Gene and Cell Therapies FAQ
What is cell and gene therapy?
Cell and gene therapy is a medical treatment in which an individual’s missing, non-working or malfunctioning cell or gene is replaced with a new working copy.
How widespread is cell and gene therapy?
Cell and gene therapies emerged in the 21st century, with just 10 treatments approved by the federal Food and Drug Administration for use in the United States and some 2,000 therapies being evaluated in clinical trials for potential approval.
How do cell and gene therapies differ from traditional medical treatment?
A primary difference is that cell and gene therapies tend to be administered in a single dose instead of a patient requiring continued treatment for chronic conditions over their lifetime.
Are cell and gene therapies more effective than traditional medical treatment?
In cases where traditional medicine offers little or no hope for improving a patient’s condition, some cell and gene therapies have the potential to fully cure patients or greatly enhance the outlook of surviving their disease.
How much do cell and gene therapies cost?
Therapies currently available range from about $373,000 to $3 million. These one-time costs have the potential to actually be lower than the cost of a lifetime of medical attention providing little or no improvement in a patient’s quality of life.
Are cell and gene therapies covered by insurance or government health care programs?
Coverage decisions for cell and gene therapies tend to be made on a case-by-case basis and can vary from state to state on Medicaid and private insurance plans. Medicare covers some cell and gene therapies when they are administered in hospitals but not in outpatient settings.
How is cell and gene therapy research supported?
Advocates for patients and families facing specific diseases and chronic medical conditions are working with pharmaceutical and biotechnology research organizations and elected officials to help develop innovative payment systems, such as those that pay providers based on proven long-term results. Ohio Life Sciences (formerly BioOhio) has a Cell and Gene Therapy Industry Council working to increase support for these therapies.
Where is more information available?
The New Drug Development Paradigms initiative (NEWDIGS) was founded at the Massachusetts Institute of Technology in 2009. NEWDIGS is a collaborative “think and do” tank focused on enhancing the capacity of the global biomedical innovation system to deliver the right treatments to the right patients at the right time, in ways that are sustainable for all healthcare stakeholders. NEWDIGS designs, evaluates, and catalyzes health system innovations that are too complex and cross-cutting to be addressed by a single organization or market sector. Click here to access their “Paying for Cures” toolkit: https://newdigs.tuftsmedicalcenter.org/toolkit/.