A little over a year ago, a prominent child neurologist published a commentary extolling the promise of gene therapies to "address thousands of rare diseases" and reveling that this branch of medical science could "transform medicine, alleviate disease burden, and ensure patients have the treatments they need."
As we entered early 2024, optimism about gene therapies' ability to address unmet medical needs and reshape our health care system into one that addresses the causes of disease instead of simply treating symptoms was abundant. the FDA approved two new gene therapies for sickle cell disease, bringing hope to those who have suffered for too long from this painful disease with no available solutions. And there was enthusiastic talk at the J.P. Morgan conference for health care investors about using gene therapies to virtually eliminate heart attacks.
In a very short time, though, that excitement has given way to concerns about the future of gene therapy and its path to meeting the needs of patients. It's not that the science has faltered. Far from it, research into targeted therapies for a wide range of diseases and serious health conditions remains robust. Rather, progress has been slowed by the lack of forward-leaning federal policy that keeps up with the science.
As a result, gene therapy companies are facing severe headwinds, forcing some to lay off employees, curtail expansive plans and discontinues several therapies in development. Several promising therapies in the pipeline are being put on ice for fear that the marketplace will not allow a reasonable return on the considerable investment necessary to develop these products.
The chilling effect of these changes results in fewer resources to invest in research, development and manufacturing of innovative therapies, and ultimately fewer patients gaining access to breakthrough treatments.
Meanwhile, patients are forced to stay on conventional, less effective treatments because they face too many obstacles in accessing potential one-time gene therapies that could profoundly change their lives. Young patients with Duchenne muscular dystrophy are being denied access to FDA-approved therapies by their health insurers because they are in wheelchairs. The federal government is telling patients with sickle cell disease that they can't be provided accompanying fertility treatments (infertility is a side effect of the therapy) forcing couples to choose between their health or starting a family.
All this said, there remains a lack of coherence in federal policy. For every positive initiatives like the MVP Act, which would provide clarity for manufacturers entering into value-based arrangements and ensure arrangements do not inappropriately distort Medicaid prices, there are federal programs like the FDA's Priority Review Voucher (PRV) initiative that have been maligned and misapplied. The PRV program is intended to incentivize manufacturers to forge ahead with their development programs, and yet one company with a promising therapy for sickle cell disease was denied a voucher. The PRV program itself expires in December, adding to the uncertainty paralyzing this sector - unless Congress acts.
By extending this program, Congress can help enable the development of more rare disease treatments at no cost to U.S. taxpayers. Other policy solutions like the Kids Access to Care Act would help reduce harmful treatment delays for children on Medicaid. Congress should advance these bills now to pave a path for the life changing potential that gene therapy poses for the future. In fact, more than 130 stakeholders recently sent a letter to Congress urging passage of these critical bills before the end of this year.
The science is working. Cutting-edge therapies are bringing hope to patients grappling with what were once viewed as uncurable, untreatable diseases. All stakeholders - regulators, legislators, and payers - must get this right so that our health care system can be transformed by one-time treatments that bring a lifetime of improved health to patients who need it most.
Congressman Erik Paulsen (MN-3) served from 2009 to 2019 as a leading member on the House Ways and Means Committee, which. has jurisdiction over healthcare, economic, and trade policy. Erik currently serves as Chairman of the Institute for Gene Therapies, a 501(c)(4) that brings together experts across the healthcare system to advocate for a modernized policy framework that encourages transformative innovations, promotes patient access, and codifies transparent reimbursement practices.
The Honorable Donna M. Christensen is the Chair of the IGT Scientific, Academic and Medical Council. Donna retired from the U.S. House of Representatives in 2015 where she served nine terms (18 years).
