Nearly 1,200 medicines are in R&D pipeline to combat expensive chronic diseases

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The emergence of preventative medicines will have a profound impact on the ability to improve patient outcomes and reduce healthcare costs associated with chronic diseases, according to PhRMA's latest Medicines in Development report.

The report highlights several promising advancements in the R&D pipeline aimed at reducing the burden of chronic diseases, including breakthroughs such as oral PCSK9 inhibitors for lowering cholesterol and monoclonal antibodies for conditions like asthma.

"This is just the tip of the iceberg," said PhRMA chief medical officer Dr. Mike Ybarra.

He pointed to the nearly 1,200 medicines in development – including 62 for heart disease, 99 for breast cancer (an all-time high) and 53 for depression.

"Despite this, drug development is incredibly challenging with only one in 10 potential new medicines often making it to patients, underscoring the need for a strong regulatory and policy environment that fosters and protects innovation," he said.

Ybarra said GLP-1s and GLP/GIP combination therapies are already being used to treat diabetes and obesity, but the emerging data is also showing the potential to prevent associated illnesses.

"For example, they could reduce heart attacks, strokes and even some cancers, improving long-term health outcomes," he explained.

WHY THIS MATTERS

In addition to better patient outcomes, these treatments could lower health care costs by reducing the need for more costly interventions.

Like GLP-1 medications, preventive medicines like monoclonal antibodies for asthma and PCSK9 inhibitors for cholesterol represent significant shifts in moving from treating a disease to preventing it.

Ybarra explained PCSK9 inhibitors have been shown to reduce cholesterol levels by up to 60% and prevent cardiovascular events like heart attacks by nearly a third.

Similarly, monoclonal antibodies targeting severe asthma have drastically reduced exacerbations, improving quality of life and preventing emergency department visits and hospitalizations.  

"Chronic disease is the largest driver of healthcare spending, and as such, preventive treatments targeted at these costly illnesses are what we should be incentivizing and expanding access to," Ybarra said.

THE LARGER TREND

When the onset of chronic diseases can be prevented the need for expensive long-term treatments is reduced, as is the risk of related comorbidities that further burden patients and the healthcare system.

"We know some of the most expensive care we provide is from the long-term damage caused by chronic disease," Ybarra said. "This includes costly hospitalizations, procedures and long-term care."

Healthcare professionals currently face several challenges to ensuring that innovative therapies, especially those in the preventive space, remain accessible and affordable for patients.

"As a migraine sufferer, I personally have benefited from the new and innovative CGRP inhibitors that offer a meaningful improvement over existing classes of treatments, but many patients first are required to try and fail on older medicines," he said.

Ybarra said that when innovative treatments like those for hepatitis C and PCSK-9s for high cholesterol first came to market, insurers deemed the treatments too costly, saying they would bankrupt the system.

"This rhetoric was not only misleading, but it created additional roadblocks and restrictions on patient access," he said.

Ultimately, case studies show that preventive treatments like these could save society hundreds of billions in healthcare costs by preventing comorbidities.

"These are the types of treatments we should incentivize and recognize the value they bring to patient health and the broader health care system," Ybarra said. "Delaying and denying access is penny rich and pound foolish."