Congress, Give Americans “Right to Try” Potentially Life-Saving Care

This article was originally published on April 9, 2017 in The Hill.

Lost in all the chaos surrounding the failed attempt to repeal the Affordable Care Act is a modest but important effort to reform healthcare. The Trickett Wendler Right to Try Act currently before Congress would achieve significant strides in making cutting-edge treatments available to patients who need them most.

Introduced in January by Senator Ron Johnson (R-Wis.), and co-sponsored by 45 other Senators including Joe Manchin (D-W.Va.) and Angus King (I-Maine), the bill is named after a Wisconsin mother of three who died in 2015 after a battle with ALS. Her daughter Tealyn poignantly explained why the legislation is necessary: without access to the newest treatments, “it feels like you’re stuck, like the government is in charge of your life and they haven’t been in your shoes either.”

The bill has two primary components. The first prevents the federal government from restricting experimental drugs or other medical products that are “intended to treat a terminal illness, provided that treatment is in accordance with state law, is approved/certified by the patients’ physician, and that the patient has exhausted all other possible treatment options.” This element of the bill would help give patients access to a broader array of medical options while preserving the ability of regulators to ensure these drugs are dispensed safely.

For the loosening of regulations to incentivize drug-makers to participate in “Right to Try” efforts, it’s important these companies are insulated from litigation or punishment for trying to help, and the second component of the legislation does just that. It would also prevent federal agencies from using any outcome of “Right to Try” use to negatively affect a drug approval decision.

For example, if a terminally ill patient — after exhausting all possible treatment options, getting approval from her physician, and following state law — used an experimental drug and subsequently experienced an adverse event, the FDA could not take that patient’s experience into account when considering whether to approve the product for wider use. Without the appropriate scientific methods in place, like those employed in clinical trials, determining the source of complications is problematic. Therefore, such cases should not influence the decision of regulators to green-light treatments for terminal illnesses.

Another patient who illustrates the importance of the legislation is Josh Hardy, a Fredericksburg, Va., cancer sufferer who contracted a viral infection after a bone marrow transplant. His family learned of an experimental drug that could help him, but the company refused to provide it, explaining that they did not have the resources to offer the drug to Hardy while still working on getting into a clinical trial that would benefit all patients. A social media campaign convinced them to provide the drug after all, but it was too late to be effective.

Virginia is now one of thirty states allowing drug companies to give unapproved drugs to terminally ill patients. There is also an existing federal “compassionate use” program that allows patients some access to life-saving remedies.

The problem with the current program, though, is that adverse events from experimental treatments are still taken into account by the FDA — regardless of whether the adverse event can be clearly attributed to the use of the drug. FDA reviewers should consider instances of compassionate use differently than actual clinical trials, given that there are no scientific controls in place to properly attribute cause and effect. Otherwise, the potential exposure from unfounded bias is a clear discouragement for the drug companies to participate in such programs.

The Right to Try Act improves on the current law by preventing FDA regulators from taking unclear compassionate use results into account when approving a new drug. Yet even this component could be strengthened: once the FDA learns of a result, it’s impossible to ensure that result won’t factor into an agency decision. To ensure unwarranted bias does not come into play, it would be preferable if the companies did not have to report instances of compassionate experimental use in advance of approval decisions.

While no law is perfect, the Right to Try Act is an important bill that deserves the bipartisan support it has garnered. It represents a rare opportunity for Congress to provide clear comfort to the suffering, and to extend — and even save — lives.

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Kyle Burgess is the co-founder of two social enterprises and has worked in strategy, communications, and program management for a decade. Kyle received her Master’s degree in International Relations & Economics from the Johns Hopkins School of Advanced International Studies (SAIS) and her Bachelor's degree in Political Science from American University.


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