One plan’s journey with gene therapy

February 11, 2022

Blue Cross and Blue Shield of Kansas (BCBSKS) began preparing for gene therapy requests back in 2017, when Luxturna® (voretigene neparvovec-rzyl) was approved.

At the time, BCBSKS had no members with retinal eye disease (the condition Luxterna treats). But as the largest insurer in Kansas, serving 920,000 members, if a rare gene therapy was going to be prescribed in Kansas, it would probably go through BCBSKS.

Other high-cost, gene therapy treatments were in the pipeline. It was only a matter of time.

Kenneth Mishler, the insurer’s chief pharmacist, put together a CAR-T/gene therapy work group. His goal was to prepare plan leadership for the anticipated release of Zolgensma® (onasemnogene abeparvovec-xioi) in early 2019.

Zolgensma treats spinal muscular atrophy (SMA), a rare, genetic disease that affects approximately one in 11,000 babies. SMA affects motor nerve cells in the spinal cord, inhibiting the ability to move and breathe. Clinical trials of Zolgensma included only children under two years of age.

By this time in 2017, BCBSKS had four members on Spinraza® (nusinersen), a treatment for SMA that had just come out in 2016. Spinraza costs $350,000 annually (in addition to first-year loading dose of $700,000).

Mishler wanted BCBSKS to be prepared for Zolgensma. The multidisciplinary gene therapy work group included:

  • Medical director
  • Chief medical officer
  • Provider relations
  • Sales and marketing
  • Actuary
  • Risk management and
  • Customer service

Mishler and his team discussed medical drug policy for Zolgensma, working with Prime on model language. The team wanted Prime to coordinate contracting with the manufacturer. Prime was already in discussions with the manufacturer, Novartis Gene Therapy, on a value-based outcomes contract.

The advance work paid off

The FDA approved Zolgensma on May 24, 2019. The BCBSKS gene therapy work group reviewed the clinical studies and the FDA-approved criteria and adopted the FDA-approved criteria for Zolgensma. Finishing up just the few final details they didn’t have, BCBSKS worked with Prime, their pharmacy benefit manager, to complete a value-based outcomes contract with favorable outcomes terms.

Ken Mishler, Chief Pharmacy Officer, BCBSKS

Ten days later: The first call for coverage of the therapy was received by the customer service specialists on June 4, 2019. Mishler’s entire team was notified within hours.

The coverage decision: BCBSKS had an employer relations specialist on the gene therapy work group. That proved to be very helpful. The employer relations specialist reached out to the benefits manager at the impacted employer group to discuss coverage of the $2.1 million drug. The benefit manager had questions about the differences between Zolgensma and the current therapy, Spinraza.

BCBSKS employer relations specialist explained:

  • How stop-loss insurance would lessen the employer’s exposure to this multi-million dollar claim
  • The promising clinical results of Zolgensma

Details on the clinical summary

SMA is divided into 5 subtypes (0-4), based on age of onset and highest motor function milestone achieved. Type I is usually diagnosed during an infant’s first six months. It is the most severe and most common. In an observational study of patients with SMA1 (17 patients were followed for at least 12 months) the median age of survival without permanent ventilation was 10-1/2 months. For those who survived to 20 months, 92% required permanent ventilation.

Eleven patients treated with Zolgensma could sit unassisted for at least five seconds, ten for at least ten seconds, and nine for at least 30 seconds. All eleven achieved head control, nine could roll over, and two were able to crawl, pull to stand, and stand and walk independently. All eleven patients could speak and feed orally. None of the patients in the historical cohorts had achieved any of these motor milestones and rarely achieved the ability to speak.

The specialty pharmacy: Novartis Gene Therapy had limited distribution of Zolgensma to just two specialty pharmacies. As it turned out, a vice president at one of the two specialty pharmacies knew a director from Prime Specialty Pharmacy solutions. That facilitated a single letter of agreement. This saved time and mark-up.

Shipping: The manufacturer shipped the treatment direct to the hospital.

Manufacturer’s authorization: Provider relations was also on the committee. That helped in coordinating activities with the physician. They had a previous working relationship. The physician wanted to administer the drug personally. The provider relations representative helped to facilitate training from the manufacturer.

There are more than 100 centers of excellence around the country where Zolgensma can be administered. The physician had to be certified by the manufacturer to administer the drug. Only then could the drug be delivered from the manufacturer.


Baby getting examThe member’s treatment was administered at the local hospital. The hospital received the Zolgensma from a limited distribution specialty pharmacy. The hospital did not directly bill for the product. The contract was executed with no cost mark-up because of the work BCBSKS did to contract the rate directly with the specialty pharmacy.

The key for BCBSKS was its preparation and community ties

Like nearly all Blue Cross and Blue Shield Plans in their markets, BCBSKS is the market leader in Kansas. It has strong community ties. The interdisciplinary BCBSKS working group provided the expertise and connections to smooth bumps along the way. Capitalizing on existing relationships proved invaluable.

Ken Mishler and his staff have taken advantage of Prime’s rich resources in following the drug pipeline. Webinars, email updates, and Prime’s drug pipeline website, help keep him on top of what drugs are coming next and what’s ahead, six to 12 months from now.

Prime also hosts forums for where we bring together Blue Plans from across the country. At these meetings we look to both problem-solve and innovate.

What we’re working towards is creating a partnership perspective for all of us: health plans, plan sponsors, PBM leaders, pharma, and providers. When we can do that – to tackle issues like gene therapy sourcing, payment, tracking and financing – we’ll be able to create a breakthrough model of care with the patient at the center.