The COVID-19 pandemic has led to a cascade of healthcare innovation never seen before. In less than a year, multiple pharmaceutical companies were able to design, test, and mass-approve vaccines capable of preventing a new and deadly disease.
These modern medical marvels were produced faster and were far more effective than the experts could have anticipated. But as healthcare companies churn out more vaccines, other lifesaving medications may be left on the chopping block. Current Food and Drug Administration (FDA) approval procedures for gene replacement therapies remain far too onerous, resulting in preventable death and suffering. The Biden administration can ensure the COVID-19 vaccination campaign is not a one-off if they drastically rethink the country’s regulatory policies.
Every year, thousands of patients will be born with rare, debilitating genetic conditions for which there is no cure. USA Today reporter Karen Weintraub recently told the story of Danish 16-month-old Alissa Feldborg who was diagnosed with a fatal genetic disorder called Sandhoff disease. Even simple movements such as grabbing for toys effectively became impossible for Alissa. Fortunately, Alissa’s parents were able to enroll her in a University of Massachusetts (UMass) gene therapy trial. In January of this year, UMass researchers gave Alissa a full dose of gene therapy designed to repair the faulty genetic code responsible for Sandhoff disease. Thanks to this cutting-edge treatment, Alissa’s condition has improved considerably, and she can now move around, smile, and drink from a bottle.
While these trials are encouraging, an onerous regulatory process can prevent promising cures from reaching thousands of patients. In December, an experimental gene therapy for hemophilia B developed by uniQure (and partner CSL) was put on clinical hold by the FDA after a trial participant was found to have liver cancer. The regulatory agency found the revelation sufficient to put a pause on the gene therapy, even though the patient had multiple risk factors for liver cancer (including hepatitis B virus, hepatitis C, and signs of nonalcoholic fatty liver disease). After four months of investigation, the liver cancer was deemed likely unrelated to the gene therapy and uniQure was given the green light to resume testing. This regulatory delay was a needless setback for the tens of thousands of Americans suffering from hemophilia. In severe cases, the condition results in a death rate four to six times higher than the normal population, and can lead to complications such as chronic, debilitating joint disease. Gene therapy offers an opportunity to cure the disease outright, rather than subject patients to lifelong treatments that fail to fully alleviate suffering and reduced life expectancy.
The hemophilia gene therapy drama is eerily similar to the regulatory pause put on the Johnson & Johnson vaccine. The FDA and CDC raised alarms about the vaccine even though only 15 people out of nearly 7 million experienced blood clots after receiving the vaccine. The regulatory bodies eventually cleared the vaccine for use, but not before undermining the science, scaring a whole bunch of people, and giving the fringe anti-vaxxers another conspiracy to spread.
Unfortunately, these baseless regulatory edicts are all-too-common. According to a 2019 study by MIT researchers, FDA statistical standards are far too strict for medications geared toward severe diseases such as pancreatic cancer. Even if a promising medication could save the life of a seemingly terminal patient, the agency doesn’t want the negative publicity of something negative happening after an ill-advised approval. George Mason University scholar Alex Tabarrok notes, “the FDA has an incentive to delay the introduction of new drugs because approving a bad drug (Type I error) has more severe consequences for the FDA than does failing to approve a good drug (Type II error). In the former case, at least some victims are identifiable, and The New York Times writes stories about them and how they died because the FDA failed. In the latter case, when the FDA fails to approve a good drug, people die but the bodies are buried in an invisible graveyard.”
The FDA needs to reevaluate its regulatory standards and continue testing while monitoring potential harms from lifesaving cures. Recent breakthroughs with COVID-19 vaccines need not be isolated feats. Thousands of lives could be saved and made better with the right rules in place.
Ross Marchand is a senior fellow for the Taxpayers protection Alliance.
Catalyst articles by Ross Marchand