HHS-OIG Issues Favorable Opinion on a Pediatric Patient Support Service Program Supporting Access to Costly Gene Therapy

The U.S. Department of Health and Human Services’ Office of Inspector General (HHS-OIG) recently released Advisory Opinion No. 25-06, a favorable opinion regarding the federal Anti-Kickback Statute (AKS) and civil monetary penalty laws (CMP) against beneficiary inducements as applied to a narrowly tailored support arrangement, provided by a pharmaceutical manufacturer, for travel and lodge related expenses incurred by financially eligible pediatric patients and their caregivers receiving a one-time FDA-approved gene therapy at limited Treatment Centers. 

Background

The Requestor, a pharmaceutical manufacturer of a one-time autologous hematopoietic stem-cell based gene therapy (the “Product”), seeks to offer a financial assistance program offered to qualifying patients. The Product is approved by the FDA to treat certain early-onset forms of a severe genetic disease in children. Without treatment, life expectancy for affected children is significantly reduced, and supportive care has been the standard due to the lack of FDA-approved options.

Treatment with the Product is highly specialized and requires multiple stages, including consultations, stem cell mobilization and collection, customized Product creation, chemotherapy-based conditioning, infusion, and extended monitoring for recovery. Given the complexity of care and the limited number of qualified Treatment Centers, many families must travel long distances and stay near the centers for weeks at a time based on the treating physician’s clinical judgement. To address the access barrier, the Requestor proposed an Arrangement covering travel, lodging, and related expenses for financially qualified patients and up to two caregivers.

The Arrangement includes assistance with airfare or ground transportation, hotel lodging, and per diem support for meals and incidental expenses. Only patients who meet certain clinical and financial criteria, including lack of existing travel or lodging support and confirmation that the patient’s household income is below 600 percent of the FPL, are eligible. A third-party Vendor assesses patient eligibility verification through a detailed benefits investigation and manages reimbursement upon receipt of receipts to ensure compliance with safeguards that are in place to avoid duplicate coverage. 

HHS-OIG’s Findings

In this instance, HHS-OIG concluded that the proposed Arrangement implicated both the AKS and CMP. However, the agency determined that while the Arrangement constitutes “remuneration,” it would not impose administrative sanctions due to satisfying the “Promotes Access to Care” exception to CMP and the risk of fraud and abuse being sufficiently low. 

In reaching its conclusion, HHS-OIG cited the following safeguards and mitigating factors within the proposed Arrangement as diminishing the risk of fraud and abuse:

• The Arrangement helps patients access medically necessary treatment that would otherwise be geographically or financially inaccessible due to the limited number of qualified Treatment Centers offering the Product.
• Financial assistance is not provided when full coverage is available through Medicaid, the Treatment Center, or third-party charities. Patients who received partial assistance from another source, are only eligible to receive partial assistance to supplement the uncovered portion.  
• It enables patients to comply with physician-recommended extended hospital stays that do not interfere with clinical decision-making allowing adequate monitoring for serious post-treatment complications, ensuring patient safety and effectiveness.
• Since all patients are minors, caregiver support is essential throughout the treatment for both recovery and emotional support, and without assistance, caregivers may not be able to accompany them which could lead to compromising the continuity of patient care.
• The Product is a one-time gene therapy that may stop or slow Disease progression, and it is likely that the Arrangement would not lead to additional referrals after treatment with the Product has ended, minimizing the risk of repeated inducement or long-term overutilization.
• The Requestor includes several safeguards, including verification of insurance coverage, income checks, and does not promote Arrangement to physicians or caregivers as a reason to prescribe the Product, to reduce fraud and abuse risks.

As a result of these factors, the HHS-OIG determined that while the Arrangement may influence a beneficiary’s selection, it meets the “Promotes Access to Care” exception under the CMP.  Further, the Arrangement improves access to critically necessary services, poses minimal fraud and abuse risk, preserves clinical judgement, does not promote overutilization, and enhances patient safety through treatment adherence and recovery monitoring. 

Therefore, HHS-OG concluded that the Arrangement does not constitute prohibited remuneration and will not be subject to sanctions.

Key Takeaways

In this Advisory Opinion, HHS-OIG determined that although the travel, lodging, and incidental assistance program implicates the AKS and CMP, the Arrangement includes sufficient safeguards to avoid sanctions. This opinion reinforces that patient assistance programs can be structured lawfully if they promote patient safety, eliminate patient barriers, and pose minimal risk of fraud and abuse.. 

© 2025 Barnes & Thornburg LLP. All Rights Reserved. This page, and all information on it, is proprietary and the property of Barnes & Thornburg LLP. It may not be reproduced, in any form, without the express written consent of Barnes & Thornburg LLP.

This Barnes & Thornburg LLP publication should not be construed as legal advice or legal opinion on any specific facts or circumstances. The contents are intended for general informational purposes only, and you are urged to consult your own lawyer on any specific legal questions you may have concerning your situation.