Within the last two years, the U.S. has seen decades of hard work pay off in the form of three ground-breaking new treatments for cancer and genetic diseases—CAR-T therapy; a cure for a rare, inherited form of blindness and, increasingly, tumor-agnostic cancer therapies. These treatment approaches are part of a suite of breakthrough medicines changing the current paradigm for people with cancer and rare diseases.
Each of these therapies was first approved in the U.S., which currently leads the world in new medicine approvals. This is not a coincidence. Rather, it’s the direct result of an environment created to foster medical innovation, supported by a mix of policies, partnerships and a talent pool unique to the American ecosystem.
Here are three groundbreaking American biopharmaceutical innovations that are changing the trajectory of disease:
- CAR-T therapies, part of the intersection between gene and cell therapy, are helping to change the landscape for cancer treatments and giving hope to patients who previously had no options. Short for chimeric antigen receptor T cell, CAR-T is a method of collecting a sample of white blood cells, genetically altering them to seek out and attack certain cancer cells, and injecting them back in the body to act as “smart bombs” that selectively target a patient’s cancer. In 2017, the U.S. Food and Drug Administration (FDA) approved the first two CAR-T therapies for certain types of blood cancers. It was not until a year later these same medicines were approved in Europe, meaning patients in other countries had to wait an extra 12 months to receive access. Even after EU approval, patients in locations like the UK were required to wait several additional months, due to more government hurdles.
- Gene therapies offer another example of the dramatic shifts that have occurred in today’s new era of medicine. In 2017, the FDA approved a therapy for a rare form of inherited blindness. This breakthrough cure, developed in the innovation hub of Cambridge, MA, is the first directly-administered gene therapy approved in the U.S. that targets a disease caused by mutations in a specific gene, reinforcing the potential of these therapies to treat many diseases caused by genetic mutations. But like CAR-T, the treatment did not receive EU approval until a year after it was approved by the FDA, and additional country-level approvals are not expected until 2020.
- Tumor-agnostic cancer therapies—which treat any cancer that has the specific molecular alteration targeted by the drug, unlike drugs that are approved to treat single specific tumor types or sites—show a similar story. These cutting-edge, promising medicines were first approved in the U.S.; at the time this article was published, they are still awaiting approval in Europe.
The U.S. innovation ecosystem excels at delivering the most advanced treatments and cures to patients desperately in need of new medicines. All the while, patients in countries with more government control are forced to wait months and even years for the same lifesaving options.
And yet, some in Washington are proposing policies that aim to move America’s ecosystem toward one that looks morelike Europe and other countries, not less, including setting prices for innovative medicines seniors receive in the hospital or doctor’s office based on the pricing policies of 14 foreign governments where patients have access to far fewer medicines. Proposals like these could mean many in the United States may find themselves with less choice and similar restrictions to those seen in Europe.
During one of the greatest eras of scientific progress, these policies would not only negatively impact biopharmaceutical innovation, they would also threaten patient access to the next lifesaving breakthroughs.