Since last year, legislators in more than twenty states have introduced bills to make experimental therapies more easily accessible to patients with terminal illnesses, thirteen of which have been signed into law.
What's the Issue?
Under current federal regulations, patients with serious or life-threatening illness have two primary options to access experimental therapies that may treat their condition but that have not yet been approved by the Food and Drug Administration (FDA). The most straightforward path is to participate as a human subject in a clinical research trial. For patients who cannot be enrolled in that trial (because of their medical status or geographic location, for example), a second option is to apply to the FDA for access to the experimental drug under the expanded access (also known as compassionate use) program, which was created to allow seriously ill patients to access treatments they would not otherwise be eligible to receive.
In the past four years the FDA has received nearly 6,000 expanded access applications and denied just thirty-three. However, critics of the program argue that the process is too cumbersome and that it discourages many patients and physicians from applying. Since last year, more than twenty states have introduced laws aimed at making experimental therapies more easily accessible to patients with terminal illnesses, thirteen of which have been signed into law. These laws are often called Right-to-Try laws. Supporters argue that patients have the right to determine what risks they are willing to undertake to save their own lives and that these laws help reduce unnecessary federal interference. Opponents argue that such laws may introduce new risks for patients while undermining laws meant to protect public safety. The ultimate effects of these state-level efforts are still unclear, and their constitutionality is open to question.
It is unclear whether any patients have successfully accessed an experimental therapy under the state laws, although this may be as a result of low levels of awareness. However, Right-to-Try laws have proved to be politically popular, and there are likely to be more state laws passed in the current legislative sessions. It is also unclear whether the FDA will involve itself in any legal challenges to the laws that have already passed. However, in December 2014 the agency announced plans to establish an expanded access working group, which will develop policies to streamline the application process.
In February 2015 the FDA redesigned its website on expanded access and released draft guidance that introduced a new, simplified form (Form 3926) that physicians could use to apply for individual expanded access. This streamlined form is intended to address concerns that the traditional IND application is too burdensome for individual physicians to complete. The FDA estimates that the new form will take just forty-five minutes to complete, instead of the 100 hours required to complete a traditional IND application. The form is currently available for public comment and is expected to be finalized later this year.
At the industry level, efforts are under way to standardize and improve the way in which individual companies manage expanded access. Two major industry trade groups recently delivered statements on the issue and have developed standards and principles for their respective member companies to follow when developing their expanded access policy for a particular therapy.
Legislation was also introduced at the federal level late last year (HR 5805), with the goal of improving the existing expanded access program. If passed, the law would require companies to provide the FDA with more information on its process for dealing with expanded access requests. It would also direct two entities—the Government Accountability Office and a separate, multistakeholder task force—to evaluate the current program and provide recommendations for its improvement. Finally, the bill would require the FDA to finalize its current guidance on expanded access, taking these recommendations into account. The text of this legislation has since been incorporated into the House Energy and Commerce Committee’s discussion draft of the 21st Century Cures Act, which is likely to be introduced in the next few months. It remains to be seen how these various national-level efforts will impact patient decisions to pursue expanded access under state Right-to-Try laws.