Americans deserve the best medicines in the world. It is crucial that we, as elected officials, scientific experts, and healthcare leaders, leverage the power of American ingenuity to continue developing new treatments that allow patients with life-threatening and debilitating illnesses to live fuller lives.
In the past 25 years, nearly 1,000 new life-saving medicines were approved for Americans with conditions such as cancer, heart disease, and neurodegenerative conditions, such as Alzheimer's disease.
Just a few years ago, some of these treatments would have seemed like science fiction. Treatments for genetic disorders that impact infants and children, cures for hepatitis C, CAR-T cell therapies, and immunotherapies are possible because of the work of American scientists and researchers.
Therapies utilizing genetically targeted technologies (GTTs) are a perfect example of American innovation. These treatments, first studied in the 1960s, work by manipulating gene expression that can, in some situations, effectively turn off or “silence” the gene that leads to disease, thereby treating the disease upstream of conventional medicines. Two American scientists earned a Nobel Prize in Physiology and Medicine in 2006 for the discovery.
Medical treatments and cures made with GTTs have major positive impacts for patients with various diseases and disorders, some of which are hereditary. For example, Huntington’s Disease has no FDA-approved treatment and impacts more than 40,000 Americans. Huntington’s is passed down in families, and estimates show that more than 200,000 Americans could inherit the condition. GTTs hold the potential to silence the disease-causing gene so patients could avoid the neurodegenerative disorder they inherited. GTTs can also help patients with common conditions, such as high blood pressure and diabetes.
The future for genetically targeted therapies is endless, and they offer hope for conditions once thought untreatable. And these therapies are only in their infancy: the first GTT was only approved less than a decade ago.
With supportive policies, we could enter a golden era for developing the most innovative medicines Americans have ever seen.
We must work together to ensure the promise of GTTs comes to fruition. To that end, Congress needs to step up and take action by passing H.R. 1672, the Maintaining Investments in New Innovations (MINI) Act. The MINI Act, which we re-introduced this Congress, is a simple, technical policy fix that would help accelerate the development of genetically targeted therapies.
Current law separates drugs into two categories based on their Food and Drug Administration (FDA) approval process to determine when they are subject to price controls. This classification determines when medicines qualify for price negotiation under the Medicare Drug Price Negotiation Program. Small molecule drugs that the FDA approves as New Drug Applications (NDAs) become eligible for negotiations seven years post-approval, whereas large molecule drugs, known as biologics, become eligible at 11 years.
By distinguishing between the two types of therapies, Congress believed it could capture the cost to manufacture and the complexity of different types of treatments, including longer development and approval timelines. However, in doing so, some medicines made with GTTs are regulated as small molecules and therefore are placed on the same negotiation timeline as molecules that are much less complex and less costly to develop and manufacture.
Penalizing these groundbreaking therapies is not what Congress intended.
Simply put, for the purposes of Medicare negotiations, the timeline for negotiating treatments made with GTTs should align with that of biologics. The MINI Act is a necessary fix to the IRA existing drug classifications and would protect landmark American-made innovation needed to further the next generation of treatments and cures.
GTTs are the next frontier in American modernization and the future of U.S. healthcare. Development of these treatments can allow American patients to enjoy longer and better lives.
Without the advocacy of our colleagues in both the U.S. House of Representatives and Senate, the promise of genetically targeted therapies may go unrealized. We have come together across the aisle on this bipartisan legislation and are committed to its advancement in Congress.
Donald Davis is a member of the U.S. House, representing North Carolina's 1st Congressional District. Congressman John Joyce, M.D., represents Pennsylvania’s 13th Congressional District in the U.S. House of Representatives.
