Recently, President Donald Trump signed into law the Right to Try Act of 2018 (RTTA).1 The RTTA represents the federal version of laws previously passed by a majority of the states, but with important differences. This WSGR Alert highlights the history and background of issues leading up to the RTTA, and pertinent RTTA features and limitations.
Background
The purpose behind right-to-try laws is straightforward: terminally ill patients in the U.S. who have exhausted all available and approved treatment options, and who are not eligible to participate in an ongoing clinical trial of an investigational, unapproved drug, may apply to become eligible to receive an experimental drug—which may save or prolong the patients' lives. The vast majority of states have passed right-to-try laws, and there is a federal pathway for requesting and obtaining investigational drugs.
Historical Issues
Right-to-try laws, in practice, have historically presented patients seeking access to experimental drugs with numerous, and sometime insurmountable, obstacles. Pharmaceutical companies are not legally obligated to provide investigational drugs to terminally ill patients outside of a clinical trial that has been authorized by the U.S. Food and Drug Administration (FDA).2 In addition, many pharmaceutical companies have traditionally declined to provide experimental drugs to these patients for a variety of important and valid reasons, some of which are described below.
Because the supply of an investigational drug manufactured for clinical trials is necessarily limited, there generally will not be a sufficient supply of drug to treat patients outside of a clinical trial. In fact, for a variety of reasons, including cost considerations, drug development companies will tend to manufacturer only enough supply of an experimental compound to complete clinical testing. Manufacturing additional batches to cover RTTA patients generally is not a consideration.
Furthermore, adverse events occurring in patients who access the drug outside of an authorized clinical study must be promptly reported to the FDA, which in turn can delay, or derail, drug approval or license—thereby significantly impacting the broader patient population for which the drug will be targeted, and the drug developer's business. The RTTA does not eliminate the FDA's consideration of adverse events that occur in patients outside of an authorized clinical study as part of the drug approval process. But, as discussed below, the RTTA does attempt to lessen the impact of these adverse events during the FDA's review process.
Finally, product liability concerns have historically been a significant factor for drug developers when deciding whether to provide access to experimental drugs outside of an authorized clinical study. Whether insurance will cover an RTTA patient who is injured, or who dies after receiving the drug, and the adverse publicity that may arise as a result of a RTTA-associated adverse event or death, are additional factors that drug developers consider when evaluating whether to provide access to an experimental drug. As discussed below, provisions in the RTTA addresses potential product liability exposure to drug developers and healthcare professionals who are treating RTTA patients, and liability concerns should now be significantly reduced, thereby helping to eliminate legal barriers to RTTA access to experimental drugs.
In the past, gaining access to an experimental drug required engaging in a complicated, expensive, and most importantly, time consuming process that can take numerous hours to complete and involved: physicians, pharmaceutical company lawyers, extensive legal documentation, an Investigation New Drug Application filed by the treating physician and approved by the FDA, preparation of a patient informed consent, and review and approval by an institutional review board (IRB). Many patients are not able to surmount these hurdles. Some patients die while attempting to do so.
The RTTA
The RTTA was passed to attempt to address some of these shortcomings and to extend the scope of right-to-try nationwide.
Eligible Patients
First, to be eligible to access an experimental drug under the RTTA, a patient must meet certain defined criteria. Eligible patients must have been diagnosed with a life-threatening disease or condition;3 have exhausted approved treatment options; and be unable to participate in a clinical trial involving the investigational drug as certified by a physician.4 Eligible patients also must provide their treating physician with written informed consent regarding the eligible investigational drug.5
Eligible Investigational Drugs
Next, only certain drugs are eligible to become eligible investigational drugs. Investigational drugs can become eligible investigational drugs in either of the following two ways:
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the investigational drug must have completed a phase 1 clinical trial;6 not been approved or licensed for any indication; and be the subject of a filed new drug application (NDA) or biologics license application (BLA); or
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the investigational drug is the subject of an active IND; under investigation in a clinical trial intended to form the primary basis of a claim of effectiveness in a new drug application (NDA) or a biologics license application (BLA); and the active development or production is ongoing, has not been discontinued by the manufacturer, and not placed on a clinical hold.
Put succinctly, to be an eligible investigational drug, a drug candidate must be the subject of an NDA or BLA, or with some exceptions, be at the phase 3 stage of clinical development. Both sets of requirements, therefore, limit the number of experimental drugs that can be eligible investigational drugs.
Conditional Removal of Liability
The RTTA attempts to remove potential liability hurdles that could be the basis of preventing eligible patients from receiving eligible investigational drugs. For example, the RTTA recites that for any alleged act or omission with respect to an eligible investigational drug provided to an eligible patient, "no liability in a cause of action" shall lie against a sponsor, manufacturer, prescriber, dispenser, or other individual entity (e.g., a physician, clinical investigator, or hospital)—unless the relevant conduct "constitutes reckless or willful misconduct, gross negligence, or any intentional tort under any applicable State law." This statutory provision represents a significant, but qualified, waiver of liability.
Importantly, and consistent with previous precedents, no liability attaches against "a prescriber, dispenser, or other individual entity for its determination to not provide access to an eligible investigational drug."
Reduced Potential of Adverse Effects Negatively Impacting FDA Approval or Licensing
As described above, one important reason drug developers have traditionally been reluctant to provide experimental drugs to terminal ill patients outside of clinical trials is that if adverse events occur, these adverse events may significantly impact the FDA's review of the drug, and potentially cause the FDA to delay or deny drug approval or licensing. The RTTA attempts to allay this concern by explicitly reciting that the FDA "may not use a clinical outcome associated with the use of an eligible investigational drug . . . to delay or adversely affect the review or approval of such drug . . ." But the statutory prohibition is not absolute. The FDA may use adverse event data in its approval evaluation if the adverse event is "critical to determining the safety of the eligible investigational drug."
Interestingly, under the RTTA, if the data from using eligible investigational drugs in eligible patients is helpful to a drug sponsor, the drug sponsor may request that the FDA consider this data.
Reporting Requirements
The RTTA imposes a duty on sponsors to submit an annual summary of all use of each eligible investigational drug, and a separate duty on the FDA to post an annual summary report on agency's website.
Summary
The RTTA removes or attenuates some traditional barriers for eligible patients to receive eligible investigational drugs—including the potential liability and the impact of adverse effects on drug approval. But the RTTA does not guarantee that eligible patients will receive eligible investigational drugs, does nothing to address supply and demand imbalances, and cannot guarantee that eligible patients will benefit from taking eligible investigational drugs.
