Whether or not you agree with its position, there is no denying that in its recently released report, “Dead on Arrival,” the Goldwater Institute takes a passionate stance on the current status of the federal “Compassionate Use” program and its counterpart, the increasingly vigorous state-led initiative known as the “Right to Try” movement, a statutory phenomenon initially ignited by the Goldwater Institute, a private think-tank.
“Compassionate Use”, formally referred to as “Expanded Access”, is a process by which terminally ill patients can petition the Food and Drug Administration (“FDA”) and drug manufacturers for access to investigational drugs that have not yet been approved by the FDA. The process for accessing these investigational drugs has been criticized as time consuming and arduous. In addition to meeting a litany of eligibility criteria, applicants must find a physician who is willing to submit an application on their behalf, convince the drug manufacturer to provide the sought after treatment, and obtain approval from the FDA. Even if the FDA approves an applicant’s Expanded Access request, it cannot require a reluctant drug manufacturer to provide access to its investigational drugs. In response to these criticisms, the FDA has recently revised the Expanded Access application form to streamline the process and has reported data which show an extremely high approval rate by the FDA for Expanded Access requests.
Pulling no punches, the Goldwater Institute unequivocally states that the current Expanded Access program is challenging by design. The bulk of the Goldwater Institute’s criticism falls on the process the FDA uses to approve new drugs to market generally, outside of the Expanded Access pathway. The only way to receive approval to market a drug is through clinical trials, for which the FDA utilizes an “all or nothing” approval approach. In other words, if the clinical trial fails at any level, the drug will never hit the market. This encourages drug manufactures to be extremely risk averse and creates disincentives for companies to make investigational products available to patients in need. Companies don’t want to take any chances providing a drug through Expanded Access, lest a reportable adverse event (like death) experienced by a “risky” patient off-protocol bring the on-going trial to a halt.
In an attempt to bypass the federal Compassionate Use program, many states are developing Right to Try laws that are premised on the theory that terminally ill patients have a fundamental right to pursue the preservation of their lives by accessing investigational products. The Goldwater Institute drafted the model bill on which many of the state laws are heavily based. As of the date of this post, twenty-four states have passed Right to Try laws and twenty-six states have Right to Try laws on their dockets. In general, state Right to Try laws are structured to allow a terminally ill patient to access investigational drugs once the drugs have completed basic safety testing in a Phase I clinical trial. As with the federal system, none of the state laws, as currently conceived, would require manufacturers to make investigational products available unwillingly.
Yet, while the federal Expanded Access program may be riddled with inefficiencies, states’ Right to Try laws are riddled with inconsistencies. It is unclear (to say the least) how, if at all, state Right to Try laws will work in tandem with one another. For instance, while all state Right to Try laws are aimed at protecting “terminally ill patients,” many lack a solid definition of what “terminally ill” means. Louisiana, for example, defines terminally ill as “death expected within twenty-four months or less,” while Arkansas defines it as death within a “relatively short amount of time,” and Utah simply punts the decision to the applicant’s treating physicians. Moreover, under some state Right to Try laws, drug manufacturers are entitled to charge for access to the drug and insurance companies are not required to cover the costs, thus making treatment prohibitively expensive for many. Equally uncertain is the implication for Right to Try laws on the existing Expanded Access program. Can companies and treating physicians make products available through state Right to Try laws without consideration to the federal requirements? If they continue to provide access through the FDA Expanded Access pathway, in accordance with its terms, do they risk violating additional criteria that may exist at the state law level? These jurisdictional complexities may further disincentivize companies from engaging with these laws, an ironic outcome given the motivation of the Right to Try laws.
With so many terminally ill patients seeking access to potentially lifesaving treatments under the existing Expanded Access structure, the motivation behind Right to Try Laws is certainly understandable. Nevertheless, it is unclear whether state Right to Try laws are the solution to the challenges presented by the Expanded Access program, or whether they simply create new problems while replicating the existing ones. For example, while they claim to provide easier access to investigational drugs, under state Right to Try laws, drug manufacturers can still ultimately refuse to grant applicants access to their drugs.
If nothing else, perhaps the Goldwater Institute’s report will increase the dialogue regarding this important topic and will encourage a greater collaboration between states and the FDA in order to address the options available to terminally ill patients.