Drug innovation is alive in America. The Federal Drug Administration (FDA) recently announced that it approved 59 new drugs in 2018, more than it has in any year in the last decade.
Many of these drugs will treat rare diseases. According to a Politico Pro (subscription required) analysis, 43 of the new drugs (73 percent) were in at least one of FDA’s categories “for speedy review for either treating an unmet need or being deemed a breakthrough therapy because they are expected to significantly improve on existing options.” Nineteen were designated “first-in-class,” which means they represent a new and unique way of treating a disease.
The FDA’s Dr. Janet Woodcock said, “Many of these new approvals will have a significant impact on improving—and indeed, saving—countless patients’ lives.” Dr. Woodcock also noted the FDA approved:
- The first drug to treat smallpox;
- The first of a new class of drugs to treat patients with HIV-1 infection who have failed other therapies;
- A new single dose treatment for influenza;
- A new drug to treat seizures in patients with the rare and severe forms of epilepsy;
- Three new drugs to treat patients with migraines;
- The first therapy to treat multiple sclerosis in children;
- New advances for patients with breast cancer, prostate cancer, lung cancer, and thyroid cancer and a variety of new drug therapies to treat blood cancers and other blood disorders; and
- Seven new biosimilars, which “will further help to create competition, increase patient access, and potentially reduce the cost of important biological drug therapies.”
According to the Independent Institute, the FDA also set a record last year by approving or tentatively approving 971 generic drugs. (Both the White House Council of Economic Advisers, President Donald Trump, and FDA Commissioner Scott Gottlieb have acknowledged generic drugs saved consumers $26 billion from January 2017 through July 2018.)
Unfortunately, as Northwestern University’s Dr. Craig Garthwaite and American Enterprise Institute Research Fellow Dr. Benedic Ippolito argue in a STAT opinion column, congressional and executive branch drug pricing proposals put the innovation we witnessed in 2018 at risk. That’s because policymakers misunderstand “the nature of early-stage drug development and the mobility of investment capital.” The two explain that, increasingly, early-stage research is undertaken by small biotech firms and startups whose funding comes from venture capital investors.
Venture capital investors who could take their dollars to other industries.
Garthwaite and Ippolito urge lawmakers to think about future patients as well. They argue, “[W]e can lower current drug prices but we’ll get fewer new drugs. That is good if you suffer from hepatitis C, but it’s not so good if you are waiting for a cure for Alzheimer’s disease or glioblastoma."