The Need for a New Regulatory Approach Toward Cell Therapy

What Problem Are We Trying to Solve?

  • Cell therapy—which involves the use of human cells to restore healthy function in the human body—represents one of the most promising areas for the next generation of groundbreaking treatments.
    • Cell therapies have been shown to halt the progression of degenerative joint disease in the knee or hip and restore function to a failing heart or damaged cornea.
    • Scientific advances in the field include applications in cardiology, neurology, oncology, ophthalmology, and orthopedics. There have been promising studies for those with Alzheimer’s disease, Parkinson’s disease, heart disease, diabetes, and cancer.
  • Despite the benefits of these treatments, cell therapies are generally not accessible to patients in the United States. This is primarily due to an outdated approach toward regulation within the U.S. Food and Drug Administration (FDA).
  • Europe and Japan have outpaced the United States in streamlining their approval pathways to recognize the unique benefits of using a patient’s cells to regenerate or repair their diseased or damaged bodies.
  • As a result, patients in our country are forced to seek treatments in other parts of the world. And increasingly, U.S. companies are also making their investments in these new innovative technologies elsewhere.

What Actions are Needed?

  • The current regulatory approach in the U.S. means that cell therapies are either treated like the practice of medicine-and therefore not regulated-or as drugs, requiring a pathway lasting on average 10 years and costing more than $1 billion.
  • A new regulatory approach is needed that will serve as a “middle ground” between the practice of medicine and the pathway ordinarily used for drugs.
  • Fortunately, a number for forward-thinking scientists, leaders, and policymakers are developing and advancing policies designed to address this problem. But more action is needed to assure that the United States has a regulatory pathway that can accelerate the availability of safe and effective cell therapy treatments and products for Americans.
  • Learn more about policies being developed by clicking here.