In the United States, between 25 and 30 million people live with a rare disease, 80 percent of which are genetic. Genetic conditions are often inheritable, affecting the health and wellbeing of over 2.8 million children. Historically, these diseases have been difficult to cure and require ongoing treatment to mitigate symptoms and improve quality of life for Patients. There is good news on the horizon though: revolutionary cell and gene therapies (CGTs) are entering the market with increasing frequency and offer potential cures to Patients in need.
CGTs use a single dose of personalized medicine and target the source of a disease to cure certain genetic conditions, decreasing the overall financial burden of these conditions and increasing rates of remission for Patients. Broader access to CGTs represents a significant stride toward effective care for genetic diseases and cancers, such as lymphoma and sickle cell disease (SCD), which require significant healthcare resources to manage debilitating symptoms and prolonged treatments.
As of October 2024, the U.S. Food and Drug Administration has approved more than 30 CGTs, demonstrating the robust pipeline dedicated to delivering these breakthrough innovations to Patients.
Given that approximately 40 percent of CGT development happens in New Jersey, expanded access to these treatments will not only bring more cures to Patients, but promote greater investment into the Garden State’s thriving healthcare and manufacturing sectors. Nearly 76,000 New Jersey residents are employed by the biopharmaceutical industry. The State’s leadership in healthcare innovation and potential breakthroughs in CGT research will bolster New Jersey’s economy and could create thousands more jobs for State residents.
Aside from the benefits to our local life sciences industry, CGTs can increase Patient health outcomes and lessen the strain on the State’s healthcare system. The efficacy of CGTs significantly reduces long-term costs for both Patients and the healthcare system through dramatic decreases in inpatient stay, readmission, and infection relapse rates. One study of Patients with blood cancers who received CGT resulted in a 20 percent decrease in length of inpatient stay and a 14 percent increase in outpatient survival. Furthermore, CGT led to significant increases in survival and remission rates for lymphoma Patients compared to standard treatment regimens.
Because hospitalizations and readmissions are the largest drivers of healthcare costs for Patients and the broader health care system, CGTs can prove more cost efficient than standard forms of care by minimizing the need for long-term monitoring and additional care after initial treatment, reducing the financial burden on the Patient as well as federal and State health care programs. The American health system will be able to reduce expenses for genetic conditions in the long-term, such as the annual $2.4 billion Medicaid spend for SCD-related treatment. Healthcare providers will also be able to redirect newly available resources, such as blood products, hospital beds, and additional funds, towards other critical areas in need of support.
Improved health outcomes promised by CGT not only benefit Patients, but also their families and society through increased work productivity and economic gain. The advantages of CGTs include increased life expectancy and notably, a higher quality of life with less risk of relapse and increased ability to perform daily activities and social roles. With reduced healthcare costs and better health outcomes, Patients can return to work, support their families, and generate income for non-healthcare related expenses while stimulating the economy.
Removing access barriers to CGTs for Patients living with rare and other genetic conditions would be lifechanging – with the ability to cure debilitating diseases through a one-time treatment. Moreover, this booming treatment pipeline grounded in New Jersey’s existing life sciences ecosystem represents an opportunity to grow the state’s leadership in bringing revolutionary treatments from labs to the hands of Patients. It is critical that policymakers at the State and federal levels recognize the Patient, societal, and economic benefits of CGTs and advance policies that increase access and incentivize the development of these innovative treatments.
