regulated by FDA. A manufacturer seeking to conduct clinical trials must first submit an Investigational New Drug (IND) application, which is necessary to allow a company to manufacture the drug and ship it across state lines for use in clinical trials. Clinical trials are typically conducted in three phases. A manufacturer typically may submit a New Drug Application (NDA) to FDA for marketing approval once a drug has completed Phase III trials.
Access to Investigational Drugs Through FDA’s Expanded Access (“Compassionate Use”) Program
FDA prefers access to investigational drugs be provided through clinical trials, because these trials may lead to approval, and wider availability, of effective drugs. However, FDA recognizes that patients who have a serious or immediately life-threatening disease or condition may not qualify (or have access to) clinical trials for a number of reasons, including:
- Specific Diagnosis
- State of Illness
- Inability to travel to clinical research site (due to advanced illness or location of clinical trial)
As explained by FDA’s website :
Under the Federal Food, Drug, and Cosmetic Act, a patient may seek individual patient expanded access (sometimes called single patient access) to investigational products for the diagnosis, monitoring, or treatment of a serious disease or condition if the following conditions are met.
- The person’s physician determines that there is no comparable or satisfactory alternative therapy available to diagnose, monitor, or treat the person’s disease or condition, and that the probable risk to the person from the investigational product is not greater than the probable risk from the disease or condition;
- FDA determines that there is sufficient evidence of the safety and effectiveness of the investigational product to support its use in the particular circumstance;
- FDA determines that providing the investigational product will not interfere with the initiation, conduct, or completion of clinical investigations to support marketing approval; and
- The sponsor (generally the company developing the investigational product for commercial use) or the clinical investigator submits a clinical protocol (a document that describes the treatment plan for the patient) that is consistent with FDA’s statute and applicable regulations for INDs or investigational device exemption applications (IDEs), describing the use of the investigational product.
Also under FDA’s statute, a sponsor or a physician may submit a protocol intended to provide widespread access to an investigational product. In this scenario, FDA will permit the investigational product to be made available under a treatment IND or treatment IDE if certain criteria are met. Learn more about treatment IND or treatment IDE.
Compliance with FDA’s expanded access or “compassionate use” program only permits manufacturers to provide investigational drugs; it does not require them to do so. Manufacturers may not be able or willing to provide investigational drugs outside of a clinical trial (for example, because there is not sufficient availability of the drug or because of a concern that use outside of the strict parameters of a clinical trial could delay FDA approval and, with it, broader access to the drug). FDA has taken recent steps to reduce the paperwork burden.
State “Right to Try” Laws
State “Right to Try” laws seek to make investigational drugs available to terminally ill patients whose physicians have determined that no currently approved and conventionally recognized treatments are likely to prolong the patient’s life. These laws are premised on the belief that a terminal patient has a fundamental right to pursue the preservation of his or her own life and that the government should not restrict a dying patient’s access to investigational drugs when the patient’s physician has determined that no government-approved therapies are available and that investigational drugs are the only remaining option.
“Right to Try” legislation has been introduced in at least 40 states, and it has passed in at least 22 states. Most of the “Right to Try” legislation has been based on model legislation prepared by the Goldwater Institute. Although there are some differences from state to state, each state’s legislation tends to largely track the model legislation. The key points of the model legislation are as follows:
- Permits, but expressly does not require, a manufacturer to make available to an “eligible patient” an investigational drug, biological product, or device, either without compensation or at cost.
- Defines “eligible patient” as an individual who has a terminal illness; has considered all FDA-approved treatment options; has received a recommendation from his or her physician for an investigational drug, biological product, or device; and has given written, informed consent for the use of the investigational drug, biological product, or device.
- Limits the definition of “investigational drug, biological product, or device” to those that have successfully completed phase 1 of a clinical trial and remains under investigation in an FDA-approved clinical trial.
- Does not require coverage of costs related to investigational drugs, biological products, or devices under any private or public insurance.
- Prohibits a licensing board or disciplinary subcommittee from revoking, failing to renew, suspending, or taking any action against a health care provider’s license based solely on the provider’s recommendations to an eligible patient regarding access to or treatment with an investigational drug, biological product, or device.
“Right to Try” laws have been wildly popular—legislation has been proposed in 40 states since early 2014 and passed in at least 22 of those states. These laws have the laudable goal of permitting dying patients (in consultation with their physicians), who have no government-approved treatment options, to weigh the risks and make the decision whether to try an investigational drug, biological product, or device without government delay or interference.
Despite the noble goals of these laws, there are significant hurdles to realizing any of the intended benefits of these laws:
- To the extent that these laws seek to make available FDA-regulated products in a manner expressly prohibited by FDA, these laws are almost certainly preempted. Moreover, “Right to Try” laws apply only to products that remain under investigation in an FDA-approved clinical trial. FDA exercises a great deal of discretion in the approval process. For this reason, preemption aside, how likely is it that a manufacturer would make a product available outside of an FDA-approved clinical trial without FDA’s express approval?
- Completion of phase 1 of a clinical trial does not provide any evidence of efficacy and only limited evidence of safety. It would be very difficult at this early stage of clinical testing for a physician competently to advise patients regarding the known or potential benefits or risks associated with an investigational drug, biological product, or device. Additionally, manufacturers who make such products available outside of clinical testing at this early stage risk potential liability.
- Use of investigational products outside of the strict parameters of an FDA-approved clinical trial could threaten the clinical trial and delay or stall FDA approval.
Since “Right to Try” laws began gaining traction in early 2014, FDA has made some efforts to ease the paperwork burdens associated with its expanded access (“compassionate use”) program. It may be that the practical and lasting effect of these state “Right to Try” laws is to demonstrate the popularity of such legislation and encourage change at the federal level.