AMAC Exclusive – By Andrew Abbott
While COVID-19 and developments surrounding therapeutics and vaccines dominated coverage of the medical world throughout 2021, the United States also saw a slew of new medical innovations and therapies for other ailments. Although the broad media landscape took shockingly little note of them, these innovations and life-saving interventions could help millions of suffering patients see substantive improvement in their lives.
Among the most notable breakthroughs was the announcement that the Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to a new drug, Enhertu (trastuzumab deruxtecan), which can reportedly “reduce the risk of disease progression or death by 72%” in patients with HER2-positive breast cancer, one of the most aggressive forms of breast cancer.
Despite billions of dollars spent on awareness, early detection efforts, and novel therapies, breast cancer is still the most common cancer worldwide. In 2020 alone, there were more than two million diagnosed cases, with considerably more likely going undetected. The disease also accounted for almost 685,000 global deaths that year.
On average, one in five of those two million cases were “HER2 positive.” Human Epidermal Growth Factor Receptor 2, also known as HER2, is a protein that promotes the growth of cancer cells. In HER2-positive breast cancer cases, the patient has extra copies of the gene. This means that the cancer is significantly more aggressive than in other cases. Being HER2-positive can dramatically affect a patient’s prognosis, treatment, and even survival rate. For this reason, HER2 is tested for very early and often in patients with breast cancer.
The new drug, Enhertu, developed by pharmaceutical corporation AstraZeneca, is for HER2-positive breast cancer patients who cannot have the cancer surgically removed, or have a metastatic cancer case where the cancer is spreading throughout the body. According to findings in clinical trials, Enhertu is 72% more effective at reducing the risk of “disease progression or death” than the current drugs used to treat HER2-positive patients.
Based on these promising results, the FDA in October granted Enhertu Breakthrough Therapy Designation (BTD). First approved for use in 2012, BTD allows the FDA to grant priority review to therapies that demonstrate “the drug may have substantial improvement on at least one clinically significant endpoint over available therapy.” It is one of several tools the FDA has to essentially “fast track” drugs whose early clinical trials suggest an immediate and significant effect on the treatment of life-threatening conditions.
AstraZeneca Oncology R&D Executive Vice President Susan Galbraith hailed the approval, stating, “This is an important step in bringing Enhertu as a potential new option in earlier lines of treatment for HER2-positive metastatic breast cancer, given the urgent need to improve outcomes. This recognition by the FDA underscores the transformative possibility of Enhertu.”
Although there is no specific timeline for when the drug may become available to all HER2-positive breast cancer patients, data suggests that drugs granted BTD spend two to three years less time in pre-market development and have shortened review periods as well. Enhertu is already available for breast cancer patients with more advanced HER2-positive cases, and BTD designation means it may soon be approved for more mild cases in the coming months.
Without these fast-track programs, drugs with incredible life-saving potential would otherwise get lost in mounds of bureaucratic red tape and might not get approved for years. As of August 2020, 298 drugs have been brought to market under BTD. As a recent example of what can happen without some measures to prioritize promising drugs, the failures of the FDA to quickly approve an effective rapid test for COVID-19 were primarily blamed on onerous red tape and other bureaucratic hurdles. Critics claim that were it not for these delays, the proliferation of the disease across America could have been significantly reduced.
President Donald Trump’s Operation Warp Speed was another example of how expediting approvals by removing unnecessary bureaucratic red tape can help save lives. Many legislators and doctors are hopeful that programs like Operation Warp Speed and breakthrough therapy designation will be adopted across the FDA with the hope of accelerating approval for other life-saving medical innovations like Enhertu in the future.
Andrew Abbott is the pen name of a writer and public affairs consultant with over a decade of experience in DC at the intersection of politics and culture.