Insights & Resources

June 24, 2024 | Alerts

OIG Greenlights Gene Therapy Assistance Program

OIG Greenlights Gene Therapy Assistance Program

The U.S. Department of Health and Human Services, Office of Inspector General (OIG) posted a favorable Advisory Opinion (24-03) permitting a pharmaceutical manufacturer (Manufacturer) to provide financial assistance  to qualified patients undergoing its gene therapy treatment for two severe genetic conditions. This financial assistance can include travel, lodging, and stipends for meals and other authorized expenses. This favorable opinion is a welcome development for manufacturers and patients alike who are searching for ways to improve access to potentially curative treatments.

The Manufacturer’s U.S. Food and Drug Administration (FDA)-approved treatment involves a lengthy, six-stage process that it uniquely tailors to each patient.  Given the serious potential complications of this treatment, the Manufacturer partners with selected treatment centers nationwide who it determines are specially equipped to administer this treatment based on several objective criteria.  Patients must also meet certain eligibility criteria to participate in the Manufacturer’s financial assistance program (Program).

Although this Program implicates the Federal Anti-Kickback Statute (AKS) and the Beneficiary Inducements Civil Money Penalty (CMP),[1] OIG determined that it would not impose sanctions under these authorities because the Program has appropriate AKS safeguards and meets the “Promotes Access to Care Exception” under the CMP.  This is because:

  1. There are a limited number of facilities capable of administering Manufacturer’s gene therapy treatment.  The Program provides access to this treatment for qualified patients who would otherwise be unable to access or afford it.
  1. The Program increases patient safety and facilitates the FDA’s drug label instructions for the patient to remain at the treatment center for 4 to 6 weeks after receiving the treatment to monitor them for potential complications.
  1. Manufacturer’s treatment is a one-time potentially curative treatment, which reduces the likelihood that it would lead to additional referrals, steering, overutilization, or increased costs to the Federal health care programs in the future.
  1. The Program does not pay for any expenses covered by the treatment center, insurance, or third-party charitable assistance programs.

Moreover, Manufacturer certified: (5) it does not require physicians or treatment centers to prescribe or use its gene therapy treatment exclusively; (6) although its website contains information about participating treatment centers, it does not advertise or market the existence of the Program; and (7) it would perform a benefits investigation as part of its patient eligibility screening process to avoid providing duplicate support.

While OIG tends to look favorably upon patient assistance programs, provided that they have appropriate safeguards in place, entities considering such programs should proceed carefully to avoid running afoul of the AKS and CMP.

A complete copy of Advisory Opinion 24-03 is available at: https://oig.hhs.gov/documents/advisory-opinions/9914/AO-24-03.pdf

Should you have any questions regarding the above, please contact the authors, the Garfunkel Wild attorney with whom you regularly work, or contact us at [email protected].


[1] According to OIG, the Program generates prohibited remuneration to patients and treatment centers for which no AKS safe harbor applies.  Similarly, the Program generates prohibited remuneration to patients under the Beneficiary Inducements CMP.
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