Desperate for Hope, Americans with Rare Diseases Need a ‘Right to Try’ Too

Right to Try has been a literal lifesaver for many Americans with deadly diseases such as cancer and amyotrophic lateral sclerosis (ALS, or Lou Gehrig’s disease). A federal law that protects terminally ill patients’ right to access treatments that could save their lives but aren’t yet approved by the Food and Drug Administration (FDA), Right to Try gives new hope to the 97% of patients who aren’t fortunate enough to get into a clinical trial. It empowers them to try to save their own lives without having to beg the federal government for permission.

But these traditional options are often off-limits for the 1 in 10 Americans who have a rare disease. Fortunately, new technology allows medical experts to tailor-make the latest medicines for some of those individuals, based on their genetics. Yet because these treatments are personalized, they cannot go through traditional clinical trial review, and thus they are not covered by the current federal Right to Try law. Innovation is outpacing regulation, and these treatments can’t go through the FDA’s decades-old approval processes in a timely manner—processes devised long before technological breakthroughs enabled these new personalized options. So these patients are out of luck, and they need their own Right to Try.

Read the rest of the op-ed at Discourse Magazine. 

Christina Sandefur is the Executive Vice President of the Goldwater Institute.