What Right to Try can teach us about cancer moonshots

Last week, President Joe Biden commemorated the 60th anniversary of former President John F. Kennedy’s historic moonshot speech in which he shared a vision for putting the first man on the moon. In his remarks, Biden reignited his original 2016 Cancer Moonshot effort to hasten progress in “ending cancer as we know it.”

It’s a deeply personal crusade for the president. He lost his son Beau in 2015 to glioblastoma, the rare and almost always fatal brain disease that has also taken the lives of his colleagues and friends, the late U.S. Sens. John McCain (R-Ariz.) and Ted Kennedy (D-Mass.), as well as thousands of Americans. That is why we should also celebrate the promise of new hope that is starting to be realized for patients diagnosed with the disease.

According to recently released real-life clinical data, a moonshot for this cruel and seemingly hopeless illness may already be in reach, thanks in part to a law known as “Right to Try.”

Until 2018, federal regulations made it difficult, if not almost impossible, for patients to access medications that have not been fully approved by the U.S. Food and Drug Administration (FDA). But in 2018, those barriers fell when Right to Try became the law of the land. The landmark reform allowed Americans with life-threatening or terminal illnesses to seek treatments — under their doctor’s care and recommendation, but without having to first beg the federal government for permission — that are considered safe enough to test on humans but have not received FDA marketing approval. And in 2019, Epitopoietic Research Corporation (ERC) became the first drug manufacturer to announce a formal Right to Try program to treat patients suffering from a glioblastoma, which, under the current standard of care, has a five-year survival rate of less than 7 percent.

Now, data shows that the Right to Try program is working, according to a new study recently published in the journal Frontiers in Oncology. In fact, with the Right to Try treatment, which includes the addition of a monoclonal antibody to the clinical trial protocol, the median overall survival was just under 20 months. That’s nearly triple the median survival figure of 6.6 months that had remained stalled for more than a decade, and it suggests a better outcome than the impressive results from the clinical trial protocol, which had a median survival of 12 months.

The study’s encouraging results contributed to the FDA’s decision last month to grant “Fast Track” designation to the treatment, making it eligible for an accelerated approval process. And the program holds promise for various other difficult-to-treat cancers too, as it’s considered a “platform” treatment that can aid in the development of other innovative therapies.

These preliminary results are undoubtedly great news for patients, their loved ones, and the medical teams who care for them. But they also offer an important proof of concept, demonstrating how Right to Try is now delivering on its original intent to expand and protect medical autonomy, while also accelerating the clinical development process.

But much work remains. Today, the latest innovative treatments are tailormade for individuals, using their unique genetic makeup. But the current clinical trial evaluation system, created more than half a century ago, operates almost exclusively on treatments intended for hundreds or thousands of patients.

If surgeons operated with 1960s technology, patients would die needless deaths. Yet, the FDA is operating with an outdated regulatory system that’s preventing patients from accessing the latest medical innovations.

It’s literally a matter of life and death. That’s why Right to Try should be expanded to include these individualized options, opening the door to even more treatments and further modernizing America’s drug approval process. These efforts may more quickly reach the approximately 30 million Americans, half of them children, facing 7,000 rare and ultra-rare diseases that are challenging to diagnose. Such diseases are often genetic, treatments for them are scarce, and illness is usually progressive and life-threatening.

For the original moonshot, the engineering to achieve the historic feat had to be designed and built from scratch. But our biggest challenges in achieving the goals of the cancer moonshot primarily lie in removing the government red tape and bureaucracy that hinder innovation. These barriers stand in the way of researchers’ discoveries, doctors’ experiences and patients’ treatment preferences — most of which do not require additional taxpayer investment and can be achieved in a manner that ensures patient safety and informed consent.

It is time for lawmakers to embrace the next important step in getting the right treatment, to the right patient, and at the right time.

Naomi Lopez is the vice president for healthcare policy at the Goldwater Institute think tank.