Game-changing medical technology is on the line.
Based on Nobel-prize winning science, genetically targeted technologies take on the proteins that cause disease. The medications are administered once or twice a year to treat rare and debilitating diseases, from Duchenne muscular dystrophy to high cholesterol.
But the novel science behind these treatments needs time and investment. Only then can patients realize the maximum benefit.
That’s where Congress’ bipartisan Maintaining Investments in New Innovation Act, HR 5547/S 476, comes in.
Complex Medications Take Time
Known as the MINI Act, the legislation acknowledges an important fact: genetically targeted technologies are sophisticated. Similar to biologics, they require years of research and a highly complex manufacturing process.
That’s why the MINI Act would correct part of the 2022 Inflation Reduction Act that groups genetically targeted technologies with much simpler drugs.
Despite similarities between complex biologics and genetically targeted technologies, the two are treated differently by the Inflation Reduction Act. Biologics have 11 years before they come up for Medicare price negotiation, while genetically targeted technologies have only seven years.
The discrepancy could discourage investment in genetically targeted technologies just as they are taking off. Tweaking the provision, on the other hand, would allow more time for these medications to go to work for patients.
The Nobel Prize-Winning Science Changing Patients’ Lives
Only 11 genetically targeted technologies are available so far. They treat:
- Rare diseases like amyloidosis
- Inherited heart conditions like familial hypercholesterolemia
- Devastating pediatric conditions, such as spinal muscular atrophy
- Neurological conditions like Duchenne muscular dystrophy
The technology could yield even more meaningful treatment options in years to come.
For some patients, genetically targeted technologies offer treatment where there was none. Underserved communities in particular stand to benefit, as genetically targeted technologies treat several diseases that disproportionately impact Black Americans.
Meanwhile, people who struggle with taking a daily pill or regularly injecting themselves at home can benefit from a medication that’s infused or injected just once or twice a year. Making treatment simpler can increase patients’ likelihood of adhering to their treatment regimen – an issue that often undermines patient outcomes for heart disease and other conditions. New treatment options could also help underserved communities, whose social and economic challenges often lead to adherence problems.
To preserve these possibilities for patients, however, Congress must first pass the MINI Act. It’s a targeted bill that could yield big results for patients.
