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MINI Act Could Shield Patients from Unintended Consequences

The way a new drug is classified by the FDA could have unintended effects on patients’ treatment options.  So explains a recent paper about genetically targeted technologies. 

These sophisticated medications work at the genetic level, targeting proteins that cause disease. The first was approved in 2016 for patients with genetic muscular dystrophy. Others in the pipeline could treat cardiovascular, neurological and autoimmune disorders, as well as pediatric conditions and rare diseases with few treatment options.  

Genetically targeted technologies offer hope to patients who have little.  

They can also make treatment less onerous. Most genetically targeted technologies are injected only once or twice a year, allowing patients to incorporate treatment into routine medical appointments. For conditions like cardiovascular disease, where adherence presents an ongoing challenge, the shift could encourage disease management and provide a valuable option for patients.  

Classification and Congressional Action 

Genetically targeted technologies face a challenge, however. 

Due to the way they were categorized during the FDA’s review process, GTTs will become eligible for Medicare price negotiation just seven years after coming to market.  

The short timeline does not reflect the time, infrastructure and investment that these complex medications require. So it could unintentionally disincentivize future research and development – just as genetically targeted technologies are beginning to offer important treatment options for patients.  

MINI Act has Major Impact for Patients 

The bipartisan Maintaining Investments in New Innovation Act (HR 5547/S 476) offers a simple solution.  

Dubbed “The MINI Act,” the legislation would extend the timeline for genetically targeted technologies to become eligible for Medicare drug price negotiation to 11 years. The move would put these advanced treatments on par with similarly complex drugs.  

For patients, that could mean continued investment and research into technology that makes treatment simpler, more effective and – for some – possible for the very first time. 

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