Sickle Cell Gene Therapy Could Significantly Impact Medicaid Budgeting, Access

Patrick DeMartino, MD

Gene therapy, while among the most promising drug classes for the treatment of sickle cell disease, would significantly impact the Medicaid budgeting plans for eligible patients, according to a new study.

In a budget impact analysis of gene therapies—which have curative potential for sickle cell disease— team of investigators suggest affordability and accessibility may be limited in current circumstances allotted to US patients under Medicaid.

Investigators from the Pacific Northwest, led by Patrick DeMartino, MD, of the Department of Pediatrics at Oregon Health & Science University and Doernbecher Children’s Hospital in Portland, sought to interpret a short-term budget impact from gene therapy for the treatment of sickle cell disease among Medicaid program populations with the most disease prevalence.

As they noted, there are currently hundreds of gene therapies undergoing clinical assessment, with about 40-50 anticipated to reach the market in the next decade. Each will likely to be priced at more than $1 million per regimen.

“The unparalleled price and potentially curative single administration presents various challenges to the existing reimbursement system in the US,” DeMartino and colleagues wrote. “The assessment of value (cost effectiveness) and affordability (budget impact) do not need to occur in tandem and, in some circumstances, early consideration of affordability may be essential.”

They conducted their budget impact analysis from January through May 2020, for a sickle cell gene therapy from the perspective of 10 states’ Medicaid plans. Selected states featured the greatest prevalence of sickle cell disease among respective programs. Their observation included a 5-year time horizon, using state-level data for disease prevalence from 2012.

Eligible population was based on the inclusion criteria of clinical trials for the gene therapy, including those aged 13-45 years old with severe sickle cell disease.

For the sake of the assessment, the gene therapy cost was set at $1.85 million in the base case, plus baseline disease-related expenditures of $42,200 annually. The therapy was assumed to be administered to 7% of the eligible patient population annually and provide curative effect—meaning there would be no subsequent disease-related expenditures for the patient.

DeMartino and colleagues sought the total Mediciad budget impact and impact per member monthly in years 1-5. They used one-way sensitivity analysis to assess uncertainty of market diffusion, size of the eligible patient population, price of therapy, and the cost of routine sickle cell care.

From their assessment, they estimated 5464 Medicaid enrollees would be eligible for sickle cell disease gene therapy across the US. Among them, 2315 were in the 10 Medicaid programs of interest, or 16 per 100,000 enrollees.

The used model projected that the mean 1-year budget impact of sickle cell gene therapy would be just less than $30 million ($29,960,000) per state Medicaid program in the observed sample, or $1.91 per member monthly.

Applying a 5-year annuity payment to the outcome provided a reduced short-term budget impact, they noted.

In discussing the findings, the team emphasized that a commercially available and federally regulated gene therapy for severe sickle cell disease will likely present eligible Medicaid program patients with challenges in affordability.

“Although there is no threshold for budgetary significance for Medicaid plans, any new technology adding $1 to $3 per member per month is noteworthy, especially when applied to few enrollees,” they wrote.

What remains unclear, they noted, is whether this budget impact would require Medicaid plans to apply new access-limiting strategies in response.

“As a society, we would need to consider the lack of alternative therapies and inequity in research funding for SCD compared with other diseases,” they wrote. “Furthermore, the population with SCD may be especially marginalized and lacking the degree of organized advocacy seen in other disease areas such as cystic fibrosis.”

More research into potential reimbursement strategies is necessary, given the likelihood of continued affordability challenges in gene therapy treatment of sickle cell disease in the US.

“This may prompt Medicaid plans to increase revenue, restrict access, or establish alternative reimbursement methods,” DeMartino and colleagues concluded. “Gene therapy for SCD is likely to provide an early demonstration of the unique financial challenges associated with this emerging drug class.”