In September 2016, the FDA granted approval for the first pharmaceutical treatment for Duchenne Muscular Dystrophy. This set off a firestorm concerning the quality of the evidence used for approving the drug. But in the current regulatory climate, barriers to clinical trial enrollment prevent those with dire health concerns from gaining access to potentially beneficial investigational drugs and devices.

For this reason, the FDA created the expanded access program (aka, compassionate use) as a path to treatment for individuals with life-threatening diseases.

While expanded access to investigational products is possible for small groups under specific circumstances, nearly all compassionate use is accessed through single patient Investigational New Drug applications. These single patient INDs are almost always approved by the FDA.

This sounds positive, but Zachary Simmons, MD of the Mayo Clinic discussed the shortcomings of the program at the American Academy of Neuromuscular and Electrodiagnostic Medicine (AANEM) meeting earlier this year. Simmons explained to attendees that an individual physician must complete an application for the patient, which takes about eight hours. The entire process consumes an average of 100 total hours of the physician’s time.

In addition, there are those who believe that these are unnecessary hurdles, and that individuals should have the autonomy to evaluate the merits and risks of a particular treatment on their own. This has spurred the Right to Try movement and resulted in Right to Try laws in 37 states. A Right to Try bill that has passed the U.S. Senate is now being considered in the House of Representatives.

Simmons points out that the Right to Try movement is driven by an unbalanced public discourse. On one side you have the strong media message highlighting personal stories of suffering and death. On the other you have more unemotional, data-driven arguments which are difficult to use for stirring public support.

Regardless, Simmons points out several ethical issues with Right to Try:

  • Informed consent may not be possible, as there is no Internal Review Board involved, risks are unknown, and data is held by manufacturers.
  • Patients and physicians lack appropriate training to assess the risks that are known, as physicians of any specialty can prescribe drugs via Right to Try.
  • The benefits are merely anecdotal in a study of N=1.
  • Right to Try laws may inadvertently limit enrollment in clinical trials.
  • The wealthy may benefit at the expense of others, as product manufacturers may still charge for their products, leaving those without money to vie for spots in clinical trials.

Still, even with these ethical concerns, the need for improved access to treatment for the terminally ill is great. Only a small percentage of individuals are eligible for clinical trials, and FDA Expanded Access presents a tall hurdle for physicians and patients. This is why the FDA receives so much public pressure to approve drugs like eteplirsen and Right to Try laws are on the books.

Many stakeholders are working toward improving the path to expanded access. The NYU Langone Health Working Group on Compassionate Use and Pre-Approval Access works to address these issues. This is an excellent resource for neurologists, who must often wrestle with the ethics of how best to help patients with limited treatment options.

As Simmons stated, it’s a balancing act. All stakeholders, including neurologists, must think creatively about how best to provide treatment for patients with little hope.