HHS, CMS to Promote Model for Improved Access to Cell and Gene Therapies, Urge Accelerated Approval Trials to Completion

The Secretary of the Department of Health and Human Services (HHS) has selected 3 new models by the Centers for Medicaid & Medicare Services for testing, 2 of which directly affect cell and gene therapies, to help lower costs and improve access to novel therapies. The announcement comes after President Biden gave Executive Order 14087 in October 2022, “Lowering Prescription Drug Costs for Americans,” directing the HHS to work to lower costs.¹

“HHS is using every tool available to us to lower health care costs and increase access to high-quality, affordable health care,” Xavier Becerra, HHS Secretary, said in a statement.¹ “We are full steam ahead in delivering the cost savings from the President’s Inflation Reduction Act of 2022, and people on Medicare are already feeling the benefits. But as President Biden has made clear, we must build on the new prescription drug law with further action, which is why HHS is implementing these new projects to bring down prescription drug costs.”

The HHS selected 3 models to test. The first model will cap “high-value drugs” for chronic conditions like high blood pressure and diabetes while the latter 2 pertain to cell and gene therapies and other novel therapies. These are the Cell and Gene Therapy Access Model and the Accelerating Clinical Evidence Model.

"The 3 proposed payment models have the potential, if implemented properly, to make gene and cell therapies more accessible to all who stand to benefit from them," Jeffrey S. Chamberlain, PhD, Professor and McCaw Chair, Muscular Dystrophy, and director, Sen. Paul D. Wellstone Muscular Dystrophy Specialized Research Center, University of Washington School of Medicine, and president-elect, American Society of Gene and Cell Therapy (ASGCT), told CGTLive. "Access is a key component of ASGCT’s mission as advocates for the clinical application of genetic and cellular therapies. Ultimately, our hope is that these models improve both quality of care and access to transformative therapies."

The Cell and Gene Therapy Access Model will seek to create multi-state, outcomes-based agreements with manufacturers for some cell and gene therapies to help Medicare beneficiaries gain access these very expensive drugs. The Accelerating Clinical Evidence Model will seek to develop payment methods for drugs approved under the accelerated approval pathway. The HHS hopes the model will encourage confirmatory trials to complete in a timely fashion and to establish post-market safety and efficacy data to reduce spending on drugs with no confirmed clinical benefit.

“Prescription drug prices in the United States are the highest in the developed world, resulting in affordability and access challenges,” Chiquita Brooks-LaSure, CMS Administrator, added to the statement.¹ “The prescription drug law is making lifesaving prescription drugs more affordable for millions of people who have Medicare, and through the selected models, the Innovation Center will lower prescription drug costs and improve access for people with Medicare and Medicaid, ranging from $2 access to certain generic drugs to better deals for expensive new therapies.”

READ MORE: Gene Therapy for Sickle Cell Disease Cost Effective in Reducing Health Disparities

“These selected models will test strategies to make it easier for Medicare patients to afford and access needed prescriptions at $2 or less, help expand access to cutting-edge cell and gene therapies for people with Medicaid, and help ensure drugs already on the market are safe and effective,” Liz Fowler, PhD, JD, CMS Deputy Administrator and Director, CMS Innovation Center, added to the statement.¹ “We look forward to working on these models and helping to lower drug costs for Americans with Medicare and Medicaid.”

In another recent venture into finding ways to improve access to novel therapies to populations with high unmet needs, key opinion leaders including Chamberlain, published a paper in Human Gene Therapy that reviewed the use of micro-dystrophin as a surrogate endpoint for an accelerated approval pathway of Duchenne muscular dystrophy gene therapy trials.² They concluded that it is a valid endpoint that correlates with clinical benefits in preclinical mouse studies. Although the accelerated approval pathway may bring therapies to markets that have not yet proven their clinical benefit, the pathway is targeted at populations that often have no other disease-modifying options and in which any timely, potential benefit could help.

“The goal really was just to do a critical evaluation of the existing data from animal models and from the extensive clinical trials that are going on to make the argument that we have an update on predicting clinical benefit. We'd like the FDA to hopefully evaluate all that data, and we think they will come to the same conclusion,” Chamberlain told CGTLive in a previous interview.

REFERENCES

1. HHS Secretary Responds to the President’s Executive Order on Drug Prices. News release. Centers for Medicaid & Medicare Services. February 14, 2023. https://www.cms.gov/newsroom/press-releases/hhs-secretary-responds-presidents-executive-order-drug-prices
2. Chamberlain J, Robb M, Braun S, et al. Micro-dystrophin expression as a surrogate endpoint for Duchenne muscular dystrophy clinical trials. Human Gene Therapy. Published online January 24, 2023. http://doi.org/10.1089/hum.2022.190