Federal Funding Drives Breakthroughs in Rare Diseases

For many years, the United States has prioritized biomedical research, trained the next generation of scientists, and accelerated the pace of disease-curing discoveries.  In the last 30 years, cancer death rates have dropped by 34% because of federally funded programs devoted to cancer prevention, early detection, and advancing treatment options. 

Maintaining strong federal support allows researchers to keep making progress, make accurate diagnoses,  expand access to clinical trials, develop new treatments faster, and provide resources for patients and caregivers.  

Read more about how federal funding fuels research.

The impact of rare disease

Rare diseases affect millions of people worldwide, yet each individual condition impacts only a small number of patients. This makes rare diseases difficult to study, understand, diagnose, and treat. For patients and families, living with a rare disease leads to uncertainty, frustration, and a lack of clear answers. Increasing awareness, expanding research, and improving access to specialized care are important steps toward bringing hope and better outcomes to the rare disease community. 

A recent study found that while most cancer trials are funded by industry, federally funded trials are more likely to focus on early-stage research, rare cancers, and cancers in children.  

These trials also tend to explore more complex treatment combinations and safer dosing approaches that can improve patient quality of life. 

Federal Funding helps support earlier diagnosis

Getting an accurate diagnosis early can be a challenge for people living with a rare disease. When there is limited research and awareness for a rare disease, symptoms are often overlooked or mistaken for more common conditions. When diagnosis is delayed, rare diseases can progress and may significantly impact a person’s quality of life.

Federal funding helps advance research, improve awareness, and speed up the diagnosis process so patients can receive the care they need sooner. 

Federal funding and clinical trials

Clinical trials help determine whether new therapies are safe and effective, and can improve the lives of people living with rare diseases. 

These trials bring together detailed information from people living with the same rare condition, giving researchers a clearer picture of disease patterns, treatment responses, and both short and long-term outcomes. 

Without consistent funding, many promising treatments never make it to the trial stage and, as a result, don’t make it to market where they are available for patients.  

Find a research study using HealthTree’s Clinical Trial Finder. 

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Sources:

The special role of federal sponsorship in the design and conduct of drug or biological agent cancer treatment clinical trials

ASCO Post: Federally Funded Clinical Trials Play a Vital Role in Cancer Research, Especially for Rare and Pediatric Cancers

American Cancer Society: Annual Cancer Facts and Figures