Real-world experience: Managing potential cures such as gene therapy

Many Spring 2020 conferences were canceled, postponed or looked drastically different because of the COVID-19 epidemic. The Academy of Managed Care Pharmacy (AMCP) annual spring event was no different as it moved to online, eLearning sessions. Brett Sahli, Pharm D, senior director of health outcomes and value at Prime and Ken Mishler, PharmD, chief pharmacist at Blue Cross and Blue Shield of Kansas (BCBSKS) presented a virtual session on gene therapies, their management and financial implications.

While a replay of the event is available, the following is a written synopsis.

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Gene and cell therapies are the “new ’drugs’ on the block” in the health care industry, first approved in 2017. According to the Food and Drug Administration (FDA), gene therapy products are biological products, regulated by the FDA using a similar framework as other prescription drugs despite the differences in their manufacturing or administration processes. Both biologics and gene therapies are similar in that they are derived from living materials such as cells or tissues.

These gene therapies are generally used to treat or cure orphan drug diseases and spending on orphan drugs has grown exponentially in recent years. The FDA doesn’t anticipate this slowing down. By 2025, the FDA predicts they will be approving 10-20 cell and gene therapy products a year.

With this increase in approvals and spend comes challenges to the existing system, explained Sahli. The most significant challenge is around the pricing methodology. Management options include:

• Annuity: converting a one-time high cost into multiple payments over two or more years.
• Outcomes- or milestone-based contracting: an annuity-style payment that is contingent upon a positive treatment outcome, with rebates available based on performance failure (i.e., efficacy, durability, safety, etc.).
• Risk-pooling: constant payments at a plan or employer level using standard reinsurance or new reinsurance, stop-loss, payment programs specific to these therapies.

For now, payers are likely to use the framework of their current payment system, adapting fee schedules and networks, and tapping into cash reserves – at least in the near term – while regulations and more sophisticated tracking systems catch up. Additionally, Prime offers options such as PreserveRx, a gene therapy reinsurance solution that offers protection to both health plans and employers from sudden, one-time costs due to covering ultra-expensive gene therapies.

Mishler took the audience through his health plan’s journey to prepare and then manage high cost gene therapies. It began with the Blue Cross Blue Shield Association organizing a gene therapy workgroup in late 2017. There were four main steps involved in their preparation:

1. Prepare for the drug therapy request: evaluated drug policy appropriateness with pharmacy benefit manager Prime, and determined what options existed for manufacturer contracting
2. Prepare the employer group: held critical, early conversations regarding decisions on coverage and payment
3. Strategize with the specialty pharmacy: limited distribution to only two specialty pharmacies
4. Address the need of the physician’s office, hospital and provider: coordinated activities, including billing and training, to help ensure site of care chosen was most beneficial for all parties.

BCBSKS’s experience shows that it takes a village and much communication to successfully manage gene therapy treatments.