If FDA can’t speed up drug testing, then give patients the right to try

Mikaela Knapp was in the prime of her life. After completing her studies at Stanford University and Berkeley College, Mikaela married her high school sweetheart, whom she had known since fifth grade. At age 25, she had her entire life before her. Then in an instant, it all changed. Mikaela was diagnosed with a rare form of kidney cancer and her world turned upside down. Unfortunately, this story doesn’t have a happy ending. After a long and hard fought battle, Mikaela passed away in 2014.

What makes her tragedy particularly difficult to accept is that it’s entirely possible to imagine a scenario in which Mikaela would still be alive today. Had Mikaela been able to take advantage of potentially lifesaving treatment, perhaps she would have beaten the odds. Unfortunately for Mikaela and so many others, we’ll never know because prohibitive rules and regulations block terminally ill patients from accessing treatment.

{mosads}The numbers tell the story. In the last 20 years, the Food and Drug Administration has approved only three new treatments for childhood cancers. It takes this long because any medical treatment must undergo rigorous testing to demonstrate that they are safe and effective. The FDA has eased some restrictions in recent years, but less than 3 percent of all cancer patients can enroll in clinical trials. What’s more, eligibility is tricky and tough to navigate. As the Goldwater Institute, a think tank supportive of easing the FDA’s requirements for new treatments, describes it, “patients must be just sick enough, but not too sick.”

To terminally ill patients and their grieving families, these policies are callous and devoid of the urgency needed when a life is on the line. We should not have to ask the government for permission to try to save our own lives. Terminally ill patients should have the right to try. After they’ve exhausted all available treatments, they should be able to work with their doctors and take part in clinical trials, without interference from government bureaucrats.

For some, this could mean accessing promising treatment already being used elsewhere. For instance, there are 22 breast cancer treatments awaiting FDA approval, some of which are currently saving lives in Europe. Knowing that there is a potentially lifesaving treatment outside the United States is what compelled the family of 10 year old Diego Morris of Arizona to travel to England to access Mifamurtide, a drug being administered to treat bone cancer which is not available in the United States. The treatment worked. Today, Diego is back in Arizona doing the things he loves.

Unfortunately, stories like Diego’s are the exception, but perhaps someday, they’ll be more common. Momentum for right to try laws is growing. In all, 37 states have enacted right to try laws and 12 more states have introduced legislation. In most cases, right to try has been approved by huge margins with overwhelming bipartisan support.

This is encouraging, but more is needed at the federal level so that the FDA cannot interfere with the implementation of state right to try laws. Even with the consent of their state government, many doctors and medical practitioners won’t administer experimental medical treatment to terminally ill patients because they rightfully fear that the FDA will come after them.

Federal right to try legislation has support in Congress, most notably from U.S. Sen. Ron Johnson (R-Wis.), whose bill to prevent this bureaucratic injustice recently passed the chamber unanimously. There are also encouraging signs that President Trump would sign right to try legislation if it got to his desk, but until then, terminally ill patients have little choice but to wait.

Right to try opponents worry that it would circumvent the FDA’s approval process and make it difficult for the government to keep track of the effectiveness of experimental medical treatment. But these worries are misplaced. Federal right to try legislation would not keep data and critical information from the FDA, but instead work with the government agency’s safety and testing approval process.

Also, the risk of inaction is far greater. As Matthew Bellina, a Navy Veteran suffering from Lou Gehrig’s disease, eloquently puts it, “What is the downside of creating new pathways for the terminally ill to access promising treatments? Maybe the law won’t help millions of people, or even many, but for those that it does help, it’s a game changer.”

It’s a question that Mikaela Knapp’s family must ask every day.

David Barnes is the policy director for Generation Opportunity.